Around the Helix: Cell and Gene Therapy Company Updates – July 30, 2025

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 Gives Sarepta Green Light to Restart Shipment of DMD Gene Therapy Elevidys to Patients who are Ambulatory

The FDA has recommended that Sarepta Therapeutics remove its voluntary hold on United States shipments of Duchenne muscular dystrophy (DMD) gene therapy delandistrogene moxeparvovec-rokl (marketed as Elevidys) for patients who are ambulatory.

2. Rocket’s PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy RP-A601 Snags RMAT Designation

Rocket Pharmaceuticals’ RP-A601, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat plakophilin-2 related Arrhythmogenic Cardiomyopathy (PKP2-ACM), has received regenerative medicine advanced therapy (RMAT) designation from the FDA.

3. ImmunoLogic, Episode 5: "Tumor Infiltrating Lymphocytes" With Michael T. Lotze, MD

In this episode, Fraietta speaks with Michael T. Lotze, MD, a professor of surgery, immunology, and bioengineering at the University of Pittsburgh, about his work on tumor infiltrating lymphoctye (TIL) therapy.

4. Regeneron’s Precision Approach to AAV Targeting

CGTLive® interviewed Sven Moller-Tank, PhD, a senior director at Regeneron, about the company's approach to AAV targeting, which is based on antibody expertise rather than random library screening.

5. Adaptimmune Sells Off Afami-Cel

Adaptimmune Therapeutics has sold afamitresgene autoleucel (afami-cel, formerly ADP-A2M4), a T-cell receptor (TCR) T-cell therapy marketed as Tecelra approved by the FDA for the treatment of synovial sarcoma, to US WorldMeds. Along with afami-cel, Adaptimmune also sold several of its other cell therapy products to the company, including lete-cel and uza-cel.

6. Krystal Biotech's Vyjuvek Approved in Japan

Krystal Biotech's beremagene geperpavec-svdt (Vyjuvek), a topical and redosable gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB) wounds, has been approved by Japan's Ministry of Health, Labour and Welfare. Notably, the therapy is the first genetic medicine for DEB to be approved in Japan. “For far too long, DEB patients in Japan have suffered from a lack of corrective therapies to manage their complex and often debilitating disease,” Hiroshi Kasamoto, the general manager of Japan at Krystal Biotech, said in a statement. “The approval of VYJUVEK in Japan is a long-awaited and urgently needed breakthrough, offering the potential to dramatically improve the lives of DEB patients across the country."

7. FDA Accepts Atara Biotherapeutics’ Resubmission of BLA for Tabelecleucel

The FDA has accepted Atara Biotherapeutics' resubmission of a biologics license application (BLA) for allogeneic Epstein-Barr virus (EBV)-specific T-cell immunotherapy tabelecleucel, which is intended to treat patients aged 2 years or older with EBV positive posttransplant lymphoproliferative disease (EBV+ PTLD) who have received at least 1 prior therapy. Notably, the BLA resubmission was accepted with priority review and the Prescription Drug User Fee Act (PDUFA) target action date has been set for January 10, 2026.