Around the Helix: Cell and Gene Therapy Company Updates – July 13, 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. Legend Biotech Drops Lymphoma CAR T-Cell Therapy

Legend Biotech has terminated its phase 1 clinical trial for LB1901, an investigational autologous anti-CD4+ chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of adults with relapsed/refractory T-cell lymphoma, according to an SEC filing.

2. Duchenne Muscular Dystrophy Gene Therapy Continues to Show Efficacy and Safety

Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne muscular dystrophy (DMD) being developed in partnership with Roche, demonstrated improvements on significant functional outcomes with a consistent safety profile, according to newly presented data from multiple studies.

3. Canavan Disease Gene Therapy Continues to Show Promising Data

Myrtelle’s gene therapy rAAV-Olig001-ASPA has shown efficacy and safety in patients with Canavan disease, with improvements in white matter and myelin, according to updated data from a phase 1/2 trial (NCT04833907).

4. TCR T Cell Therapy for HBV-Related Liver Cancer Cleared for US Trial

The FDA has cleared SCG Cell Therapy’s investigational new drug application for their T-cell receptor (TCR) T cell therapy SCG101 for hepatitis B virus (HBV)-related liver cancer. 

5. Manufacturing Facility to Support Cell Therapy Pipeline Completed by Kiromic BioPharma

The cGMP manufacturing facility in Houston will support the company's cell therapy oncology pipeline. Its completion also addresses a citation in a clinical hold communication received from the FDA.

6. Research on in vivo regenerative medicine approaches for sensorineural hearing loss to be carried out by Mogrify and Astellas

The collaboration will leverage Mogfrify's cellular reprogramming platform and utilize Astella's expertise in AAV based genetic medicine in an attempt to identify combinations of transcription factors involved in cell differentiation to generate new cochlear hair cells.

7. Vertex Pharmaceuticals announces upcoming acquisition of ViaCyte

Accelerating the development of ViaCyte's VX-880, an investigational cell therapy for Type 1 Diabetes seen as having curative potential, is the focal point of Vertex's plans with the acquisition.

8. Solvias' Cell and Gene Therapy Testing Solutions to be Bolsetered by Acquisition of Cergentis

Solvias expects the acquisition to help them meet the pharmaceutical industry's growing need for cell and gene therapy testing.

9. LG Chem's Allogeneic CAR-T Programs to be Bolstered via Strategic Platform License Signed with MaxCyte

LG Chem plans to advance the development of CAR-T cell treatments for solid tumors with clinical and commercial rights to MaxCyte's Flow Electroporation technology and ExPERT platform.

10. Strategic Partnership for the Development of New CAR-T Cell Therapies Entered by GeneScript ProBio and ACT Therapeutics

ACT Therapeutics expects that the patnership will help accelerate the development of treatments targeting refractory and intractible solid cancers.

11. Renovacor Initiates AAV Gene Therapy Research Program for Arrhythmogenic Cardiomyopathy

The company will collaborate the University of Utah’s Nora Eccles Harrison Cardiovascular Research and Training Institute, and will focus its research on the 3 largest genetic segments of arrhythmogenic cardiomyopathy.