Around the Helix: Cell and Gene Therapy Company Updates – November 9, 2022

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.

1. DMD Patient Dies in CRISPR Gene Therapy Trial Led By Nonprofit Biotech Cure Rare Disease

Terry Horgan, the primary patient in an N-of-1 clinical trial evaluating a CRISPR-based gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has died, according to an announcement from Cure Rare Disease, the nonprofit biotech sponsoring the trial.

2. Serum-free, Suspension-based Lentiviral Vector Platform May Allow for Accelerated Production

ElevateBio BaseCamp has unveiled its new LentiPeak Lentiviral Vector platform, a serum-free, suspension-based, scalable production platform intended to produce vector yields for cell and gene therapy products according to regulatory standards with high volumetric productivity.

3. ICER Suggests $2.9 Million Cap for Hemophilia Gene Therapies

The Institute for Clinical and Economic Review (ICER) has concluded that the price of valoctocogene roxaparvovec (val-rox; Roctavian; BioMarin) should be capped at $1.9 million and the price of etranacogene dezaparvovec (EtranaDez; uniQure and CSL Behring) should be capped at $2.9 million.

4. BioMarin Prepares for PDUFA Delay for Val-Rox Hemophilia Gene Therapy

In a status update for its biologics license application (BLA) for valoctocogene roxaparvovec (val-rox), BioMarin disclosed that the gene therapy's PDUFA target action date may be delayed if the FDA deems the submission of new data from a phase 3 analysis as a major amendment to the application.

5. Complement Therapeutics' CTx001 Gets ILAP Innovation Passport from MHRA

CTx001 is an investigational adeno-associated virus (AAV)-vector based gene therapy intended to treat visual impairment from geographic atrophy.

6. Affimed, Artiva Biotherapeutics to Collaborate on Combination Therapy for Patients With CD30+ Lymphoma

The combination therapy will consist of Affimed's investigational Innate Cell Engager AFM13 and Artiva's AB-101, an investigational cord blood-derived allogeneic NK cell product.

7. NKGen, Parkinson's Foundation Partner to Advance Natural Killer Cell Therapy Product for New Indication

SNK01 is an investigational autologous Natural Killer cell therapy currently in clinical trials for the treatment of advanced refractory solid tumors. The new collaboration will seek to bring SNK01 to clinical trials for the additional indication of advanced Parkinson disease.

8. Charles Rivers Laboratories Expands Cell Therapy Manufacturing Facility in Tennessee

The expansion added 9 new processing suites to the Memphis facility.

9. Cyagen, Neurophth Partner to Discover and Develop New AAV Gene Therapy Vectors with AI Platform

The collaboration will utilize Cyagen's artificial intelligence platform to discover new AAV vectors for use in potential gene therapy candidates intended to treat ophthalmic disorders. Both companies will collaborate on preclinical research with the vectors.

10. Tessa Therapeutics Gains Commercial Rights to Allogeneic EBVS Technology Platform Developed in Collaboration With Baylor College of Medicine

Several of Tessa Therapeutics' investigational candidates, including TT11X, an allogeneic cell therapy product intended to treat relapsed/refractory CD30+ lymphomas, were developed with the Epstein-Barr virus specific T-cell (EBVST) technology platform.

11. LEXEO Therapeutics to use Cerveau Technologies' Investigational Imaging Agent in Gene Therapy Clinical Trial

Cerveau's [F-18]MK-6240 will be used as biomarker in the clinical trial for LEXEO's LX1001-01, an investigational gene therapy intended to treat APOE4-associated Alzheimer disease.