Around the Helix: Cell and Gene Therapy Company Updates – February 8, 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.

1. FDA Blocks 4D’s Gene Therapy Program for Fabry, Clears IND for Diabetic Macular Edema

The FDA has placed a clinical hold on 4D Molecular Therapeutics’ Fabry disease gene therapy program 4D-310 while also clearing the company’s investigational new drug application (IND) for its diabetic macular edema (DME) gene therapy program, 4D-150.

2. Dual-Targeting, FasTCAR-T Therapy Cleared for Phase 1b/2 Trial in R/R Multiple Myeloma

The FDA has cleared the IND application for Gracell Biotechnologies’ phase 1b/2 clinical trial of its FasTCAR-T chimeric antigen receptor (CAR) T-cell therapy, GC012F, in patients with relapsed/refractory multiple myeloma (R/R MM).

3. Danon Disease Gene Therapy Granted RMAT Designation by FDA

Rocket Pharmaceuticals’ RP-A501, an investigational adeno-associated serotype 9 (AAV9) vector-based gene therapy being evaluated in a phase 1 clinical trial (NCT03882437) for Danon disease, has received regenerative medicine advanced therapy (RMAT) designation from the FDA.

4. Otoferlin-Related Hearing Loss Gene Therapy Cleared for UK Clinical Trial

Decibel Therapeutics’ DB-OTO, an investigational AAV dual-vector-based gene therapy intended to treat otoferlin-related hearing loss, has received clearance of its clinical trial application from the United Kingdom’s Medicines and Healthcare products Regulatory Agency.

5. Investigators at Penn Medicine Developing Myasthenia Gravis Cell Therapy

Muscle-specific tyrosine kinase (MuSK) chimeric autoantibody receptor T-cells were evaluated in an animal model for the ability to specifically target disease-causing autoimmune B-cells.

6. GenKOre to Collaborate With 2 Undisclosed Pharmaceutical Company Partners on Gene Editing Therapies

The 2 separate collaborations will both utilize GenKOre's TaRGET technology and together have a potential value adding up to $650 million.

7. Ischemic Heart Failure Cell Therapy's IND Cleared in China

Help Therapeutics' HiCM-188, an allogeneic human induced pluripotent stem cell-derived cardiomyocyte therapy, has received clearance from China's Center for Drug Evaluation to be assessed for the treatment of worsening ischemic heart failure.

8. Pierre Fabre Acquires European Distribution Rights to EBVALLO from Atara Biotherapeutics

EBVALLO (tabelecleucel), which is intended to treat relapsed or refractory Epstein‑Barr Virus Positive Post‑Transplant Lymphoproliferative Disease, received marketing authorization from the European Commission in December of last year for patients aged 2 and older who have received at least 1 prior therapy.

9. Cell Therapy Collaboration Center Program Launched in Singapore

Thermo Fisher Scientific's new center is intended to support the development of cell therapies by the company's collaborators in the Asia-Pacific region.

10. AGC Biologics to Provide Manufacturing Services for Genenta Science's Clinical Trials

Genenta Sciences, which is developing hematopoietic stem progenitor cell immuno-gene therapy for cancer, has expanded its pre-existing relationship with AGC Biologics in preparation for a phase 2 clinical trial.

11. NeuShen Therapeutics to Support UMass Chan Amyotrophic Lateral Sclerosis Gene Therapy Research via Sponsored Research Agreement

In addition to providing funding, NeuShen announced intention to collaborate closely with the UMass Chan Medical School investigators on project management, biological and analytical assessment, and more.

12. Curapath Becomes Accredited GMP Investigational Drug Product Manufacturer in Spain

Curapath is engaged in the development and production of polyamino-acid- and lipid nanoparticle delivery systems for therapeutics, including cell and gene therapies.