Around the Helix: Cell and Gene Therapy Company Updates – January 18, 2023

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.

The Prescription Drug User Fee Act (PDUFA) date for Krystal Biotech's B-VEC, an investigational, redosable gene therapy intended to treat dystrophic epidermolysis bullosa, has been extended to May 19, 2023, a 3-month extension from its original February 17, 2023, PDUFA date.

Tenaya Therapeutics’ TN-201, an investigational adeno-associated virus (AAV)-vector based gene therapy intended to treat hypertrophic cardiomyopathy caused by mutations in the MYBPC3 gene, has received clearance of its investigational new drug application (IND) by the FDA.

Seelos Therapeutics’ SLS-004, an investigational gene therapy candidate for the treatment of Parkinson disease, has generated promising data in preclinical research; meanwhile the world’s largest observational study of Parkinson disease genetics, the Rostock International Parkinson's Disease (ROPAD) Study, has been extended by CENTOGENE and Denali Therapeutics.

AlloVir’s posoleucel (ALVR105, formerly Viralym-M), an investigational virus-specific T-cell therapy currently under evaluation across several clinical trials for multiple indications, is on track to complete enrollment in several phase 3 trials by the end of 2023 and has shown promising safety and efficacy in preventing common viruses that infect patients who have recently received allogeneic hematopoietic cell transplants in a phase 2 study.

The company reported that the eXpDNA plasmid platform is capable of 5-week batch turnaround for HQ plasmid and 10-week batch turnaround for GMP plasmid.

The companies will focused their efforts, which will be partially supported by a Dutch Top Sector Life Sciences and Health grant, on a severe form of genetic dilated cardiomyopathy.

The guidance, which still needs to be approved by European Medicines Agency (EMA) expert panels, will advise physicians on monitoring patients for liver damage after the use of Zolgensma, and is based on 2 patient deaths initially reported in August 2022.

CARSgen Therapeutics' New Drug Application for zevorcabtagene autoleucel (zevor-cel), an investigational CAR-T intended to treat relapsed/refractory multiple myeloma, was accepted for review by China's NMPA in October of last year.

The company reported that the Curate Cell Processing System is capable of recovering approximately 90% of white blood cells in patient-sourced leukapheresis blood product, while eliminating more than 90% of red blood cells and 99% of platelets.

RZ-001, an investigational RNA-based gene therapy intended to treat hepatocellular carcinoma, received clearance of IND applications from the FDA and South Korean Ministry of Food and Drug Safety last year.

The newly launched clinical research organization (CRO) services are intended to expedite the process of initiating and carrying out clinical trials for companies developing cell and gene therapies.

The company expects that the drug master file will help expedite regulatory actions for cell and gene therapies using the plasmid.

The first CliniPrime product is a new leukopak and represents the company's second of its 2 current offerings for GMP-compliant cellular starting material.

The newly launched company will seek to address inefficiencies related to late-stage development and the transition to commercial production.