The Size of The Cell and Gene Therapy Market

The global market for cell and gene therapy in 2020 is estimated to reach $3,866 million with therapies for cancer generating about 47% of the total market.  This is according to Kalorama Information’s latest report.  Oncology drugs — Kymriah, Provenge, and Yescarta — are primarily responsible for generating sales in the segment. Genetic disorder treatments Givlaari, Onpattro and Zolgensma are generating the majority of sales for other conditions.

The market for 2025 is estimated at $12,954 million and will more than double five years after to $29,960 million for 2030. Sales from oncology, cardiovascular-blood and musculoskeletal conditions will contribute strongly to market performance. Genetic conditions will also provide steady growth.

It is an exciting and interesting time to be involved in the cell and gene therapy industry. The science is moving ahead and now the industry needs to industrialize and standardize the manufacturing and commercialization of products. The industry is moving in the direction of a standardized set-up and a closed automated system.

Consequently, cell and gene therapy products are transforming the treatment of cancers and genetic diseases. Additionally, cell and gene therapies are expanding into other areas of medicine including autoimmune diseases, cardiovascular diseases, musculoskeletal disease, dermatological diseases and many others.

COVID-19 and Cell Therapy Cell and Gene Therapy Pipeline

There are a number of companies that are responding to the call to develop a therapeutic or vaccine for the coronavirus including:

  •  Vitro Biopharma offers its umbilical cord derived stem cells AlloRx stem cells and has a patent-pending and scalable manufacturing platform to provide stem cell therapies to COVID-19 patients.
  • Celularity and Sorrento Therapeutics entered into collaboration for CYNK-001, an allogeneic, off the shelf, placental-derived NK cell therapy. The companies on January 30, 2020, launched a clinical and manufacturing collaboration designed to expand the therapeutic use of Celularity’s CYNK-001 to COVID-19. Sorrento and Celularity agreed to assess
  • CYNK-001 as a potential novel therapy for coronaviruses, specifically SARS-CoV-2
  • CanSino Biologics is developing a vaccine for the prevention of COVID-19, Ad5-nCoV, a recombinant vaccine incorporating the adenovirus type 5 vector. On March 8, 2020, CanSino received approval from the Chinese authorities to being human trials. This is the first coronavirus vaccine to advance to Phase 1 trials.
  • Mesoblast is exploring Ryoncil (Remestemcel-L), an allogeneic mesenchymal stem cell product candidate in the treatment of COVID-19 patients.
  • Acturus Therapeutics is developing a vaccine that relies on engineering RNA. The company edits the RNA virus to encode for proteins that will protect against infection and load it into a liquid nanoparticle. The vaccine being developed in conjunction with Duke University and is in preclinical development.
  • Entos Pharmaceuticals is developing Fusogenix DNA vaccine developed using the Fusogenix drug delivery platform to prevent COVID-19 infections. Fusogenix drug delivery platform is a proteo-lipid vehicle that introduces genetic payload directly into the cells. Entos is working on developing an optimized payload containing multiple protein epitopes derived from SARS-COV-2 proteins, which will stimulate an immune response in the body to prevent COVID-19 infection.
  • Zydus Cadila announced the launch of an accelerated research program to develop a vaccine for COVID-19 using two novel approaches. The first approach includes the development of a DNA vaccine against the viral membrane protein of the virus, while a live attenuated recombinant measles virus (rMV) vectored vaccine will be developed in the second approach. The rMV-based vaccine works by inducing specific neutralizing antibodies, which will provide protection from the coronavirus infection.
  • AlloVir and Baylor College of Medicine have partnered to develop T-cell therapies against COVID-19.
    Athersys’ Multistem is a developmental cell therapy and according to the company and is the only therapy in development that targets ARDS in COVID-19 patients.
  • Exothera is developing vector-based gene therapies based on an Adeno Virus-based platform.
    Biopharmaceutical company Sirnaomics is hoping to use a gene-silencing technique known as RNA interference to turn off key genes in the new coronavirus. But first, the company must identify viral genes to target. “We are currently testing 150 of them using cell-based culture,” says Patrick Lu, Sirnaomics president and CEO. “We are working with groups in the U.S. and China.”
  • Mesoblast’s CEO Silviu Itescu stated that “remestemcel-L could be an effective treatment to reduce the high mortality in patients with COVID-19 who are older, have elevated inflammation biomarkers, and develop moderate to severe ARDS.”

Leading Influencers

The leading influencers in the market are those which have become first-to-market participants in the cell and gene therapy segment; have new developments which may disrupt current market conditions; and/or have an extensive pipeline sure to impact the market in the long-term forecast:
Advantagene

  •  Advaxis
  • AlloSource
  •  Alnylam Pharmaceuticals
  •  Amgen
  •  AnGes USA
  •  Anterogen
  •  Atara Therapeutics
  •  Athersys
  •  AveXis
  •  BioCardia
  • BioMarin Pharmaceuticals
  •  Bluebird bio
  •  BrainStorm Cell Therapeutics
  •  Caladrius Bioscience
  •  Celyad
  •  Cook MyoSite
  •  Cytori Therapeutics
  •  Dendreon
  •  Ferring Pharmaceuticals
  •  Fibrocell
  •  Gamida Cell
  •  GenSight Biologics
  •  Genzyme
  •  Gilead Sciences
  •  Gradalis
  •  Inovio Pharmaceuticals
  •  Kiadis Pharma
  •  Medeor Therapeutics
  •  Medipost
  •  Merck Mesoblast
  • Moled
  •  Neurotech USA
  •  Nightstar Therapeutics
  •  Northwest Biotherapeutics
  •  Novartis
  •  NuVasive
  •  Orchard Therapeutics
  •  Organogenesis
  • Orthofix
  • Osiris Therapeutics (part of Smith & Nephew)
  •  Pharmicell
  •  Pluristem Therapeutics
  •  Quark Pharmaceuticals
  •  Renova Therapeutics
  •  Sanofi Pasteur
  •  SOTIO
  •  Spark Therapeutics
  •  Tessa Therapeutics
  •  Tocagen
  •  uniQure
  •  VBL Therapeutics
  •  ViroMed

 

Advantagene, Inc.

-1: Advantagene Corporate Summary
Company Details Description
Address 117 Kendrick Street Suite 450
Needham, MA 02494
Country United States
Telephone 617-916-5445
URL www.advantagene.com
Source: Company website
Company Summary
Headquartered in Massachusetts, Advantagene is a private biotechnology company with a focus on cancer and immunotherapy products. The company was started in 1999 and works primarily in gene-based biologics using its Gene Mediated Cytotoxic Immunotherapy (GMCI). The technology can be applied to different solid tumors using a tumor-specific response and is designed to work with other therapies while preserving a good safety profile for patients.
Products
The company offers GMCI, which is a solid tumor immunotherapy that uses the modified common cold virus to infect tumors utilizing the herpes virus(tk). The TK protein is manufactured inside the cells. Then patients are provided with an activating agent that triggers an immune response against the tumor.
A second technology is the rQNestin34.5, a virus engineered for enhanced potency in cancer cells that enables the patient’s immune system to identify and destroy cancer cells.
In the pipeline, the company offers ProstAtak (aglatimagene besadenovec – AdV-tk) in Phase 3 clinical trials. ProstAtak kills tumor cells and stimulates a cancer vaccine effect. Killing tumor cells in an immune stimulatory environment induces the body’s immune system to detect and destroy cancer cells. The product is indicated for newly diagnosed intermediate and high prostate cancer cases. The estimated completion date for the study is December 2022.
A second Phase 2 trial is in progress to evaluate the effectiveness of ProstAtak in patients undergoing active surveillance for localized prostate cancer involving the use of AdV-tk to kill tumor cells and stimulate a cancer vaccine effect. The estimated completion date for the study is September 2020.
For brain cancer, the company is pursuing the use of AdV-tk injection into the tumor site and oral valacyclovir to kill tumor cells and stimulate the immune system. The study is in Phase 2 and is estimated to be completed in February 2021.
In NSCLC, AdV-tk is injected into the tumor followed by oral valacyclovir prodrug to kill tumor cells and stimulate a cancer vaccine effect. The study is in Phase 1b and is anticipated to be completed in May 2022.
For pancreatic adenocarcinoma, the company has a Phase 2 clinical trial to evaluate GMCI injection of AdV-tk into the tumor followed by oral valacyclovir to kill the tumor and stimulate a cancer vaccine effect. The study is estimated to be completed in December 2021.

Advaxis, Inc.

Address 305 College Road East
Princeton, NJ 08540
Country United States
Telephone 609-452-9813
URL www.advaxis.com
Source: Company website
Company Summary
Advaxis is a company located in Princeton, New Jersey that is dedicated to the discovery, development and commercialization of technology based on the Listeria monocytogenes. The company began as a part of Great Expectations, which was incorporated in June 1987. In 2004, the company changed its name to Advaxis.
Products
Advaxis is focused on the development of Lm technology, which is a Listeria-based immunotherapy designed for antigen delivery through a process of insertion of multiple copies of the proprietary tLLO-fusion protein into each extrachromosomal protein expression.
The company also was focusing on axalimogene filoisbac (AXAL) treatment for HPV related cancers. However, in June 2019, the company decided to close the AIM2CERV Phase 3 trial for advanced cervical cancer. The company has no plans to fund further studies using AXAL.
Advaxis granted Global BioPharma, or GBP, an exclusive license for the development and commercialization of AXAL in Asia, Africa, and the former USSR territory, exclusive of India and certain other countries. GBP is responsible for all development and commercial costs and activities associated with the development in their territories. GBP anticipates initiating its Phase 2 trial in HPV-associated NSCLC in patients following first-line chemotherapy in late 2019 or early 2020. The study will be assessing the effects of AXAL when combined with pemetrexed in patients with HPV+ NSCLC, following first line induction therapy
An additional program, ADXS-NEU, is an individualized Lm Technology antigen delivery product that uses whole-exome sequencing of the tumor to identify neoantigens. The company currently has two clinical trials:
— ADXS-PSA for treatment of prostate cancer in conjunction with Merck’s Keytruda. The study is in Phase 1/2
— ADXS- HOT for NSCLC for treatment of non-small cell lung cancer currently being evaluated in a Phase 1/2 clinical trial

AlloSource

Address 6278 Troy Cir
Centennial, CO 80111
Country United States
Telephone 720-873-0208
URL www.allosource.com
Source: Company website
Company Summary
AlloSource was founded in 1994 and is a manufacturer of cartilage tissue used for joint repair and skin grafts for severe burns. The company sports over 200 types of precise bone, skin, soft-tissue and custom-machined allografts.
Products
AlloSource offers AlloStem, a fat-derived mesenchymal stem cell combined with partially demineralized allograft bone. It is an alternative to autograft bone graft, as it offers three key properties necessary for formation of new bone: an osteoconductive natural scaffold, growth factors mediating osteoinductive signals and osteogenic stem cells.

Alnylam Pharmaceuticals
-4: Alnylam Pharmaceuticals Corporate Summary
Company Details Description
Address 675 West Kendall St
Cambridge, MA 02142
Country United States
Telephone 617-551-8200
URL www.alnylam.com
Revenues (2019) $166.4 million USD
Source: Company website
Company Summary
Alnylam Pharmaceuticals is a leading RNAi therapeutics company that is providing a new class of medicines to treat rare genetic, cardio-metabolic, hepatic infection and central nervous system/ocular diseases. Founded in 2002, the company has commercialized two RNAi therapeutic products and it has a deep pipeline of new products. The company employs over 1,200 individuals worldwide and is headquartered in Cambridge, MA.
Products
Alnylam offers several cell therapy products including:
— Onpattro( patisiran) which is a commercialized RNAi product for the treatment of hATTR amyloidosis with polyneuropathy. The company is continuing to pursue additional applications for the product. The APOLLO-B study is an on-going Phase 3 study for ATTR amyloidosis patients with cardiomyopathy. Product sales for 2019 totaled $166.2 million.
— Cemdisiran, which is an siRNA, RNA interference product that is for complement-mediated diseases. The product is in Phase 2.
— Fitusiran, which is an siRNA, RNA interference product that is designed for hemophilia and rare bleeding disorders. The product concluded Phase 2 clinical trials and Alnylam’s partner, Sanofi, is enrolling patients in the ATLAS Phase 3 program for fitusiran in patients with hemophilia A or B.
— Givlaari, which is designed for the treatment of acute hepatic porphyria. The product has received breakthrough designation and is on the market in the EU, in the US and in Brazil. The product revenues for 2019 totaled $0.2 million.
— Lumasiran, which is an RNAi product in development for the treatment of primary hyperoxaluria type 1 (PH1). The company-initiated NDA rolling submission to the FDA with remaining sections submitted by mid-2020. The company initiated the Illuminate-C Phase 3 study for the treatment of PH1 with advanced renal disease. The company also received a pediatric rare disease designation from the FDA for PH1.
— Vutrisiran, which is an RNAi product in development for the treatment of ATTR amyloidosis. The company initiated the Helios-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy.
— Inclisiran, which is an RNAi product in development for the treatment of hypercholesterolemia. The company submitted an NDA and MAA for the product.
— ALN-AAT02, ALN-AGT (hypertension) and ALN-HBV02 are other products in the RNAi pipeline . Alnylam is also coordinating with partner, Regeneron for ALN-APP for treatment of cerebral amyloid angiopathy and potentially other neurodegenerative diseases. ALN-HTT is in development for treatment of Huntington’s disease.

Amgen

Address One Amgen Center Drive
Thousand Oaks, CA 91320-1799
Country United States
Telephone 805-447-1000
URL www.amgen.com
Revenues (2019) $23.4 billion USD
Source: Company website
Company Summary
Amgen is a global biotechnology leader in the discovery, development, manufacturing, and delivery of innovative human therapeutics based on technologies in recombinant DNA and molecular biology. The company sells products around the world in approximately 100 countries. It is headquartered in California and has more than 20,000 employees. For future growth the company is focusing on global expansion, branded biosimilars, improving biologic drug delivery systems, and next generation biomanufacturing.
Products
Amgen offers Imlygic, an FDA approved viral therapy that is injected into melanoma tumors. Imlygic is a gene therapy solution that is made by modifying the HSV type 1. It attacks cancer cells and destroys them. This triggers an immune response.
The product is also being studied in combination with Merck’s Keytruda in patients with mid-stage to late-stage metastatic melanoma and is in Phase 1b.
Another product, AMG 119 CAR T cell, is in development for SCLC. The product is in Phase 1 clinical trials.

AnGes Inc.

Address 1F, Saito Bio-Incubator,7-7-15
Saito-asagi, Ibaraki
Osaka 567-0085
Country Japan
Telephone +81-3-5730-2641
URL www.anges.co.jp
Revenues (2019) $3.03 million USD
Source: Company website
Company Summary
AnGes is a Japanese company that was founded in 1999. The company is headquartered in Osaka, Japan and has 38 employees. The company was originally called MedGene Co but changed its name in June 2000 to better reflect its mission.
Products
The company’s lead product is HGF Plasmid (gene therapy) for the treatment of critical limb ischemia. The company received marketing approval for marketing the product in Japan in March 2019.
In February 2020, the company announced that it had enrolled its first patient in a Phase 2b clinical study in the US for HGF gene therapy product, beperminogene perplasmid, in patients with arteriosclerosis obliterans with lower limb ischemic ulcers.

Anterogen Co Ltd.

Address 130, Digital-ro Geumchum-gu
Seoul
Country Republic of South Korea
Phone 82-2-2104-0391
URL www.anterogen.com
Source: company website
Company Summary
Anterogen is a biotechnology company that researches, develops and commercializes cell therapy products and orphan drugs using adult stem cells. The company is located in Seoul, Korea and was founded in 2000.
Products
The company’s main products include:
— Cupistem is for the treatment of Crohn’s fistula by using autologous adipose-derived mesenchymal stem cells
— Quencell is for tissue damage

Atara Biotherapeutics

Address 611 Gateway Blvd, Suite 900
South San Francisco, CA 974080
Country United States
Phone 650-278-8930
URL www.atarabio.com
Source: company website
Company Summary
Atara Biotherapeutics is a novel company that develops allogeneic T-cell immunotherapy for patients with cancer, autoimmune diseases, and viral diseases. The company was founded in 2012 and has an active pipeline.
Products
Tab-cel (tabeleucel) is an allogeneic T-cell immunotherapy that is currently in Phase 3 clinical trials. The product is indicated for the treatment of patients with EBV+PTLD. In 2015, the FDA granted Tab-cel Breakthrough Therapy Designation for Epstein-Barr virus associated post-transplant lymphoproliferative disorder following HCT.
In October 2016, the product accepted into the EMA Priority Medicines pathway. The product has orphan status in the U.S. and EU. Tab-cel is in Phase 3 development for the treatment of EBV + PTLD and Atara has initiated a Phase1/2 study for Tab-cel in NPC.
The company has initiated a study of Tab-cel in combination with Merck’s Keytruda in patients with platinum-resistant or recurrent EBV associated with NPC
Other products include:
— ATA188 – has the potential to target EBV-infected B -cells and plasma cells in the CNS that may catalyze autoimmune response and MD pathophysiology.
— ATA2271- a mesothelin-targeted autologous CAR T for advanced mesothelioma in preclinical development
— ATA3271 – allogeneic CAR T immunotherapy targeting mesothelin in preclinical development
— ATA3219 – allogeneic CAR T immunotherapy targeting CD19 in preclinical development for B-cell malignancies.

Athersys, Inc

Address 3201 Carnegie Ave
Cleveland, OH 44115-2634
Country United States
Telephone 216-361-9495
URL www.athersys.com
Revenues (2019 thousands) $5,633 USD
Source: Company website
Company Summary
Athersys is a biotechnology company that is engaged in the discovery and development of cell therapy treatments.
Products
The company’s leading platform is MultiStem cell therapy, an allogeneic stem cell product. MultiStem is designed for treating neurological, inflammatory and immune, and cardiovascular diseases. The MultiStem allogeneic stem cell therapy has several pipeline projects including:
— Ischemic stroke and other neurological programs – MultiStem can be administered to ischemic stroke patients up to 36 hours after the stroke. The project , MASTERS-2 Study, is in Phase 3 clinical trials. FDA has granted this program a Fast Track Designation and EMA granted a Final Scientific Advice positive opinion.
— In Japan, the HEALIOS Treasure trial is ongoing with enrollment expected to be completed in 2020
— ARS – MultiStem shows promise in treating acute pulmonary inflammation in a Phase 2 trial. The program was granted Fast Track designation by FDA
— Transplant support – MultiStem is being evaluated for patient at high risk of GvHD.

AveXis, Inc.

Address 2275 Half Day Road, Suite 200
Bannockburn, IL 60015
Country United States
Telephone 847-572-8280
URL www. avexis.com
Source: Company website
Company Summary
AveXis is a biotechnology company that engages in the development of gene therapies for neurological disorders. The company was founded in 2015 and is located in Bannockburn, IL. AveXis is part of the Novartis family.
Products
Zolgensma is a gene therapy product that is approved for children under two years old with spinal muscular atrophy. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression. The product is given IV in a one-time dose that takes about 60 minutes. The product was approved in May 2019.
Other products in the pipeline include:
— AVXS-101- gene therapy for SMA Types 2 and 3 (in Phase 2 clinical trials – expected filing in 2020).
— AVXS-201 – gene therapy for Rett syndrome in preclinical
— AVXS-301 – gene therapy for genetic ALS-SOD1 in preclinical
— AVXS-401 and AVXS-501 – gene therapy for undisclosed treatment in preclinical

BioCardia

Address 125 Shoreway Road, Suite B
San Carlos, CA 94070
Country United States
Telephone 650-226-0120
URL www.biocardia.com
Revenues (2019) $689 in thousands USD
Source: Company website
Company Summary
BioCardia is a regenerative medicine company that offers treatments for cardiovascular diseases. The company operates in three product lines:
— CardiAMP cell processing system including CardiALLO
— Helix biotherapeutic delivery system
— Morph Deflectable Guide and Sheaths Product
Products
The company offers the CardiAMP Cell Therapy that is designed as a heart failure solution. CardiAMP therapy uses the patient’s own cells with a point of care platform to prepare the cells.
Projects in the pipeline using the CardiAMP system include:
— BCDA-01 in Phase 3 trials for ischemic heart failure
— BCDA-02 in Phase 3 for chronic myocardial ischemia with refractory angina
The company additionally offers the CariALLO Cell Therapy program that uses culture-expanded bone marrow-derived mesenchymal stem cells from a universal donor.
Projects in the pipeline using the CardiALLO system include:
— BCDA-03 in Phase1/2 for ischemic heart failure

Biogen, Inc.

Address 225 Binney Street
Cambridge, MA 02142
Country United States
Phone 917-679-2000
URL www.biogen.com
Source: company website
Company Summary
A global biopharmaceutical company, Biogen is focused on development and commercialization of therapies for patients with serious neurological and neurodegenerative diseases. The company’s focus is on MS, Alzheimer’s disease, dementia, SMA, ALS and Parkinson’s disease and ophthalmology.
In June 2019, the company acquired Nightstar Therapeutics, a clinical-stage gene therapy company focused on adeno-associated virus treatments for inherited retinal disorders.
Products
In February 2020, the company entered into collaboration with Sangamo Therapeutics to develop and commercialize ST-501 for tauopathies including Alzheimer’s disease, and ST-502 for synucleinopathies including Parkinson’s disease using Sangamo’s proprietary zinc finger protein technology delivered via adeno-associated virus.
The company has completed enrollment in Phase 3 gene therapy product, timrepigene emparvovec for treatment of choroidremia (CHM), a rare degenerative, X-linked inherited retinal disorder that leads to blindness.

BioMarin Pharmaceuticals

Address 770 Lindaro Street
San Rafael, CA 94901
Country United States
Telephone 415-506-6700
URL www.biomarin.com
Revenues (2019) $1.7 billion USD
Source: Company website
Company Summary
BioMarin is a biotechnology company that is engaged in developing and commercializing innovative therapies for life-threatening rare diseases and medical conditions. The company has an active pipeline along with providing several products for rare diseases including PalynziQ, Brineura, Vimizim, Kuvan, Naglazyme, Aldurazyme, and Firdapse. The company was founded in 1997.
Products
Valoctocogene roxaparvovec is BioMarin’s gene therapy product designed for the treatment of hemophilia A. It is administered in a single infusion and is designed to help the body produce factor VIII. The product was submitted to FDA in February 2020 for approval. Approval is expected in August 2020. A second study of Valoctocogene roxaparvovec for hemophilia A is in Phase 1/2 in the EU.
BMN 307 is a gene therapy product for PKU. In January 2020, both the FDA and MHRA granted BioMarin IND status and approved its clinical trial. The company expects to start dosing patients in PHEARLESS, a Phase 1/2 study in the first quarter of 2020.
BMN 331 is a gene therapy for hereditary angioedema (HAE). The company plans to begin IND studies in early to mid-2020.

bluebird bio, Inc.

Address 60 Binney Street
Cambridge, MA 02142
Country United States
Telephone 339-499-9300
URL www.bluebirdbio.com
Revenues (2019) $44.7 million USD
Source: Company website
Company Summary
bluebird bio is a pioneering gene therapy company working to develop gene therapies for severe genetic diseases and cancer. The company is focusing on a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing. Its strategy involves gene transfer, or the introduction of functional genes in replacement of mutated genes that cause disease.
Products
Zynteglo is a gene therapy treatment for treating TDT for patients who do not have a B genotype for whom hematopoietic stem cell transplantation is appropriate. The product is an autologous CD34 cells encoding B A-T87Q-globin gene. The product is approved for patients in the EU , Iceland, Liechtenstein, and Norway in 2019.
The company has several gene therapy products in the pipeline including:
— Lentiglobin for TDT Northstar-3 HGB-212 in Phase 3
— Lenti-D for cerebral adrenoleukodystrophy in Phase 2/3
— Lentiglobin for sickle cell disease in Phase 2/3
— BCL11A shRNA for sickle cell disease in Phase 1
— MPSI Gene Therapy for Hurler Syndrome in preclinical trials
— IDE-Cel (BB2121) for multiple myeloma
— BB 21217 for multiple myeloma
— MCC1 TCR for Merkle cell carcinoma

Brainstorm Cell Therapeutics

Address 1325 Ave of Americas, 28th Floor
New York City, NY 10019
Country United States
Telephone 201-488-0460
URL www.brainstorm-cell.com
Source: Company website
Company Summary
Brainstorm Cell Therapeutics is a developer of autologous adult stem cell therapeutics for neurodegenerative diseases. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. The company is located in New York City, NY.
Products
The company’s lead product, NuOwn, in development is clinical development for autologous MSC-NTF cellular therapy for ALS. The product is in Phase 3 clinical trials and has received FastTrack designation and Orphan Status from FDA and EMA. The MSC-NTF cellular therapy is also being developed for:
— Progressive MS in Phase 2
— Huntington’s disease in Preclinical
— Parkinson’s disease in Preclinical
— Autism Spectrum Disorder in Preclinical

Caladrius Biosciences

Address 110 Allen Road, 2nd Floor
Basking Ridge, NJ 07920
Country United States
Telephone 908-842-0100
URL www.caladrius.com
Source: Company website
Company Summary
Caladrius Biosciences is a clinical stage biopharmaceutical company that develops cell therapies designed to reverse cardiovascular disease.
Products
The company is developing a first-in-class cell therapy that is based on the premise that the body contains mechanisms for self-repair. The company offers three developmental projects for ischemic disease based on its CD34+ cell therapy platform:
— CLBS 12 under development in Japan for critical limb ischemia in Phase 2
— CLBS16 CD34 cell therapy for coronary microvascular dysfunction in Phase 2
— CLBS03 for the treatment of type 1 diabetes using the patient’s own Tregs in Phase 1

Celyad

Address 60 Broad Street, Suite 3502
New York, NY 10004
Country United States
Telephone 857-990-6900
URL www.celyad.com
Source: Company website
Company Summary
Celyad is a biopharmaceutical company that specializes in CAR-T cell therapy. In 2015, Celyad acquired OnCyte along with its oncology portfolio with CAR T-cell therapies. The company originally began as Cardio3 SA and in 2007 became Cardio3 BioSciences licensing the cardiac stem cell technology developed by Mayo clinic. In May 2015, Cardio3 BioSciences became Celyad.
Products
C-Cure is Celyad’s product candidate based on its cardiopoiesis platform being evaluated for heart failure. The research underlying this technology was originally conducted at Mayo Clinic C-Cure consists of a patient’s own cells harvested from bone marrow, treated with a combination of cytokines and growth factors and then re-injected into the heart. It is designed to enhance reparative capabilities in the failing heart.
The company has numerous products in development including:
— CYAD-01 in development for AML/MDS in Phase 1 (autologous)
— CYAD-02 in development for AML/MDS in Phase I (autologous) preconditioning with CyFlu
— CYAD-101 in development for mCRC in Phase 1 (allogeneic) TIM Technology
— CYAD-103 in development for solid tumors in preclinical (allogeneic) TIM technology
— CYAD-211 in development for multiple myeloma (shRNA Platform)
— CYAD-221 in development for B-cell malignancies(shRNA Platform)
— CYAD-231(shRNA Platform)

Cook Myosite, Inc

Address 105 Delta Drive
Pittsburgh, PA 15238
Country United States
Phone 855-867-9368
URL www.cookmysite.com
Source: company website
Company Summary
Founded in 2002, Cook MyoSite develops and commercializes technology relating to the collection, selection and expansion of human skeletal muscle cells for the treatment of various disorders.
Products
Cook MyoSite is researching autologous muscle derived cells (AMDC) and is involved in a number of clinical trials. The company’s clinical trials involve AMDC for:
— Stress incontinence in Phase 3 – the patient’s own cells are injected into the urinary sphincter
— Fecal incontinence – in Phase 1/2 – the patient’ s own cells are injected into the anal sphincter
— Underactive bladder – in Phase 1/2
— Tongue dysphasia – in Phase 1/2 – the patient’s own cells are injected into the tongue.

Cytori Therapeutics, Inc

Company Address 3020 Callen Road
San Diego, CA 92121
Country United States
Phone 858-458-0900
URL www.cytori.com
Source: Company website
Company Summary
Lorem Cytori develops and commercializes specialty therapeutics. In 2019, Lorem Vascular Pte. Ltd. acquired the global cell therapy business from the former Cytori Therapeutics, Inc. Lorem Cytori has been operating the cell therapy business in China, Australia, Singapore, Malaysia and Hong Kong since 2013. Lorem Cytori enables researchers and clinicians to study and administer cell therapies using its novel Celution System platform which harnesses the potential of stem and regenerative cells from adipose tissue.
Products
Cytori offers ECCO-50, through a process called adipose-derived regenerative cells (ADRC) using adult fat tissue. The cells are prepared with the proprietary Celution System platform technology and administered to the patient for a specific disease or medical condition all within one day. It is designed to repair, preserve function, improved quality of life and modify disease progression.

Dendreon Pharmaceuticals LLC

Address 1700 Saturn Way
Seal Beach, CA 90740
Country United States
Telephone 877-256-4545
URL www.dendreon.com
Source: Company website
Company Summary
Dendreon is an emerging biotechnology company focused on the development of targeted therapies for the treatment of cancer. The company was started in 1992 as Activated Cell Therapy, Inc. It was acquired by Valeant Pharmaceuticals in the past and is now changing parents once again, acquired by Sanpower Group, to resume operations as a standalone company.
Products
The company’s proprietary immunotherapy approach includes antigen discovery, antigen engineering and dendritic cell technologies that are combined to develop therapeutic vaccines that stimulate cell-mediated immunity – the body’s key defense against cancer. The company has collaborated with two other companies to develop emerging products for cancer treatment including collaboration with Genentech for the research, co-development and commercialization of products derived from Dendreon’s Trp-p8 platform.
Provenge (sipuleucel-T) was approved by the FDA for prostate cancer in April 2010 and has showed success in extending life in men with prostate cancer. At the end of 2018, Provenge had been prescribed to more than 30,000 men.

Ferring Pharmaceuticals

Address Ch.de la Vergogausaz 50
1162 Saint-Prex
Country Switzerland
Telephone +44 58 301 00 00
URL ww.ferring.com
Source: Company website
Company Summary
Ferring Pharmaceuticals is a specialty biopharmaceutical company that is dedicated to research and development of new products for reproductive medicine, gastroenterology and urology. The company was founded in the 1950s and is located in Switzerland.
Products
Ferring offers rAd-IFN/Syn3, a novel gene therapy for non-muscle invasive bladder cancer for patients that are unresponsive to BCG treatment. The product is in Phase 3 clinical trials.

Fibrocell (Castle Creek Biosciences)

Address 405 Eagleview Blvd
Exton, PA 19341
Country United States
Telephone 484-713-6000
URL www.castlecreekbio.com
Source: Company website
Company Summary
Fibrocell was acquired by Castle Creek Biosciences in September 2019. The company is located in Exton, Pennsylvania and is a gene therapy company researching and developing regenerative therapies for patients with rare and serious diseases based on its autologous proprietary viroblast technology.
Products
The company offers marketed product LaViv, indicated for the improvement of moderate to severe nasolabial fold wrinkles. It is an autologous cellular product for intradermal administration.
Fibrocell’s portfolio includes FCX-007, an investigational, late-stage stage gene therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a congenital and progressive orphan skin disease caused by the deficiency of the protein COL7. FCX-007 is a genetically modified autologous fibroblast that encodes the gene for COL7. By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing them and then treating wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas while avoiding systemic distribution. A Phase 3 trial was initiated recently, and if successful, a Biologics License Application (BLA) filing is expected in 2021.
Additionally, the company offers FCX-013, a gene therapy candidate for the treatment of moderate to severe localized scleroderma. FCX-013 is an autologous fibroblast genetically modified lentivirus and MMP-1. The product is in Phase 1/2.

Gamida Cell Ltd.

Address PO Box 34670
Jerusalem 91340
Country Israel
Telephone +972-2-6595666
URL www.gamida-cell.com
Source: Company website
Company Summary
Gamida Cell has focused its research and development efforts in the area of advanced cell therapies, including stem cells. The company has developed two distinct platform technologies to address diseases that may respond to stem cell therapy, including cancer, hematological diseases, autoimmune diseases, inflammatory conditions and regenerative medicine.
Products
Omnidubicel is the lead product which consists of NAM-expanded hematopoietic stem cells and differentiated immune cells, including T cells. The product has the potential to be a universal stem cell graft for those with leukemias and lymphomas who require HSCT but lack access to a matched donor and hematologic disorders such as severe aplastic anemia. The product is in Phase 3 clinical trials for leukemias and lymphomas and is expected to be completed in the second quarter of 2020. A second trial for hematologic disorders is in Phase 1/2.
The company is also applying the technology to develop GDA-201 for innate immunotherapy of expanded natural killer cells for cancer indication. NK cells are highly potent cytotoxic lymphoid cells that can kill tumor cells in the absence of prior sensitization by other components of the immune system. By expanding NK cells with the company’s NAM technology platform, it has the potential to increase the number and functionality of therapeutic NK cells targeting tumors. When GDA-201 is combined with targeted antibodies, there is enhanced antibody-dependent cellular toxicity, or ADCC. GDA-201 is currently being evaluated in an ongoing investigator-sponsored Phase 1/2 clinical study.

GenSight Biologics S.A.

Address 74, rue du Faubourg Saint-Antoine
75012 Paris
Country France
Telephone +33 176 54 72 20
URL www.gensight-biologics.com
Source: Company website
Company Summary
GenSight is a clinical-stage gene therapy enterprise that is focused on the development and commercialization of therapeutics for severe retinal neurodegenerative diseases. The company was founded in 2012 and is located in Paris, France.
Products
The gene therapy platform uses Adeno-Associated Virus (AAV) vectors for the treatment of retinal diseases. The company has two products in clinical trials:
— GS010 – an AAV2 gene therapy vector that encodes human wild-type ND4 protein. This is being developed for Leber’s hereditary optic atrophy (LHON) caused by a mutation of the ND4 gene. The product is in Phase 3 trials.
— GS030 – a combination of gene therapy encoding a photoactivatable channelrhodopsin protein delivery by a modified AAV2 -AVV2 7m8 and biomimetic goggles that stimulate the engineered retinal cells. This product uses optogenetics and is Phase 1/2 for retinitis pigmentosa.

Gilead Sciences, Inc.

Address 333 Lakeside Drive
Foster City, CA 94404
Country United States
Telephone 650-574-3000
URL www.gilead.com
Revenues (2019) $22.4 billion USD
Source: Company website
Company Summary
Gilead Sciences is a biopharmaceutical company engaged in marketing its approved therapies and investigating new drugs for conditions like HIV/AIDS, cancer, cardiovascular condition and others. The company, started in 1987, currently has about 11,000 employees, and operations in Europe, North America, Asia, Australia, South America, and Africa. Gilead has been called one of the most innovative companies, along with one of the best companies to work for in recent years. Sales in 2019 were reported at $22.4 billion.
On August 28, 2018, Gilead Sciences announced that it had acquired Kite Pharma and its entire portfolio. The company spent $11.9 billion in the acquisition of chimeric antigen receptor T cell (CAR-T) therapy developer Kite Pharma Inc. The company will build its upcoming oncology business around cell therapies with an eye on improving Kite’s products and developing combination regimens that incorporate CAR-T and T-cell receptor (TCR) therapies. The company expects growing competition from Novartis’ Kymriah, another CAR-T cell therapy.
In its related research and development through programs acquired during the Kite acquisition, Gilead reported in the fourth quarter of 2018 that the KITE-585 program, targeting multiple myeloma, did not satisfy efficacy requirements for further research efforts.
Products
Gilead offers Yescarta (adicabtagene ciloleucel) and was CE marked in August 2018 for treatment of relapsed or refractory DLBCL and PMBCL. This is the first CAR-T to receive European approval for two types of aggressive non-Hodgkin lymphoma. Yescarta sales increased by 73% to $456 million in 2019 compared to $264 million in 2018.
In May 2019, Kite and Humanigen entered into a collaboration to conduct a Phase 1/2 study of lenzilumab (anti-GM-CSF monoclonal antibody) with Yescarta in patients with relapsed or refractory diffuse large B-cell lymphoma.
In December 2019, Kite submitted application to the FDA for KTE-X19 for the treatment of adults with relapsed or refractory mantle cell lymphoma. The BLA submission is based on data from the Phase 2 ZUMA-2 trial and was granted priority review in February 2020. The product is under review by the EMA as well, submitted in January 2020.
Other selected indications in the pipeline include:
— Axi-cel for 2L DLBCL in Phase 3
— Axi-cel for indolent NHL in Phase 2
— Axi-cel for 1L DLBCL in Phase 2
— Axi-cel for DLBCL (+rituximab or lenalidomide) in Phase 2
— KTE-X19 for adult ALL in Phase 2
— KTE-X19 for pediatric ALL in Phase 2
— KTE-X19 for CLL in Phase 1
— Kite-718 for solid tumors in Phase 1

Gradalis, Inc

Address 2545 Golden Bear Drive, Suite 110
Carrolton, TX 75006
Country United States
Telephone 214-442-8100
URL www.gradalisinc.com
Source: Company website
Company Summary
Gradalis is a biotechnology company that is focused on the development, manufacturing and commercializing of drugs, vaccines and diagnostics for the treatment of cancer.
Products
The company is developing Vigil, a personalized cancer immunotherapy for individuals with Ewing’s sarcoma, ovarian cancer and breast cancer. The pipeline includes:
— Autologous cancer vaccines
— Vigil EWS – Ewings sarcoma in Phase 3
— Vigil OvC – ovarian cancer in Phase 2
— Vigil M – melanoma in Phase 2
— Vigil ST – solid tumors in Phase 1
— Vigil CRC – colorectal cancer in Phase 1
— Vigil HCC – hepatocellular in Phase 1
— Vigil breast- breast cancer in Phase 1
— Vigil lung – lung cancer in Phase 1
— IO combinations
— Vigil/Atezo – ovarian cancer in Phase 1
— Vigil/Durva – breast cancer in Phase 1
— Vigil Pembro – melanoma in Phase 1
— Vigil/Nivo – lung cancer Phase 1
— Fusion genes
— LPX EWS/FLI1 T1 – Ewing’s Type 1 in Phase 1
— LPX EWS/FLI1 T2 – Ewing’s Type 2 in PC
— Genes/proteins
— STMN1 – solid tumors in Phase 1
— Kras – solid tumors in PC

Inovio Pharmaceuticals, Inc.

Address 660 West Germantown Pike, Suite 110
Plymouth Meeting, PA 19462
Country United States
Telephone 267-440-4200
URL www.inovio.com
Revenues (2019) $30.3 million USD
Source: Company website
Company Summary
Inovio is researching and developing targeted therapies for cancer and infectious diseases. It is basing its development on its advanced DNA immunotherapy platform and also entered into partnership agreements with other leading pharmaceutical companies like AstraZeneca, Regeneron Pharmaceuticals, Inc., F. Hoffmann-La Roche AG/Genentech, Inc., ApolloBio Corporation, The Bill and Melinda Gates Foundation, The Wistar Institute, the University of Pennsylvania, The Parker Institute for Cancer Immunotherapy, Coalition for Epidemic Preparedness Innovations (CEPI), Defense Advanced Research Projects Agency (DARPA), GeneOne Life Science, Inc., Plumbline Life Sciences, Inc., National Institutes of Health (NIH), HIV Vaccines Trial Network (HVTN), National Cancer Institute (NCI), United States Military HIV Research Program, Drexel University and Laval University for further development.
Products
In the pipeline, Inovio offers:
— VGX-3100- an investigational immunotherapy to treat cervical cancers caused by HPV. VGX-3100 includes DNA plasmids targeting the E6 and E7 proteins of HPV. The product is in Phase 3 trials
— VGX – 3100 for vulvar HSIL in Phase 2
— VGX – 3100 for anal HSIL in Phase 2
— Inovio Pharmaceuticals announced that CEPI provided a grant to develop a vaccine against COVID-19 called INO-4800. The company is collaborating with Beijing Advacine Biotechnology to advance the product. Preclinical testing has begun with human clinical trials to commence in April 2020 in US, China and South Korea. It is anticipated that with positive results that 1 million doses of INO-4800 DNA vaccine can be produced by the end of 2020. Phase 1 clinical trials are expected to start in April 2020. On April 6, 2020, the company received approval to begin Phase 1 trials. The Bill and Melinda Gates Foundation, along with other nonprofits, are putting money into Inovio’s COVID-19 vaccine project.

Kiadis Pharma B.V.

Address Paasheuvelweg 25A
1105 BP
Amsterdam
Country The Netherlands
Phone +31 (0) 20 240 52 50
URL www.kiadis.com
Source: company website
Company Summary
Kiadis Pharma is a biopharmaceutical company that is focused on developing personalized cell-based immunotherapies. The company was founded in 1997 and is located in Amsterdam, the Netherlands.
Products
The company has had disappointing information regarding its ATIR101 candidate. It previously had been submitted to EMA for approval but that did not materialize. The company is currently enrolling for an additional Phase 3 study for potential regulatory approval in the US and the EU. The product is indicated for patients with late-stage blood cancer undergoing a hematopoietic stem cell transplant.
The company has an active pipeline including:
— K-NK002 for HSCT blood cancer in Phase 1/2
— K-NK003 for AML beginning Phase 1/2a
— K-NK00X for solid blood tumors in PC

Medeor Therapeutics

Address 611 Gateway Blvd, Suite 120
South San Francisco, CA 94080
Country United States
Phone 650-627-4531
URL www.medeortx.com
Source: company website
Company Summary
Medeor Therapeutics is a clinical-stage biotechnology company that develops and commercializes personalized cellular immunotherapies. The company was founded in 2012 by Stanford University School of Medicine professors and was originally called SERC Therapeutics – renamed Medeor Therapeutics in 2014.
Products
The company offers chimeric immunotherapy which enables transplant patients to avoid organ rejection. According to the company the therapy has potential for a wide range of serious conditions from solid organ transplants to immune and hematological disorders.
MDR-101 is the company’s lead product which is a cellular therapy manufactured from an HLA-matched living kidney donor’s blood and bone marrow cells. The product is in Phase 3 clinical trials.
MDR-102 is for kidney transplant recipients with a genetically mismatched living donor. The company opened a Phase 2/3 trial in the first quarter of 2020.
MDR-103 is designed to treat transplant patients who received an HLA-matched living donor kidney to eliminate all immunosuppressive drug use, while preserving transplant kidney function. The company is initiating Phase 2 clinical trials for delayed tolerance LDKT.
MDR-301 is designed for sickle cell disease. The CD34+ hematopoietic stem and progenitor cells leads to the establishment of sable mixed chimerism and the production of red blood cells with normal hemoglobin. Clinical trials are initiated for Phase 1.

Medipost Co, Ltd.

Address 21, Daewangpangyo-ro 644
Bundang-gu, Seongnam-si
Gyeonggi-do
Country Korea
Phone 82-2-3465-6677
URL www.medi-post.com
Source: company website
Company Summary
Medipost is a biotechnology company that is focused on the development and commercialization of cord-blood derived stem cell therapeutics. Founded in 2000, the company is a leading stem cell company with the world’s first regulatory approved allogeneic human umbilical cord blood-derived mesenchymal stem cell produds.
The company’s first stem cell product, Cartistem was launched in Korea in 2012 for patients with knee osteoarthritis. Cartistem is the only approved cell therapy for treatment of repetitive traumatic cartilage degeneration with age limit. The product is in Phase 3, clinical trial in the US. The product is cleared for Phase 2 trials in Japan.
A second product, Pneumostem is in Phase 2 clinical trial in Korea. The product is indicated for the preventive treatment of bronchopulmonary dysplasia in the US in Phase 1/2. Pneumostem received Fast-Track designation by FDA and orphan drug status by FDA and EMA.
Neurostem completed Phase 1/2a in Korea and the US for the treatment of Alzheimer’s disease

Merck & Co., Inc.

Address 2000 Galloping Hill Road
Kenilworth, NJ 07033
Country United States
Telephone 908-740-4000
URL www.merck.com
Revenues (2019) $42.3 billion USD
Source: Company website
Company Summary
Incorporated in 1970, Merck & Co. is a global provider of healthcare products including vaccines, prescription medicines, biologics, and animal health products. In 2014, the company divested its Consumer Care segment. Products in development are in process internally and through collaborative agreements with other leading market providers.
Products
In December 2019, Merck received FDA and EU approval for an Ebola vaccine which contains the virus known as recombinant vesivular stomatitis virus (rVSV-ZEBOV). It will be marketed as ERVEBO. The virus is a live replication -competent virus derived with the African green monkey Vero cell line. VSV-ZEBOV is produced through genetic modification, combining the rVSV-ZEBOV in which the gene for native envelop glycoprotein (P03522) is replaced with that from the Ebola virus. Merck will manufacture the vaccine in Germany, and it will likely be available in late 2020.

Mesoblast Ltd.

Address Leve 38, 55 Collins Street
Melbourne, VIC 3000
Country Australia
Phone +61 (3) 9639 6036
URL www.mesoblast.com
Revenues (2019) $16,722 (in thousands $)
Source: company website
Company Summary
In 2004, Mesoblast is a biotechnology company that is using its proprietary mesenchymal lineage cell technology platform to develop and commercialize allogeneic cellular candidates to treat inflammatory diseases.
Products
Mesoblast has several product candidates in clinical trials including:
— MSC-100-IV (remestemcel-L) for steroid refractory acute graft versus host disease in Phase 3
— MSC-100-IV (remestemcel-L) for Crohn’s disease in Phase 2
— MSC-100-IV (remestemcel-L) for osteoarthritis/cartilage repair in Phase 2
— MPC-150-IM (Revascor) for advanced heart failure in Phase 3
— MPC-150-IM (Revascor) for end stage heart failure in Phase 2
— MPC-06-ID for chronic low back pain due to degenerative disc disease in Phase 3
— MPC-300-IV for rheumatoid arthritis and diabetic nephropathy in Phase 2
Two allogeneic MSC products developed and commercialized by Mesoblast have been approved in Japan and Europe. Temcell HS was the first allogeneic cellular medicine to receive full regulatory approval in Japan. JCR Pharmaceuticals commercialized the product for acute graft-versus-host disease. The product was launched in 2016.
In 2017, Mesoblast granted TiGenix (Takeda) exclusive access to the commercialization of Alofisel, formerly known as Cx601, the first allogeneic MSC therapy approval in the EU. It is indicated for the treatment of perianal fistulae.

Moderna, Inc

Address 200 Technology Square
Cambridge, MA 02139
Country United States
Telephone 617-714-6500
URL www.modernatx.com
Source: Company website
Company Summary
Moderna was founded in 2010 and is headquartered in Cambridge, MA. The company is focused on the development and commercialization of mRNA medicines. The company has concentrated its discovery in six modalities including prophylactic vaccines, cancer vaccines, intratumoral immuno-oncology, systemic secreted therapeutics and systemic intracellular therapeutics.
Products
Within the prophylactic vaccine modality, the company offers eight programs, six of which have entered clinical trials. Those include:
— mRNA 1440 – H10N8 flu vaccine in Phase 1
— mRNA-1388 – Chikungunya vaccine in Phase 1
— M1653 – hMP+PIV3 vaccine in Phase 1
— mRNA-1647- CMV vaccine in Phase 1
— mRNA-1278 – YZV vaccine in preclinical
— mRNA-1777 – RSV vaccine in Phase 1 with Merck
— mRNA-1893 – Zika vaccine in preclinical
— mRNA-4197 – PCV solid tumors in Phase 1 with Merck
— mRNA-4650 – PCV solid tumors in Phase 1 with Merck
— mRNA-5671 – KRAS vaccine CRC,NSCLC, pancreatic cancer in preclinical
For COVID -19 development, Moderna offers its MRNA-1273, a novel lipid nanoparticle-encapsulated mRNA vaccine encoding for a perfusion stabilized form of the Spike (S) protein. On March 16, 2020, the company began its Phase 1 open-label, dose-ranging trial, occurring at Kaiser Permanente Washington Health Research Institute in Seattle.
Neurotech Pharmaceuticals, Inc.

-34: Neurotech Corporate Summary

Building #1, Suite 101
Cumberland, RI 02864
Country United States
Phone 401-333-3880
URL www.neurotechusa.com
Source: company website
Company Summary
Neurotech Pharmaceuticals is a biotechnology company that is privately owned and focused on the development and commercialization of therapies for chronic eye diseases. The core technology platform, Encapsulated Cell Therapy (ECT is a genetically engineered ocular implant that is effective for at least two years. The company is located in Rhode Island.
Products
NT-501 is the company’s lead product in development for retinal degenerative diseases. NT-501 ECT delivers ciliary neurotrophic factor. The product is also being evaluated for treatment of macular telangiectasia and glaucoma in Phase 2. NT-502 consists of encapsulated genetically modified cells to secrete therapeutic doses of CNTF into the back of the eye.
Northwest Biotherapeutics, Inc.

-35: Northwest Biotherapeutics Corporate Summary

Address 4800 Montgomery Lane, Suite 800
Bethesda, MD 20814
Country United States
Telephone 240-497-9024
URL www.nwbio.com
Source: Company website
Company Summary
Northwest Biotherapeutics is a clinical stage biotechnology company focused on the development of personalized cancer vaccines designed to treat a broad range of solid tumor cancers. The company was incorporated in Delaware in July 1998 as the successor to Northwest Biotherapeutics, LLC, a limited liability company formed in Washington in March 1996.
Products
The company has several different products built on its DCVax technology based on two main components – the patient’s own dendritic cells and cancer biomarkers (antigens). These products include:
— DCVax-L cancer antigens from tumor lysate from the patient’s own tumor tissue. It has been used in GMB brain cancer in Phase 3 trials and ovarian cancer trials in Phase 1 trials. DCVax-L is expected to be used for any solid tumor cancers
— DCVax -Direct – cancer antigens from tumor lysate from the patient’s own tumor tissue in Phase 1/2 for all solid tumors
— DCVax – Prostate is designed for late stage hormone independent prostate cancer in Phase 3 trials.

Novartis AG

Address Lichtsrasse 35
CH 4056 Basel
Country Switzerland
Telephone +011-41-61-324-1111
URL www.novartis.com
Revenues (2019) $48.7 billion USD
Source: Company website
Company Summary
Novartis AG employs approximately 125,000 people and sells its products to more than 155 countries worldwide. Biologics and targeted therapy are growing parts of Novartis’ business. Novartis has several biologics that produce sales exceeding $1 billion annually. Specific to oncology, these include Afinitor, Gleevec, Mekinist+Tafinlar, and Tasigna. Other notable products include Jakavi, Kisqali, Kymriah and Votrient.
It is also making substantial investments in its Sandoz Division (generics) for bioequivalent or generic versions of marketed biotechnology products.
Products
Novartis offers Kymriah (tisagenlecleucel) which is indicated for the treatment of large B-cell lymphoma and it has a second indication for the treatment of pediatric B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse. The product is a CD-19 directed genetically modified autologous T cell immunotherapy.
Other products include:
— JEZ567 , CD123 CAR-T for acute myeloid leukemia I Phase 1
— JJO686, CD22 CAR-T for acute lymphoblastic leukemia in Phase 1
— LXF821, EGFR, CAR-T PD1 inhibitor for glioblastoma multiforme Phase 1
— MCM998, BCMA CAR-T for multiple myeloma in Phase 1
— YTB323 + ibrutinib for hematological malignancy in Phase 1

NuVasive Biologics

Address 7475 Lusk Blvd
San Diego, CA 92121
Country United States
Telephone 858-455-1476
URL www.nuvasive.com
Revenues (2019) $1,168.1 in million USD
Source: Company website
Company Summary
NuVasive Biologics is part of NuVasive, Inc., a medical device company that focused on developing minimally invasive surgical products and integrated solutions for spinal surgery. The company is headquartered in San Diego, CA.
Products
NuVasive offers autograft (autologous human tissue) and Allograft (donated human tissue). Osteocel Plus and Pro are cellular bone matrix designed to mimic the biologic profile of autograft including mesenchymal stem cells and osteoprogenitor cells to aid in spinal fusion. Other allograft products include Triad and ExtenSure.

Orchard Therapeutics

Address 108 Cannon Street
London EC4N 6EU
Country United Kingdom
Phone +44 (0) 203 808-8286
URL www.orchard-tx.com
Revenues (2019) $2,513 in thousands USD
Source: company website
Company Summary
Orchard Therapeutics is a biotechnology company focused on gene therapy. The company was founded in 2015 and has one of the deepest pipelines for gene therapy candidates. It has a total of seven clinical stage programs for a variety of therapeutic areas. The company is headquartered in London.
Products
Strimvelis is a gammaretroviral vector-based gene therapy approved by EMA in 2016. It was the first ex vivo autologous gene therapy approved by EMA. It has not been approved by FDA. It is used to treat adenosine deaminase deficiency (ADA-SCID). ADA-SCID is a rare inherited condition in which there is a change in the gene needed to make an enzyme called adenosine deaminase (ADA).
The company focuses on developing treatment for rare, inherited diseases. The company has several products in development including:
— OTL-200, an ex vivo autologous gene therapy being developed for MLD (metachromatic leukodystrophy)
— OTL-203, an ex vivo autologous gene therapy for MPS-1 (mucopolysaccharidosis type 1)
— OTL-201, an ex vivo autologous gene therapy for MPS-IIIA (Sanfilippo syndrome type A)
— OTL-202 , an ex vivo autologous gene therapy for MPS-IIIB (Sanfilippo syndrome type B)
— OTL-101, an ex vivo autologous gene therapy for ADA-SCID
— OTL-103, an ex vivo autologous gene therapy for Wiskott Aldrich syndrome
— OTL-102, an ex vivo autologous gene therapy for X-linked chronic granulomatous disease
— OTL-300, an ex vivo autologous gene therapy for Beta thalassemia

Organogenesis

Address 85 Dan Road
Canton, MA 02021
Country United States
Telephone 781-575-0775
URL www.organogenesis.com
Revenues (2019) $260,981 in thousands USD
Source: Company website
Company Summary
Organogenesis was founded in 1985 as a spin-off of technology development at MIT. The company specializes in bioactive wound healing and soft tissue regeneration. The company is located in Canton, MA. The company acquired Dermagraft from Shine in February 2014. This acquisition will help Organogenesis to offer a lower cost alternative to Apligraf.
Products
Organogenesis offers Dermagraft, a bioengineered human dermal substitute for the healing of diabetic foot ulcers and Apligraf, bioengineered living cell therapy indicated for the healing of venous leg ulcers and diabetic foot ulcers. Other products include:
— NuCel is a cryopreserved, bioactive amniotic suspension allograft that creates an enhanced environment that supports tissue repair.
— ReNu is a cryopreserved, amniotic suspension allograft for osteoarthritis.
— NuShield is a dehydrated placental allograft for soft tissue repair.
— Gintuit is a bioengineered bi-layered cellular tissue that supports the healing of oral soft tissue.
— TransCyte is a bioengineered tissue scaffold that promotes burn healing.
— Novachor is a fresh chorionic membrane for treatment in chronic and acute wounds. The product is expected to launch in the fourth quarter of 2020.

Orthofix Medical Inc.

Address 3451 Plano Parkway
Lewisville, TX 75056
Country United States
Phone 214-937-2000
URL www.orthofix.com
Source: company website
Company Summary
Orthofix is a medical device company that specializes in musculoskeletal products and therapies. The company was formed in 1987 in Curacao. In 2018, the company changed its name to Orthofix Medical, Inc. and is headquartered in Lewisville, TX.
Products
The company offers the Trinity ELITE and Trinity Evolution tissue forms, which are cortical cancellous allografts that contain viable cells and are used during surgery in the treatment of musculoskeletal defects for bone reconstruction and repair.

Osiris Therapeutics, Inc.

Address 7015 Albert Einstein Drive
Columbia, MD 21046-1707
Country United States
Telephone 443-545-1800
URL www.osiris.com
Revenues (2019) $24,308 in thousands USD
Source: Company website
Company Summary
Osiris Therapeutics was founded in 1992 and is involved in the research and commercialization of mesenchymal stem cell (MSC) science and clinical application of cellular therapies. From 2010-2013 the company was divided into business segments:
— Biosurgery
— Therapeutics
In the latter part of 2013, the company divested its therapeutics business arm to focus on biosurgical applications in wound care, orthopedics, and sports medicine.
Products
Osiris developed the first approved stem cell drug, remestemcel-L for graft vs host disease. The company also offers Grafix, a cryopreserved placental membrane that is comprised of mesenchymal stem cells and epithelial cells. GrafixPL Prime is also available.
An additional product, Stravix, is a cryopreserved human placental tissue of umbilical amnion and Wharton’s jelly.
The company entered into two strategic alliances in 2014 including an agreement with Arthrex for commercialization and distribution of Cartiform (Cartiform is a cryopreserved osteochondral allograft.
The company entered into a partnership with Stryker for commercialization and development of bone matrix tissue form, Bio 4 (also known as Ovation OS). Bio 4 contains angiogenic growth factors including mesenchymal stem cells, osteoprogenitor cells, and osteoblasts.
Pharmicell

-42: Pharmicell Corporate Summary

Address 7ty Floor, Ssangbong Building
874 Unju-ro
Gangnam-gu
Seoul-si
Country Korea
Telephone +82-2-3496-0114
URL www.pharmicell.com
Source: Company website
Company Summary
Founded in 2002, Pharmicell is a biopharmaceutical company that develops and commercializes stem cell drugs. The company is headquartered in Korea.
Products
Cellgram-AMI (Hearticellgram-AMI) is the first stem cell drug approved by the Ministry of Food and Drug Safety of Korea. It was approved in July 2011. The product is manufactured using autologous bone marrow-derived mesenchymal stem cell. It is indicated to improve the ejection fraction in patients with acute MI reperfused by coronary angioplasty with 72 hours after expression of chest pain.
The company has several other products in the pipeline including
— Cellgram_LC for hepatic insufficiency in Phase 2 in Korea and Phase 1 in US
— Cellgram_ED for impotence in Phase 1
— Cellgram_DC for prostate cancer and ovarian cancer in PC

Pluristem Therapeutics, Inc.

Address MATAM Advanced Technology Park. Building #5
Haifa 3508409
Country Israel
Telephone +9724-710-8600
URL www.pluristem.com
Revenues (2019) $54,000 (sales of RUO PLX cells) in USD
Source: Company website
Company Summary
Pluristem is a clinical stage biotherapeutics company that developed a proprietary three -dimensional technology platform to develop cell therapies for inflammation, ischemia, muscle injuries, hematological disorders and exposure to radiation. The company was founded in 2001 and is headquartered in Israel.
Products
Placental eXpanded (PLX) cells are derived from a class of placental cells that are harvested from donated placenta at the time of full-term healthy delivery of a baby. PLX cell products require no tissue matching prior to administration. They are produced using the company’s proprietary three-dimensional expansion technology. The company has several products in clinical trials including:
— PLX-Pad indicated for critical limb ischemia – the product has been granted FDA Fast Track Approval and was accepted into the FDA Expanded Access Program, EMA Adaptive Regulatory Pathway and PMDA Accelerated Regulatory Pathway. The product is in Phase 3 trials.
— PLX-R18 is in development to treat incomplete recovery of transplanted hematopoietic cells and acute radiation syndrome. FDA granted an orphan drug designation by FDA for treatment of ARS and FDA has also cleared the product for IND application to treat victims who may have been acutely exposed to high dose radiation due to nuclear attack or accident PLX-18 is also being evaluated in preclinical studies for a variety of hematological indications.
— PLX_Immune are cells that had been induced with TNF-a and IFN-g to transiently alter their secretion profile. The product is in PC.

Quark Pharmaceuticals

Address 7999 Gateway Blvd Ste 310
Newark, CA 94560
Country United States
Phone 510-402-4020
URL www.quarkpharma.com
Source: company website
Company Summary
Quark Pharmaceuticals is a clinical-stage pharmaceutical company that is involved in the development of RNAi-based therapeutics. The company is a privately held company that is headquartered in Newark, CA.
The company has entered into several alliances with Novartis, Pfizer, RiboQuark and Biocon.
Products
Products in the pipeline include:
— QPI-1002 in Phase 3 for delayed graft function
— QPI-1002 in Phase 2 for acute kidney injury
— QPI-107 in Phase 3 for non arteritic anterior ischemic optic neuropathy
— PF-655 in Phase 2 for diabetic macular edema and age-related macular degeneration

Regenerative Medical System

Address 5FL 83 Uisandang-daero
Yeongdeungpo-gu
Seoul 150-724
Country Korea
Phone +82 2 2167 9100
URL www.rmsbio.net
Source: company website
Company Summary
Regenerative Medical System (RMS) is a stem cell company located in South Korea. The company was founded in 1996 and develops and commercializes cell therapeutic products.
Products
The company offers three kinds of cell therapeutic products:
— Chondron, autologous chondrocyte implantation – approved by MFDS in 2001, EU 2007
— Ossron, autologous bone marrow derived stem cell implantation – approved by MFDS in 2009, EU 2007
— BabyCell, cord blood stem cell banking and implantation – CE marked in 2007

Renova Therapeutics

Address 71 Palomar Airport Road, Suite 300
Carlsbad, CA 92011
Country United States
Telephone 858-461-1837
URL www.renova.com
Source: Company website
Company Summary
Founded in 2009, Renova Therapeutics is a biopharmaceutical company that develops gene therapy treatment for CHF and other chronic diseases. The company is headquartered in San Diego, California.
Products
RT-100 (AC6 gene transfer) is the company’s lead product for the treatment of congestive heart failure. It is a first in class, single dose gene therapy candidate that was granted Fast Track designation by FDA. The product is in Phase 3 clinical trials.
Other products in the pipeline include:
— RT-110 in preclinical for congestive heart failure
— RT-200 and RT-210 for type 2 diabetes, obesity, NAFLD, metabolic syndrome in preclinical development
— RT-300 in preclinical for CHF and pulmonary hypertension

Sangamo Therapeutics

Address 7000 Marina Blvd
Brisbane, CA 94005
Country United States
Phone 628-252-7500
URL www.sangamo.com
Source: company website
Company Summary
Founded in 1995, Sangamo Therapeutics is a biotechnology company that was previously named Sangamo Biosciences. The company is headquartered in Brisbane, CA and focuses on the development and commercialization of DNA-binding proteins for the therapeutic regulation and modification of disease-related genes. The company has four distinct technology platforms- gene therapy, cell therapy, in vivo genome editing and in vivo genome regulation.
The company is involved in several collaborations including Gilead developing ex vivo cell therapies to treat cancer using Sangamo’s zinc finger nuclease (ZFN) technology; Sanofi developing gene-edited cell therapies for treatment of beta thalassemia and sickle cell using Sangamo’s ZFN; Pfizer developing therapeutics using ZFN to treat ALS and frontotemporal lobar degeneration like to mutations of the C9ORF72 gene; and Takeda developing therapeutics for Huntington’s disease using ZFP-TF genome regulation platform.
Its newest collaboration is with Biogen to develop gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases. The companies will be initially focusing on development of ST-501 for tauopathies for Alzheimer’s disease, and ST-502 for synucleinopathies including Parkinson’s disease and a neuromuscular target.
Products
The company has a robust pipeline. Its gene therapy pipeline consists of:
— ST-920 for Fabry disease in Phase 1/2
— SB-525 for hemophilia in Phase 1/2 – Pfizer is advancing SB-525 into a Phase 3 study in 2020
— The company’s gene edited cell therapy consists of:
— ST-400 for beta thalassemia in Phase 1/2
— BIVV003 for sickle cell disease Phase 1/2
— Pre clinicals for IBD, MS, solid organ transplant

Sanofi

Company Details Description
Address 54, Rue La Boétie
Paris 75008
Country France
Telephone +33 (0)1 53 77 40 00
URL www.sanofi.com
Revenues (2019) $40.8 billion USD
Source: Company website
Company Summary
Sanofi was formed in 2004 through a merger between two leading French pharmaceutical developers, Sanofi-Synthelabo and Aventis. It has since opted to change its name to simply, Sanofi. The company is focused on providing both human pharmaceuticals and vaccines (through Sanofi-Pasteur) and also operates a successful consumer healthcare business. The majority of sales are generated from pharmaceutical products with sales attained through a direct international presence in 100 countries and availability of products to more than 170 countries.
Products
Sanofi has two products in cell and gene therapy development:
— ALVAC-HIV is a recombinant Canarypox vector that was genetically engineered to express HIV-1 Gag and Protease from the first HIV isolated, LAI (IIIB/Bru), and the HIV-1 gp120 protein from the clade E. The product is in Phase 3 clinical trials.
— UshStat, which is a gene-based therapy for the treatment of Usher syndrome (which leads to progressive retinitis pigmentosa combined with a congenital hearing loss) – a mutation of the gene encoding myosin VIIA.

SOTIO a.s.

Address Jankovcova 1518/2
170 00 Prague 7
Country Czech Republic
Telephone +420 224 175 111
URL www.sotio.com
Source: Company website
Company Summary
SOTIO is a biotechnology company that is focused on the development of immune system therapies for oncology. The company is owned by the PPF Group and is headquartered in Prague, Czech Republic.
Products
The company offers DCVAC for several indications including:
— DCVAC/PCa for prostate cancer in Phase 3
— DCVAC/OVa for ovarian cancer in Phase 3
— DCVAC/LuCa for lung cancer in Phase 2

Spark Therapeutics

Address 3737 Market Street
Philadelphia, PA 19104
Country United States
Phone 215-220-9300
URL Wwe.sparktx.com
Source: company website
Company Summary
Spark Therapeutics is a biotechnology company that was founded in 2013. The company is involved in the development of gene therapies for genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. The company is headquartered in Philadelphia, PA. In December 2019, the company was acquired by Roche.
Products
Luxturna was approved in December 2017 by FDA and 2018 in Europe. The product is a gene therapy product used for the treatment of patients with inherited retinal disease due to mutations in both copies of the RPE65 gene.
In the pipeline, Spark offers:
— SPK-7001 -a gene therapy for choroideremia, an X-linked inherited retinal disease that usually manifests in affected males. The product is in Phase 1/2.
— Stargardt disease – a gene therapy for the most common type of inherited juvenile macular degeneration cause by ABCA4 gene. The product is in discovery.
— SPK-9001 now called fidanacogene elaparvovec is a bioengineered AAV vector utilizing F9 transgene for hemophilia. The company transitioned its ongoing development to Pfizer.
— SPK-8011 is gene therapy for hemophilia A, characterized by mutations in the F8 gene. It is in Phase 1/2 trials.
— SPK-8016 is a gene therapy for hemophilia A in Phase 1/2 clinical trials.
— SPK-3006 is a gene therapy for treatment of Pompe disease in Phase 1/2.
— SPK-1001 is a gene therapy for treatment of TPP1 deficiency, a form of Batten disease. The company has received orphan product designation from FDA for SPK-1001 for treatment of CLN2 disease (neuronal ceroid lipofuscinosis caused by TPP1 deficiency.
— Huntington’s disease – Spark is developing a gene therapy for treatment of Huntington’s disease.

Tessa Therapeutics, Ltd.

Address 8 Temasek Blvd #24-02
Suntec Tower 3, 038988
Country Singapore
Phone +65 6384 0755
URL www.tessatherapeutics.com
Source: company website
Company Summary
Tessa Therapeutics is a biotechnology company that is focused on the development of cancer immunotherapies. The company was formerly known as FF CanVac Ptd Ltd.
Products
Products in development include:
— TT10 EBVSTs is in development for nasopharyngeal cancer and it is in Phase 3 clinical trials. The product has US FDA orphan drug and Fast Track designations.
— TT11 CD30 CAR-T is in Phase 2 for relapsed or refractory classical Hodgkin lymphoma. CD30-targeting CAR enables the recognition and elimination of CD30-positive lymphoma cells.
— TT11X Allogeneic CD30-CAR EBVSTs is a next generation allogeneic VST therapy targeting CD30-postitive lymphomas in Phase 1. The cell lines are derived from healthy donors and are modified with a CD30 chimeric antigen receptor and matched to suitable patients to target CD30+ cancer cells
— TT12 HPVSTs is an autologous T cell therapy composed of HPVSTs for HPV-associated cancers. In collaboration with Merck, Tessa will conduct Phase 1b/2 trials.
— TT16 HER2 CAR-Ts + CadVEC is in preclinical evaluation for treatment of HER2-positive solid tumors

Tocagen, Inc.

Address 4242 Campus Point Court, Suite 500
San Diego, CA 92121
Country United States
Telephone 858-412-8400
URL www.tocagen.com
Source: Company website
Company Summary
Tocagen is a clinical-stage, cancer-selective gene therapy company developing first-in-class, broadly applicable product candidates designed to activate a patient’s immune system against their own cancer.
Products
Tocagen’s lead investigational product candidate, Toca 511 & Toca FC, is under evaluation in a pivotal Phase 3 trial (Toca 5) for recurrent high-grade glioma (HGG), a disease with significant unmet medical need.
The U.S. Food and Drug Administration awarded Tocagen an orphan drug grant for the Toca 5 trial and has granted Toca 511 & Toca FC Breakthrough Therapy Designation for the treatment of recurrent HGG. The European Medicines Agency has granted Toca 511 PRIME (PRIority MEdicines) designation for the treatment of glioma.
TOCA 6 is for advanced solid tumors such as pancreatic, sarcoma and lung cancer in Phase 1 and TOCA8 is in Phase 1 for recurrent high-grade non-muscle invasive bladder cancer.

uniQure N.V.

Address Paasheuvelweg 25a
1105 BP
Amsterdam
Country The Netherlands
Telephone +31 20 240 6000
URL www.uniqure.com
Revenues (2019) $7,281 in thousands USD
Source: Company website
Company Summary
uniQure is a gene therapy company that is involved in the discovery, development and commercialization of gene therapies. The company is headquartered in Amsterdam, Netherlands.
In May 2015, the company entered into a collaboration with BMS that provided BMS with exclusive access to its gene therapy technology platform for the development of multiple targets in cardiovascular and other diseases. In 2019, the company is revisiting the terms of the collaborations.
In 2018, the company entered into a research and option agreement with DRK Blutspendedienst for development of hemophilia A and B gene therapy.
Products
The company’s lead product etranacogene dezaparvovec for the treatment of hemophilia B, is currently being evaluated in a Phase 3 HOPE-B pivotal trial. In September 2019, the company completed the enrollment of the lead-in phase of the HOPE-B pivotal trial.
uniQure is also developing a novel gene therapy product candidate, AMT-130, for patients with Huntington’s disease. In January 2019, the company initiated a Phase 1/2 clinical study of AMT-130.
Beyond its lead clinical program for hemophilia B and Huntington’s disease program, the company has a pipeline of additional AAV-based gene therapy programs in various stages of preclinical development including:
— AMT-180 for hemophilia A
— AMT-190 for Fabry disease
— AMT150 for SCA Type 3

VBL Therapeutics (Vascular Biogenics Ltd.)

Address 8 HaSatat St
Modi’in 7178106
Country Israel
Phone +972-8-9935000
URL www.vlbrx.com
Source: company website
Company Summary
VBL Therapeutics is officially named Vascular Biogenics Ltd. and was incorporated in Israel in 2000 under the name Medicard. In January 2003, the company changed its name to Vascular Biogenics. The company is located in Modi’in, Israel. The company is a clinical stage biopharmaceutical company that is focused on discovery, development and commercialization of treatments for cancer. The program is based on the company’s proprietary vascular targeting system platform, which uses genetically targeted therapy to destroy newly formed or angiogenic blood vessels.
Products
The company’s lead project is VB-111, a gene therapy for solid tumors. Based on a non-integrating, non-replicating, Adeno 5 vector, it utilizes VBL’s proprietary Vascular Targeting System (VTS) to target the tumor vasculature for cancer therapy. The product is in Phase 3 trials for platinum resistant ovarian cancer and has received orphan designation for treatment of ovarian cancer in the EU.
Other products in the VTS pipeline include:
— VB-511 for an oncology indication in PC
— VB-211 for ischemia in PC
— VB-411 for ischemia in PC

Vericel Corporation

Address 64 Sidney Street
Cambridge, MA 02139
Country United States
Phone 800-556-0311
URL www.vcel.com
Revenues (2019) $117,850 in thousands USD
Source: company website
Company Summary
Vericel is a provider of advanced cell therapies and specialty biologic for the treatment of severe burns and sports medicine.
In 2014, the company acquired the cell therapy and regenerative medicine business of Sanofi.
Products
Vericel has three marketed products:
— MACI is an autologous cellularized scaffold product indicated for the repair of cartilage defects of the knee with or without bone involvement. The product was approved by FDA in 2016 and it was launched in 2017.
— Epicel is a permanent skin replacement for the treatment of full-thickness burns.
— Carticel for repair of cartilage defects caused by acute or repetitive trauma. – the product is no longer marketed in the US.
— The company’s pipeline includes Imyelocel-T which has been granted orphan drug designation by FDA for the treatment of DCM. In 2017, the FDA required the company to conduct at least one more Phase 3 clinical. Given the expense required for further development, the company elected to focus on its existing products and has no plans to initiate a Phase 3 trial on its own.

ViroMed Co Ltd (Helixmith)

Address 203 Dong, Basic Research Dong,
Seoul National University
Gwanak-ro
GWanak-gu Seoul
Seoul 08826
Country South Korea
Telephone +82 2 2102-7200
URL www.viromed.co.kr
Source: Company website
Company Summary
ViroMed is a biotechnology company that focuses on DNA and protein-based therapeutics designed for treatment of cardiovascular diseases such as peripheral artery disease and coronary artery disease; neuropathy diseases, such as diabetic peripheral neuropathy disease; cancer, such as breast cancer and thrombocytopenia; arthritis, such as osteoarthritis; and allergy such as atopic dermatitis and muscular fatigue. The company was established in 1996 as ViroMedica Pacific but later changed its name to ViroMed in 1999. The company is headquartered in Seoul, Korea. The company later changed its name to Helixmith.
Products
The company offers VM202 which is in Phase 3 clinical trials. VM202 (donaperminogene seltoplasmid) is a first-in-class, proprietary, non-viral, potentially regenerative plasmid DNA gene therapy. VM202 is a novel genomic cDNA hybrid human hepatocyte growth factor (HGF) gene with a novel and proprietary coding sequence (HGF-X7) expressing two isoforms – a configuration that closely mimics HGF productions in humans that is needed for optimal therapeutic benefits. However, the company is struggling with inconsistencies in the Phase 3 trial process that found that the product was not meeting primary endpoints, but investigations found that people in the control group may have been administered VM202. Helixmith will work to start two or three smaller trials to generate more data on VM202. Helixmith expects to initiate the trials within the next six months and deliver data in early 2022.