Cell and Gene Therapy Business Outlook

 

A New Publication Covering The CELL AND GENE THERAPY INDUSTRY

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.

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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

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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.


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THE CELL AND GENE THERAPY MARKET IN ONCOLOGY is $1,582M

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.

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AAV CAPSID DISCOVERY UPDATE

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 LATEST NEWS FROM CELL AND GENE THERAPY OUTLOOK

 

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10 Recent Developments in Cell and Gene Therapy

There have been a number of recent developments in cell and gene therapy, as detailed in our bimonthly newsletter, Cell and Gene Therapy Business Outlook.

  1. Intellia Therapeutics, based in Cambridge, MA, has acquired Berkeley, CA-based Rewrite Therapeutics, a private biotech company specializing in developing novel genome editing technologies. Rewrite’s Rewriter platform utilizes CRISPR-guided DNA polymerases capable of making targeted corrections, insertions, deletions, and the full range of single-nucleotide changes in the genomes of both dividing and non-dividing cells, without making double-stranded breaks.  The technology can be administered with adeno-associated viral (AAV) vector and lipid nanoparticle (LNP) delivery systems.  As part of the acquisition deal, Intellia will pay Rewrite shareholders $45 million up front, with an additional $155 million in milestone payments as a mix of Intellia common stock and cash.
  2. Orchard Therapeutics, based in London, UK, has announced an agreement with the National Health Service (NHS) that enables access to Libmeldy (atidarsagene autotemcel) for all children with metachromatic leukodystrophy (MLD) in England and Wales who fall within the scope of the European marketing authorization. MLD is rare genetic disorder caused by defective copies of the ARSA gene encoding the enzyme arylsulfatase A.  Classified as a lysosomal storage disease, the enzyme deficiency results in the accumulation of fats called sulfatides which eventually destroy the myelin sheath surrounding nerve fibers in both the central and peripheral nervous systems.  Libmeldy consists of an autologous (patient-derived) CD34+ cell enriched population containing hematopoietic stem and progenitor cells (HSPCs) which have been transduced ex vivo with a lentiviral vector to express the ARSA gene encoding the human arylsulfatase-A enzyme.  The therapy is the first lentiviral hematopoietic stem cell treatment to be approved for reimbursement by England’s NHS.
  3. CRISPR Therapeutics, based in Zug, Switzerland, and ViaCyte, based in San Diego, CA, have announced the first patient has been dosed in their Phase I clinical trial of VCTX210 for the treatment of type 1 diabetes (T1D). VCTX210 is an allogeneic, gene-edited, stem cell-derived product designed as a functional replacement for insulin-producing beta cells and other blood glucose-regulating islet cells of the pancreas. Immune-modulatory genes within the cell line have been specifically engineered to avoid destruction by the host’s immune system using CRISPR’s CRISPR/Cas9 ex vivo editing platform.  (See Cell and Gene Therapy Business Outlook 1, issue 11, p. 18 for more on CRISPR Therapeutics and ViaCyte’s clinical trial for the treatment of T1D.)
  4. LogicBio Therapeutics, based in Lexington, MA, has announced that the FDA has placed a clinical hold on its Phase I/II clinical trial of LB-001, its single-dose, adeno-associated virus (AAV) genome editing therapy for the treatment of pediatric patients with methylmalonic acidemia (MMA). Four patients have been dosed so far, and the first two patients, both in the 3 to 12 years old group, are doing well and have not experienced serious adverse events (SAEs) related to the therapy.  The third patient, who was in the 6 months to 2 years old age group, experienced a drug-related SAE in the form a blood clotting disorder, thrombotic microangiopathy (TMA), which has been previously associated with AAV therapies.  That patient responded well to treatment and the SAE eventually resolved.  LogicBio implemented additional safety measures in the trial, however in January the fourth patient dosed, also in the younger age group, experienced an SAE in the form of TMA.  That patient has been steadily improving, and for the present the trial remains on clinical hold.
  5. NexImmune, a clinical-stage biotech company based in Gaithersburg, MD, has announced a collaboration with Rutgers University, The State University of New Jersey. NexImmune’s proprietary Artificial Immune Modulation (AIM) platform uses engineered nanoparticles that function as “synthetic dendritic cells” and are able to direct specific immune responses by delivering signaling proteins to T cells, much like natural dendritic cells do in a healthy immune system. The collaboration with Rutgers will focus on targeting immune checkpoint proteins for indications in neuroendocrine tumors and other cancers.
  6. Oxford Biomedica, based in Oxford, UK, has announced that Sio Gene Therapies, based in New York, NY, intends to cease work on AXO-Lenti-PD (formerly OXB-102) and return the therapy’s global rights to Oxford. AXO-Lenti-PD is an investigational gene therapy for the Parkinson’s disease that utilizes a lentiviral vector to deliver three genes required for dopamine synthesis:  TH, encoding tyrosine hydroxylase; GCH1, encoding GTP cyclohydrolase 1; and DDC, encoding aromatic L-amino acid decarboxylase, also known as DOPA decarboxylase.  The therapy is currently being evaluated in a Phase II clinical study with six patients already dosed, and strong improvement observed in two evaluable patients at a six-month followup.  Oxford plans to out-license AXO-Lenti-PD again rather than to invest in its further development.
  7. Bristol Myers Squibb, based in New York, NY, has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Breyanzi (lisocabtagene maraleucel) for the treatment of adults with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), and follicular lymphoma grade 3B (FL3B) after two or more lines of systemic therapy. Breyanzi is a chimeric antigen receptor T cell (CAR-T) therapy targeting CD19, a protein ubiquitously expressed on the surface of human B cells.  The European Commission (EC) will review the CHMP recommendation and render a final approval decision within 67 days.  Breyanzi is approved by the U.S. FDA for the treatment of adults with R/R large B-cell lymphoma after two or more lines of systemic therapy, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B, and has been approved in Japan for third-line plus R/R LBCL and follicular lymphoma.
  8. Excision BioTherapeutics, a clinical-stage biotech company based in San Francisco, CA, has announced the initiation of a Phase I/II clinical trial of EBT-101 in individuals living with human immunodeficiency virus type 1 (HIV). Excision is developing CRISPR-based therapies to cure chronic viral infectious diseases, and EBT-101 is a potentially curative therapy for HIV.  The therapy utilizes an adeno-associated virus (AAV) to deliver CRISPR-Cas9 and dual guide RNAs targeting three sites within the HIV genome in order to excise large portions of HIV proviral DNA in vivo.  (See Cell and Gene Therapy Business Outlook 1, issue 7, p. 15 for more on EBT-101 and the rest of Excision BioTherapeutics’ pipeline.)
  9. FUJIFILM Corporation, based in Tokyo, Japan, has announced that it will acquire a 90,000-square-foot cell therapy manufacturing facility in Thousand Oaks from Atara Biotherapeutics for $100 million. FUJIFILM Diosynth Biotechnologies, a contract development and manufacturing organization (CDMO) subsidiary of FUJIFILM, will run the new facility, and plans to offer approximately 140 current highly-skilled manufacturing positions and quality staff at the site. FUJIFILM says the site is readily expandable and will be able produce cell therapies at both clinical and commercial scale, including allogeneic T-cell and CAR T immunotherapies. FUJIFILM Diosynth and Atara will also sign a long-term manufacturing and services agreement to support the Atara’s clinical pipeline, including tabelecleucel for the treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+PTLD).  FUJIFILM is strongly invested in expanding its cell therapy manufacturing capabilities. The West Coast campus will join FUJIFILM Diosynth’s existing facilities in College Station, TX, Research Triangle Park, NC, Billingham, UK, and Hillerød, Denmark, with new facilities under construction in Holly Springs, NC, and Watertown, MA, as well as plans to substantially expand the Denmark and UK facilities.
  10. Nanoscope Therapeutics, a clinical-stage optogenetic biotech company based in Bedford, TX, has announced it has received IND clearance from the FDA to begin a Phase II study evaluating its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy for the treatment of Stargardt disease, an inherited form of macular degeneration. MCO-010 uses a proprietary adeno-associated virus, serotype 2 (AAV2) vector, delivered by a single intravitreal injection, to transduce retinal bipolar cells to express a gene encoding multi-characteristic opsin (MCO) on their surfaces, effectively transforming them into functional photoreceptor cells.  Since the therapy bypasses the diseased photoreceptor cells entirely, it is “gene-agnostic” and potentially capable of treating multiple vision indications, regardless of underlying gene mutations.  Nanoscope is currently evaluating MCO-010 in a Phase IIb study for the treatment of retinitis pigmentosa (RP), and expects to begin the Phase II Stargardt study in the first half of 2022.

 

/by Daniel Granderson
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This Week in Cell and Gene Therapy: 12 Recent Developments

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.
/by Daniel Granderson