Cell and Gene Therapy Business Outlook



The Best Way to Keep up with the Growing Cell and Gene Therapy Industry

From Science and Medicine Group, the company behind Instrument Business Outlook, Kalorama Information, SDi and other publications, comes a new publication: Cell and Gene Therapy Business Outlook.


With thousands of potential therapies on the market, cell and gene therapy promises future potential for pharmaceutical developers and those serving them.

  • A new twice-monthly publication dedicated to cell and gene therapy, Cell and Gene Therapy Business Outlook will offer the following:
  • Market Sizing and Forecasting of CGT Markets in Every Issue
  • Executive News Summaries – What is Happening in CGT Markets and Why It Matters
  • Deals Between CGT Companies Tracked in Every Issue
  • Important Science That Will Shape Tomorrow’s Business
  • Updates on Pipelines and Important Clinical Trials
  • Cell and Gene Therapy Tools, CMOs, Manufacturing Developments
  • Market Analysis of a Cell and Gene Therapy Segment in Every Issue




There are many websites, publications and sources on cell therapy. Cell and Gene Therapy Business Outlook differs from these sources in that it is created by market researchers and editors focused on business opportunity. Each issue will track the market size and potential for a key market segment.

Who Is Dealing with Whom? Tracking of Cell and Gene Company Deals In Every Issue.

There is a never-ending stream of activities in this market. How can you keep up? Each issue of Cell and Gene Therapy Business Outlook will keep track of mergers, investments, licensing, technology transfers and partnerships in the industry. Each issue of Cell and Gene Therapy Business Outlook contains an updated CGT Recent Deals Table with information on these important events.

Future issues will also analyze of the number of deals and increases or decreases in activity as a measure of business. You’ll never miss an important happening with Cell and Gene Therapy Business Outlook. Also, the Recent Deals Table is a great resource for tracking companies in the market.

The News That Matters

Edited by Blake Middleton, a professional CGT researcher and former Staff Research Associate at UCLA Department of Pharmacology, Cell and Gene Therapy Business Outlook is designed to provide the most relevant news. Included is news that could affect business decisions near-term. Cell and Gene Therapy Business Outlook also explains the relevant science.
With a focus on what the recent news of the day means for business, our curated news and news analysis means that you and your organization can be confident you won’t miss an important development in cell and gene therapy.




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Open up access: If more than one person will be reading, you can unlock access to other members of your organization. It’s easy to do: team subscription prices are as little as $4,995 annually for up to five readers. Larger team? Other licenses are available. Consult our website.  Convenient and Cost-Effective Seat-Based Pricing: Pricing depends on the number of users. Subscriptions can be as low as $2,200 annually for a limited one-person (single user) subscription.



MARKET SIZE: The global market for cell and gene therapy for oncology reached $1,582 million in 2020 and is expected to climb to $2,744 for 2021.

There are over 100 different types of cancer; some of the more prominent include lung, breast, brain, blood, prostate and colon cancer. The immune system plays a primary role in the body’s defense against malignancy. Although a tumor is derived from the body’s own cells and is expected to possess proteins that are recognized as self and nonantigenic, neoplastic cells can express antigens that are not recognized as self. These cells can often be eliminated by the immune system.

FORECAST: projected to increase to $7,391 in 2025; $17,490 million by 2030.

Treating cancer is difficult because it is not a single disease and because all the cells in a single tumor do not behave in the same way. Although most cancers are thought to be derived from a single abnormal cell, by the time a tumor reaches a clinically detectable size, the cancer may contain a diverse population of cells.

Market Forecast:  Strong increases in the CAR-T therapy market, increasing from just $16 million in 2017 to $1,081 million in 2020 and projected to increase to $7,391 in 2025; $17,490 million by 2030.  Blood cancers are the leading driver in the segment, representing 68% of total sales. This is expected to be the primary segment through the forecast, representing 80% of sales by 2025 and 80% in 2030.  The United States and Europe are the largest markets due to overall product approvals and cost associated with the therapies. The US market represented nearly 77%, while Europe represented 19% in 2020.  Gilead and Novartis combined represent 68% of the market for cell and gene therapy in oncology.  Industry refocuses on oncology cell and gene therapies in a post-pandemic arena, returning to pre-pandemic growth.



Adeno-associated viruses (AAV) are small human viruses which provoke only a mild immune response and are not known to cause any human disease. AAVs are quite simple in organization, possessing a small (4.7kb) single-stranded DNA genome with only two open reading frames (ORFs), rep and cap, flanked by short (145 base) inverted terminal repeats (ITRs). The rep ORF encodes multiple overlapping sequences for proteins required for replication, and the cap ROF does the same for capsid proteins, which are the proteins forming the outer viral protein coat. These genes alone are not sufficient for viral replication, and AAVs require co-infection with a second, helper virus (such as an adenovirus or HSV) to supply the remaining gene products for replication (hence the name adeno-associated virus).

Gene therapy AAV vectors are further modified to remove the rep and cap genes from the viral genome (along with their promoters and polyadenylation signal), replacing them with a therapeutic expression cassette. Production of recombinant AAV vectors in cell lines requires the rep and cap genes to be supplied by a plasmid transfected in trans, in addition to the genes supplied by the helper virus. None of these externally supplied viral genes are packaged into the final construct, so the resulting viral delivery vehicle consists only of the therapeutic cassette encased in an AAV capsid, without any viral genes present. The gene therapy vector is therefore incapable of replication, even with co-infection by a suitable helper virus.
In addition to their safety, AAV vectors possess many features which make them attractive gene therapy candidates. They have extremely low immunogenicity, they can infect both dividing and non-dividing cells, and they can persist outside the genome to offer stable, long-term expression without the risks associated with host genome integration.

AAV vectors also suffer from several shortcomings, however:
• Because of their wide distribution, many individuals have already been exposed to naturally occurring AAV serotypes and produce immune responses against them.
• AAV vectors cannot reach most tissues efficiently, and do not spread easily within those tissues if they do.
• Vectors will preferentially target some cell types but not others.
• Transduction efficiency is often extremely low.

Each of these shortcomings can be addressed by innovations in capsid structure. In addition to protecting the DNA payload, the capsid is responsible for binding to specific receptors on the target cell and safely delivering the DNA payload to the cell machinery that so will be transported to the nucleus. Viral packaging efficiency, host immunological response, tissue and cell type specificity, and transduction efficiency are all determined by the capsid serotype. Unfortunately, initial gene therapy experiments were restricted to a handful of natural AAV serotypes which had limited tropism in many human cell types. Common serotypes also present problems with pre-existing immunity (PEI), as up to 90% of the human population have already been exposed to at least one AAV serotype. For these reasons, novel capsid discovery is a current hotbed of gene therapy research.

More information on this topic can be found in the latest issue. SUBSCRIBE TODAY




The following are a dozen developments in the cell and gene therapy market from Volume 1, Issue 14 of Cell and Gene Therapy Business Outlook, published January 10, 2022.


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  1. Allogene Therapeutics, based South San Francisco, CA, has announced that the Food and Drug Administration has cleared the company to resume clinical trials its CAR-T cell therapies. In October of 2021, the FDA placed a hold on all of Allogene Therapeutics’ allogeneic CAR-T cell clinical trials following a report of a chromosomal abnormality in a patient treated with ALLO-501A, a TALEN (Transcription Activator-Like Effector Nuclease)-edited CAR-T cell therapy targeting CD19 for the treatment of relapsed or refractory non-Hodgkin lymphoma (NHL). (See Cell and Gene Therapy Business Outlook 1, issue 12, p. 13 for more information this therapy.) Allogene has since thoroughly investigated the abnormality, and concluded it was an isolated event unrelated to the therapy’s administration and had no clinical significance. Allogene says the abnormality involved regions of the T cell receptor and immunoglobulin genes that naturally undergo rearrangement during T cell or B cell maturation.  The FDA agrees with Allogene’s findings.
  2. SK Group’s holding company, SK Inc., based in Seoul, South Korea, has invested $350 million in the Philadelphia, PA-based Center for Breakthrough Medicines (CBM), making the Korean company the second-largest stakeholder in the U.S. gene therapy company. CBM is a contract development manufacturing organization (CDMO) established in 2019, and offers plasmid DNA and viral vector manufacturing in addition to process and analytical development, good manufacturing process (GMP) testing, cell therapy bioprocessing, and cell banking services for a full product’s life cycle.  SK says the deal with the CBM will help build toward SK’s goal of becoming the world’s leading CDMO with a chemical and biologic drug value chain across the United States, Europe, and Asia by 2025.
  3. ONK Therapeutics, based in Galway, Ireland, has announced that is has closed $21.5 million in Series A financing, led by current investors Acorn Bioventures and ALSHC, who were joined by new investor Cormorant Asset Management. The financing will enable the company to advance three programs currently in preclinical development: ONKT102, for relapsed/refractory multiple myeloma; ONKT103, for solid tumors such as ovarian cancer, non-small cell lung cancer (NSCLC), and breast cancer; and ONKT104, for acute myelogenous leukemia (AML).  ONK was recently granted a US patent for their cytokine-inducible SH2-containing (CISH) knockout (KO) in natural killer (NK) cells, and has since launched two new programs based on their CISH KO NK cell technology: ONKT105, knocking out the CISH gene in cord blood-derived NK cells; and ONKT106, knocking out the CISH gene in induced pluripotent stem cell (iPSC)-derived NK cells.  NK cells require cytokines signaling to maintain activity and function, but the high doses required can be toxic.  The CISH gene encodes a negative regulator of cytokines, and knocking this gene out in NK cells has been shown to make them hypersensitive to cytokine stimulation, lowering the dose required to maintain their expansion and anti-tumor functions.   (See Cell and Gene Therapy Business Outlook 1, issue 7, p. 23 for more information on ONK Therapeutics’ CISH KO technology and the company’s development pipeline.)  ONK’s shareholders include Acorn Bioventures, Cormorant Asset Management, ALSHC (principally Seamus Mulligan), and Enterprise Ireland.
  4. Univercells, based in Brussels, Belgium, has announced that it has acquired SynHelix, based in Évry-Courcouronnes, France.  SynHelix is developing a unique proprietary enzyme-based DNA synthesis technology as an alternative to bacterial DNA amplification, promising automated, one-step gigaprep-scale production of long DNA fragments.  SynHelix will become a separate legal entity called Quantoom Research Center, a new affiliate of the Univercells Group, after the acquisition.  SynHelix’s DNA synthesis platform will complement the RNA platform being developed by another Univercells affiliate, Quantoom Biosciences’ end-to-end RNA production  SynHelix was supported by AdBio partners (formerly Advent France Biotechnology), a French life sciences venture capital firm, before the acquisition, and the founders of both SynHelix and AdBio will become minority shareholders of Univercells after the acquisition.
  5. Ray Therapeutics, based in San Diego, CA, has announced the closing of $6 million in seed financing, led by 4BIO Capital. The financing will be used to advance its optogenetic therapy, Ray-001, into clinical trials for the treatment of retinitis pigmentosa (RP). Preclinical studies indicate that Ray-001 could be a one-time treatment that is sustainable for a lifetime. Rather than attempting to repair the specific genetic defect responsible for RP, Ray-001 is a mutation-independent optogenetic therapy that bypasses the defective photoreceptors entirely by targeting other retinal cells along the vision circuit pathway.  The therapy is administered via intravitreal injection, where it diffuses from the vitreous into the retina to transduces the retinal ganglion cells (RGCs).  Over 100 mutations in more than 70 different genes can lead to RP, and no effective treatment is available.
  6. Curocell, based in Daejeon, South Korea, has begun construction of a new 17,325 square-meter CAR-T Center in Dungok Residential & Industrial Area in Daejeon International Science & Business Belt. The center will house a GMP facility for commercial manufacturing of CAR-T cell therapies and an R&D center for further pipeline development.  Construction is expected to be completed by the first half of 2023, with the center fully operational by 2024.  Curocell plans to use the facility for in-house development and production of their CAR-T therapies, and to expand their business to reach a global market.  Curocell is a clinical-stage company, and their lead CAR-T product, CRC01 (anbalcabtagene autoleucel), is currently in a Phase Ⅰ clinical trial in South Korea for the treatment of relapsed/refractory diffuse large B-cell lymphoma (DLBCL), with Phase II scheduled to begin in the first half of 2022.
  7. Emendo Biotherapeutics, based in New York, and Seattle Children’s Research Institute have announced a research collaboration to study priming treatments for hematopoietic stem cells (HSCs) extracted from patients with severe congenital neutropenia (SCN). Emendo has developed EMD-101, a CRISPR-based treatment for ELANE-related SCN (SCN1). SCN1 is an autosomal dominant disease caused by a mutation in one allele of the ELANE gene which encodes neutrophil elastase, preventing hematopoietic stem cells (HSCs) from differentiating into neutrophils.  HSCs require priming before transplantation, which is typically achieved with granulocyte colony stimulating factor (G-CSF). G-CSF also increases neutrophil count and is commonly administered as a treatment for SCN, however.  The collaboration will therefore evaluate priming treatments for HSCs extracted from SCN patients, with the goal of developing a clinical trial protocol for Emendo’s SCN1 gene therapy.  Such a trial is expected to begin in late 2022, pending regulatory approval, and Seattle Children’s Research Institute has preferred rights to serve as the clinical trial site.
  8. Novartis, based in Basel, Switzerland, will acquire London-based Gyroscope Therapeutics, a clinical-stage gene therapy company specializing in the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). GA is an advanced form of dry AMD, which is a leading cause of irreversible vision loss in people over 55.  Gyroscope has developed GT005, a one-time adeno-associated serotype 2 (AAV2)-based gene therapy for the treatment of treatment of GA, currently being  evaluated in one Phase I/II and two Phase II clinical trials.  An overactive complement system is believed to play a role in GA, leading to inflammation which damage eye tissues.  GT005 treats GA by increasing production of a protein called Complement Factor I (CFI), which regulates the activity of the complement system.  Under the acquisition agreement, Novartis will make an upfront payment of $800 million, with additional milestone payments of up to $700 million. Novartis and Gyroscope will continue to operate as separate and independent companies until the deal closes.
  9. Ambys Medicines, based in South San Francisco, CA, has announced today announced the completion of a $47 million extension of its Series A financing, bringing its total Series A funding to $107 million. The funding was led by Third Rock Ventures with participation from Takeda, Schroders Capital, Laurion Capital, Smilegate Investment, and Alexandria Venture Investments, among others.  Ambys is a cell therapy company specializing in treating liver disease with mature hepatocytes to or even replace liver function.  The funding will support the company’s lead program, AMI-918, through pre-IND studies, with the goal of initiating a Phase I/II clinical trial in the 2nd quarter of 2023.  AMI-918 is an allogeneic liver-cell therapy consisting of mature hepatocytes that function in vivo as healthy liver cells and is designed for the treatment of acute liver disease.  Ambys’ second program is designed to treat chronic liver disease by extending the durability of replacement cells and by improving dosing and administration without the need for immunosuppressive therapy, the company says.
  10. EXUMA Biotech, a clinical-stage biotech company based in West Palm Beach, FL, has announced the completion of a $41 million Series B2 financing, bringing the company’s total financing raised since its inception to approximately $130 million. New investors included Americo Life in addition to existing investors. The funding will be used to support development of EXUMA’s autologous subcutaneous rapid point-of-care (rPOC) chimeric antigen receptor T- and NK-like (CAR-TaNK) platform, which does not require lymphodepleting chemotherapy, can be manufactured in less than six hours, and may produce a lower systemic cytokine burden during expansion than conventional CAR-based therapies. EXUMA will also continue clinical investigation of its tumor metabolism regulated (TMR) CAR-T technology targeting solid tumors, which uses the tumor microenvironment to activate the CARs, limiting damage to healthy tissue by reducing on-target, off-tumor toxicity.  Two TMR CAR-T products, CCT301-38 (targeting receptor tyrosine kinase AXL) and CCT301-59 (targeting receptor tyrosine kinase-like orphan receptor 2) are currently being evaluated in clinical trials at Shanghai Public Health Clinical Center sponsored by Shanghai PerHum Therapeutics.
  11. Pfizer, based in New York, has announced the death of a patient participating in the non-ambulatory cohort of a Phase Ib clinical trial of its mini-dystrophin gene therapy candidate PF-06939926 in Duchenne muscular dystrophy (DMD). (See Cell and Gene Therapy Business Outlook 1, issue 8, p. 31 for more information on Pfizer’s PF-06939926 and DMD.) Screening and dosing in the clinical trial have been paused while Pfizer investigates the incident with the trial site investigator and independent External Data Monitoring Committee, and the U.S. FDA has placed PF-06939926’s IND on clinical hold.
  12. Lineage Cell Therapeutics, based in Carlsbad, CA, has announced that the company and its subsidiary, Jerusalem, Israel-based Cell Cure Neurosciences, have inked an exclusive worldwide collaboration and license agreement with Swiss giant Roche and South San Francisco, CA-based Roche Group member Genentech, for the development and commercialization of a retinal pigment epithelium (RPE) cell therapy for the treatment of ocular disorders, including advanced dry age-related macular degeneration (AMD) with geographic atrophy (GA). Under the agreement, Genentech take over clinical development and commercialization of Lineage’s OpRegen program, currently in a Phase I/IIa clinical trial in patients with advanced dry AMD with GA. Lineage will complete activities related to the current clinical trial and handle certain manufacturing activities. In exchange, Genentech will pay Lineage $50 million up front, with up to $620 million in additional milestone payments, plus tiered double-digit royalties.

The following are recent developments in Cell and Gene Therapy, from Volume 1, Issue 13 of Cell and Gene Therapy Business Outlook, published December 22, 2021

  1. Arrowhead Pharmaceuticals, based in Pasadena, CA, has completed a purchase of 13 acres in the Verona Technology Park in Verona, WI, where it plans to invest between $200 million and $250 million to build a 140,000 square-foot drug manufacturing facility and a 115,000 square-foot laboratory and office facility to support its process development and analytical activities, creating about 250 new jobs in the local area. The company expects to begin construction is in the first quarter of 2022, with completion of the lab and office space anticipated in the first quarter of 2023, and the manufacturing facility in the fourth quarter of 2023. The new Arrowhead campus will support the company’s growing pipeline and establish manufacturing capabilities for its targeted RNAi molecule (TRiM) drug candidates while the company continues to operate its R&D facilities in Madison, WI, and San Diego, CA.  Arrowhead’s TRiM platform treats genetic diseases by using  RNA interference (RNAi) to silence the genes that cause them.
  2. Abeona Therapeutics, New York-based a gene and cell therapy company, has announced the pricing of an underwritten public offering of 44,700,000 shares and warrants to purchase 44,700,000 shares at a price of $0.39 per one share of stock and one warrant to purchase one share. The warrants have an exercise price of $0.39 per share and can be exercised immediately, with an expiration date five years from the date of issuance. The offering is expected to close on December 21, 2021, with proceeds of approximately $17.5 million. Abeona will use the funds to continue clinical development of pipeline products which include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa currently in Phase III development; ABO-102, an AAV-based gene therapy for Sanfilippo syndrome type A (MPS IIIA) currently in Phase I/II development; ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B (MPS IIIB) currently in Phase I/II development; and preclinical programs for indications in juvenile Batten disease (CLN3), cystic fibrosis (CF), retinal diseases, and undisclosed targets.
  3. Spark Therapeutics, a Roche company based in Philadelphia, PA, has announced plans to invest $575 million to build a new, state-of-the-art gene therapy center on Drexel University’s campus. The center will be located at the intersection of 30th and Chestnut Streets, with a 99-year ground lease of Drexel University’s F Lot. The planned 500,000 square-foot multi-story building will more than double Spark’s campus in Philadelphia to a projected one million square feet.  The announcement kicks off a long-term partnership between Spark and Drexel University, and the new facility is anticipated to begin construction in the fourth quarter of 2022.
  4. BioMarin Pharmaceutical, based in San Rafael, CA, and Skyline Therapeutics (formerly Geneception), based in Shanghai, China, have announced a multi-year strategic collaboration to discover, develop, and commercialize adeno-associated virus (AAV)-based gene therapies for genetic cardiovascular diseases. Skyline Therapeutics’ proprietary AAV vector engineering and design technology and manufacturing capability will be combined with BioMarin’s experience in gene therapy development, cardiovascular biology, and insights into genetic diseases to develop gene therapies targeting dilated cardiomyopathies (DCM), a group of progressive diseases which result in an enlarged heart with reduced circulatory efficiency.  Under the agreement, BioMarin and Skyline will collaborate on discovery and research through to IND application, after which each company will be responsible for clinical development in their pre-defined territories (United States, Europe, and Latin America for BioMarin, and the Asia-Pacific region for Skyline).  Skyline Therapeutics will receive an undisclosed upfront payment and an equity investment from BioMarin, along with potential milestone payments and royalties on future sales by BioMarin in its territories.
  5. Chugai Pharmaceutical, based in Tokyo, Japan, has announced a license agreement with Basel, Switzerland-based Roche for delandistrogene moxeparvovec (SRP-9001), an investigational gene therapy for Duchenne muscular dystrophy (DMD) currently being developed by Roche and Cambridge, MA-based Sarepta Therapeutics. Under the agreement, Chugai will have exclusive marketing rights in Japan for delandistrogene moxeparvovec in exchange for an upfront fee and milestone payments. Sarepta and Roche are currently conducting a global Phase III clinical trial for delandistrogene moxeparvovec for the treatment of DMD, and Sarepta will handle future clinical studies, including in Japan. Chugai will be responsible for the regulatory filing and marketing in Japan.
  6. Philadelphia, PA-based iECURE, has announced that it has entered into an exclusive agreement with the University of Pennsylvania (Penn) to develop next-generation lipid nanoparticles (LNP) for liver gene editing applications. The agreement grants iECURE exclusive rights to gene editing in the liver using LNP technology developed in collaboration with the laboratory of Michael Mitchell, PhD, at Penn. New startup iECURE is a mutation-agnostic in vivo “knock-in” gene editing company, and was founded in 2020 in collaboration with Penn’s Gene Therapy Program, directed by James M. Wilson, MD, PhD. Mitchell’s laboratory has already collaborated for many years with Penn’s Gene Therapy Program and will continue that collaboration with the new partnership.  The new agreement expands iECURE potential pipeline of programs with Penn from 13 to 15.
  7. Inceptor Bio, based in Research Triangle Park, NC, has announced that it has executed an in-licensing agreement with the University of California, Santa Barbara (UCSB) for a chimeric antigen receptor macrophage (CAR-M) technology invented by the laboratory of Denise Montell, PhD. Macrophages play a key role in innate immunity, attacking and engulfing (through a process called phagocytosis) anything that does not display surface markers characteristic of healthy cells. They also initiate adaptive immune responses by displaying antigens and secreting cytokines to recruit other immune cells.  CARs combine the functions of tumor-specific antigen binding and macrophage activation into a single receptor molecule, and program the macrophages to selectively target and engulf cancer cells.  The macrophages then recruit other cells to generate a coordinated immune response against the cancer cells, attacking even difficult-to-treat solid tumors.
  8. New York-based Pfizer has announced the opening of a new 85,500 square-foot clinical manufacturing facility in Durham, NC, part of its $800 million investment to build three scalable, state-of-the-art gene therapy manufacturing facilities with 300,000 square feet of capacity to support its continued investment in that field. The new facility will house Pfizer’s BioTherapeutics Pharmaceutical Sciences Group and is expected to create more than 50 new jobs supporting Pfizer’s gene therapy and biologics portfolio, with approximately 40 employees relocating from Pfizer’s Chapel Hill site.  With these investments, Pfizer has built up one of the world’s largest production capacities for gene therapy vectors, with a total bioreactor manufacturing capacity of 22,000 liters.  Pfizer’s worldwide R&D capacity includes locations in La Jolla, CA; Boulder, CO; St Louis, MO; Pearl River, NY; Groton, CT; and Cambridge and Andover, MA.
  9. Genenta Science, a clinical-stage biotech company based in Milan, Italy, has gone public, selling 2.4 million American depositary shares (ADSs), at $11.50 each to generate total proceeds of about $36 million. Genenta specializes in hematopoietic stem-cell gene therapies for the treatment of solid tumor cancers, and its lead candidate, Temferon, is currently in a Phase I/IIa dose-escalation clinical trial for the treatment of newly diagnosed glioblastoma multiforme (GBM), a common brain cancer.  Temferon modifies autologous hematopoietic stem and progenitor cells (HSPCs) by ex vivo gene transfer to create tumor-infiltrating Tie2 expressing monocytes (TEMs) which carry a payload of IFN-α, a proinflammatory therapeutic.
  10. Myrtelle, based in Wakefield, MA, has announced that it entered into an exclusive worldwide licensing agreement with New York-based Pfizer for rAAV-Olig001-ASPA, an investigational recombinant adeno-associated virus (rAAV)-based gene therapy for the treatment of Canavan disease. Canavan disease is a fatal childhood genetic neurological disorder caused by a defective ASPA gene encoding the enzyme aspartoacylase.  Aspartoacylase deficiency interferes with growth of the myelin sheath around the nerve fibers of the brain, resulting in spongy degeneration of the white matter in the brain.  The investigational gene therapy targets oligodendrocytes, which responsible for producing myelin, directly with a proprietary AAV vector designed to restore ASPA gene function.  Myrtelle is currently conducting a Phase I/II clinical of the gene therapy in children with Canavan disease at Dayton Children’s Hospital in Dayton, Ohio.
  11. Neochromosome, a wholly owned subsidiary of Opentrons Labworks based in New York, and Mekonos, based in San Francisco, CA, have formed a new partnership to advance cell engineering for personalized cell therapies. The partnership will test and optimize the delivery of Neochromosome’s de novo synthesized “Big-DNA” cargo directly into the nuclei of human induced pluripotent stem cells (hiPSCs) using Mekonos’ proprietary Iris technology for ex vivo delivery of large DNA payloads. The companies say their cell-based therapies have the potential to treat a variety of diseases, including diabetes, Parkinson’s, and cancer. The research collaboration launches in January 2022 in the San Francisco Bay Area, with initial results expected mid-year.
  12. Novartis, based in Basel, Switzerland, has terminated its agreement with Melbourne, Australia-based Mesoblast to develop, commercialize and manufacture remestemcel-L for the treatment of acute respiratory distress syndrome (ARDS), including that associated with COVID-19. Remestemcel-L is an investigational cell therapy composed of culture-expanded allogeneic mesenchymal stromal cells (MSCs) derived from bone marrow of an unrelated donor.  The treatment had previously been rejected by the FDA for the treatment of graft-vs-host disease in late 2020, but Mesoblast had hopes that a 300-patient, Phase III clinical trial evaluating Remestemcel-L for the treatment of COVID-19-related ARDS would lead to an Emergency Use Authorization (EUA) by the FDA. The clinical trial failed to meet its primary endpoint, however, and Novartis has backed out the deal.  Based on the trial’s observed mortality reduction with remestemcel-L in patients aged under 65 with COVID-related ARDS, Mesoblast is still optimistic about the drug’s potential, and is preparing to launch another Phase III trial in order to pursue an EUA for the treatment of COVID-related ARDS.
  13. Accellix, based in Jerusalem, Israel, has raised $10 million from BroadOak Capital Partners. Accellix is a biotechnology company that provides flow cytometry results in a compact, easy-to-use platform for use by cell therapy companies on the manufacturing floor and at the point-of-need. Their products streamline a complex but necessary part of cell analysis, providing more robust outcomes and reducing overall quality-control costs in cell therapy manufacturing.  The investment is part of BroadOak’s fifth fund and marks the firm’s third investment in the last six months in the rapidly growing market of tools and services for cell and gene therapy development and manufacturing.  BroadOak previously invested in Accellix as part of a prior fund.
  14. Rejuveron Life Sciences, a Zürich-based biotech company, has funded a further $20 million in Francisco-based Endogena Therapeutics’ $29 million Series A financing, strengthening its support to a near-majority holding. Endogena specializes in endogenous regenerative therapeutics targeting degenerative diseases of the eye, and Endogena will use the funding to advance its two lead programs. EA-2353 is an endogenous photoreceptor regeneration treatment for retinitis pigmentosa (RP) and is scheduled to enter human trials in the first half of 2022.  EA-2351 is a pre-clinical treatment for geographic atrophy (GA), an advanced form of age-related macular degeneration (AMD).
  15. Cambridge, MA-based bluebird bio’s investigational therapy bb1111 (lovotibeglogene autotemcel) is beginning to show promising results as a potential permanent cure for sickle cell disease (SCD). In a Dec 12 paper published in The New England Journal of Medicine, researchers led by Julie Kanter, MD, at the University of Alabama at Birmingham (UAB) report that patients treated with bb1111 are showing signs of stable expression of normal hemoglobin. The therapy consists of autologous transplantation of hematopoietic stem and progenitor cells (HSPCs) transduced with the BB305 lentiviral vector encoding HbAT87Q, a modified β-globin gene which produces a form of hemoglobin less likely to cause sickling when it’s expressed alongside the mutated hemoglobin gene responsible for SCD (HbS). A few days after publication of those results, bluebird announced that the FDA had placed a partial, temporary clinical hold on the gene therapy due to an ongoing investigation by bluebird into an adolescent patient with persistent, non-transfusion-dependent anemia following treatment with the drug.  Enrollment and dosing for SCD patients 18 and older are unaffected by the hold, and those studies will continue as planned.
  16. Smart Immune, based in Paris, France, has announced a research collaboration with New York’s Memorial Sloan Kettering Cancer Center (MSK). The clinical-stage company specializes in a proprietary ex-vivo biomimetic “thymus-in-a-dish” technology to create ProTcells, which are allogeneic T-cell progenitors designed to reset a rapid, safe, and full immune reconstitution.  The MSK collaboration will expand Smart Immune’s ProTcell pipeline with a CAR-ProTcell platform which uses its progenitor ProTcells instead of mature T-cells to generate a naïve, long lasting, exhaustion-free CAR-T cell population.  MSK will develop the first mouse-to-mouse model CAR-ProTcell proof-of-concept, and Smart Immune will handle the humanized model proof-of-concept.  The company says the work has the potential to expedite the clinical development of next generation CAR-T cells combining long-term persistence, natural protection against infections, and low risk of graft-versus-host disease (GvHD) in allogeneic setting.
  17. Aziyo Biologics, a commercial-stage regenerative medicine company based in Silver Spring, MD, has announced the closing of approximately $14.0 million of previously announced private investment in public equity (PIPE) financing. The financing was led by a fund affiliated with Birchview Capital, with participation from existing investors including funds affiliated with Deerfield Management Company and HighCape Capital.  Aziyo specializes in products to improve outcomes for patients undergoing cardiovascular, orthopedic, and reconstructive surgery, with a focus on patients receiving implantable medical devices.
  18. PDC*line Pharma, a clinical stage biotech company based in Liège, Belgium and Grenoble, France has announced the completion of its Series B2 round of financing, €11.8 million ($13.7 million) in financing was led by Korea Investment Partners, a leading multi-billion dollar South-Korean fund, and also included a syndicate of new South Korean investors in life sciences: Alpha Holdings, Brain Asset Management, and Hansongneotech Co. Ltd. Existing investors Noshaq Group (ex-Meusinvest), Sambrinvest (the Charleroi investment fund), SFPI-FPIM (the Belgian Federal Holding and Investment Company), SRIW (The Regional Investment Company of Wallonia), and Smart Korea UTC BioHealthcare Venture Fund also participated. The company has also received a €5.7M ($6.6M) grant in repayable aid from the Walloon Region of Belgium, bringing the B2 round total to €17.5 million ($20.3 million).  Together with €20 million ($23.2 million) in B1 fundraising from January 2020, the company has raised a total of €37.5M million in this Series B round.  The company’s lead candidate, PDC*lung01, consists of PDC*line’s professional antigen-presenting cells loaded with HLA-A2 restricted peptides derived from seven shared tumor antigens, and is currently in a Phase Ib/II clinical trial for the treatment of non-small cell lung cancer.
  19. TrinCE, a new spin-off of Ghent University (UGent) based in Ghent, Belgium, has raised €4 million ($4.5 million) from Novalis Biotech Acceleration, Qbic II, and private investors. The company’s LumiPore transfection platform uses photothermal nanoparticles in combination with laser illumination to transiently heat and permeabilize cell membranes.  The platform has demonstrated efficient cytosolic delivery of a wide variety of molecules up to 500 kDa, including antisense oligonucleotides (ASOs), small interfering RNA (siRNA), peptides, antibodies, proteins, and quantum dots into virtually any cell type, including hard-to-transfect cells such as immune cells for cell therapies.