Around the Helix: Cell and Gene Therapy Company Updates – November 29, 2023


Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

CGTLive Around the Helix

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive™’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. FDA to Investigate Risk of T-Cell Malignancy After CAR T-Cell Therapy

The FDA has announced that it will be investigating the risk of T-cell malignancies in patients that have received B-cell maturation antigen (BCMA)-directed or CD19-directed autologous chimeric antigen receptor (CAR) T-cell immunotherapies. The FDA stated that it has received reports from clinical trials and/or postmarketing adverse event data sources of cases of T-cell malignancies after treatment.

2. More Data Supports Approval as Lifileucel Review for Melanoma Pushed Back

The FDA has extended its priority review of Iovance Biotherapeutics’ biologics license application (BLA) for the tumor infiltrating lymphocyte therapy lifileucel for treating advanced melanoma that has progressed on or after anti–PD-1/PD-L1 therapy. The new Prescription Drug User Fee Act (PDUFA) date is February 24, 2024.

3. Krystal’s Dystrophic Epidermolysis Bullosa Gene Therapy B-VEC Under Review in EU

Krystal Biotech’s marketing authorization application for beremagene geperpavec (B-VEC, marketed as Vyjuvek in the United States), an investigational gene therapy intended to treat dystrophic epidermolysis bullosa (DEB), has been validated by the European Medicines Agency’s Committee for Medicinal Products for Human Use.

4. Second Gene Therapy for Epidermolysis Bullosa up for Priority Review

The FDA has accepted Abeona Therapeutics’ BLA for its autologous, gene-corrected epidermal sheet therapy Pz-cel (prademagene zamikeracel; EB-101) with priority review for the potential treatment of recessive DEB. The FDA has set a PDUFA date of May 25, 2024.

5. BioCardia Gets FDA Greenlight for Modified CardiAMP Heart Failure II Trial

The FDA has given the go-ahead to BioCardia for its planned phase 3 clinical trial of its investigational CardiAMP autologous cell therapy, intended for the treatment of patients with ischemic heart failure, according to a company announcement. The trial seeks to build upon the data established in the ongoing phase 3 CardiAMP Heart Failure trial (NCT02438306), which has completed its enrollment and is expected to read out its final data set in the final quarter of 2024.

6. Newly Launched Company Quotient Therapeutics to Study Somatic Genome With Discovery of New Treatments in Mind

Quotient Therapeutics, a company newly launched with funding from biotech company Flagship Pioneering, has announced its intention to investigate mutations found in somatic cells with the intention of gathering information that could be helpful for the development of drugs and treatments of various modalities for indications in immune disease, oncology, neurodegenerative disease, rare disease, and more.

7. Genethon and its Partners Seek to Harness AI to Speed Up Gene Therapy Development

Genethon is leveraging its partnership with Whitelabs Genomics to create new artificial intelligence (AI) tools that will in turn be used to develop novel adeno-associated virus capsids for gene therapies. Genethon is also working with Thales to streamline gene therapy manufacturing with the use of new AI tools.

8. BioMarin and German Government Agency Agree on Hemophilia A Gene Therapy Reimbursement Plan

BioMarin and the German National Association of Statutory Health Insurance Funds, which handles coverage for about 90% of people living in the country, have agreed on a reimbursement amount for patients receiving valoctocogene roxaparvovec-rvox (marketed as Roctavian), BioMarin’s gene therapy for the treatment of severe hemophilia A.

9. Genetic Cures for Kids to use Charles River’s Platform in Development of SPG56 Gene Therapy

Under a new collaboration agreement, Genetic Cures for Kids will utilize contract development and manufacturing organization Charles River’s eXpDNA™ platform for the production of plasmid DNA in its pursuit of developing a gene therapy for the treatment of the extremely rare genetic disease hereditary spastic paraplegia type 56 (SPG56).

10. BCS Financial Seeks to Reduce the Financial Risks of Gene Therapy for Insurance Payers

Among new product solutions announced by BCS are a reinsurance pool product and a gene-therapy-only stop loss product, both of which are intended to reduce monetary risks and/or help cover costs for health insurance payers. “Gene therapies offer the promise of potential cures, but with treatment costs ranging from $2 million to $4 million payers and self-insured employers will be challenged to finance these unprecedented costs,” Peter Costello, BCS Financial’s president and CEO, said in a statement. “At BCS, we are focused on finding new ways to help health plans, employers, and ultimately insureds finance and utilize these costly treatments as part of our mission to improve affordability and access to care.”

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