Around the Helix: Cell and Gene Therapy Company Updates – June 25, 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. Vertex’s Islet Cell Therapy Zimislecel (VX-880) Restores Endogenous Insulin Secretion in Type 1 Diabetes

Vertex Pharmaceuticals’ zimislecel (also known as VX-880), an investigational allogeneic insulin-producing islet cell therapy, has restored endogenous insulin secretion in patients with type 1 diabetes (T1D) treated in the phase 1/2 FORWARD clinical trial (NCT04786262). The data were announced in an oral presentation at the 85th Scientific Sessions of the American Diabetes Association® (ADA) , held June 20 to 23, in Chicago, IL, and simultaneously reported in the New England Journal of Medicine.

2. FDA Announces Investigation of Deaths Following Treatment With Sarepta’s DMD Gene Therapy Elevidys

The FDA has announced that it is investigating the 2 deaths that Sarepta Therapeutics has reported in patients who received treatment with delandistrogene moxeparvovec-rokl (Elevidys), the company’s marketed adeno-associated virus (AAV) vector-based gene therapy for Duchenne muscular dystrophy (DMD).

3. FDA Indicates AdComm Not Currently Required for Capricor’s BLA for DMD Cardiomyopathy Cell Therapy Deramiocel

The FDA has indicated an advisory committee (AdComm) meeting regarding the biologics license application (BLA) for Capricor Therapeutics’ Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy under review for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy, is not currently required.

4. The Emergence of CRISPR-Based Medicines and the Resurgence of AAV

CGTLive® interviewed Brian Kim, MBA, the chief executive officer of Mission Bio, to learn more about the company's activities and his thoughts on the field of cell and gene therapy in general. Kim discussed his thoughts on the trends currently being seen in the industry and also spoke about Mission Bio's goals for collaboration and partnerships.

5. enGene Snags FDA RMAT Designation for Bladder Cancer Gene Therapy

The FDA has granted regenerative medicine advanced therapy (RMAT) designation to enGene's detalimogene voraplasmid (also known as detalimogene, and previously referred to as EG-70), an investigational nonviral immuno-oncology gene therapy, for the treatment of high-risk bacillus Calmette-Guérin (BCG)-unresponsive nonmuscle-invasive bladder cancer with carcinoma in situ.

6. Bio-Techne and US Pharmacopeia Team Up to Tackle Gene Therapy Development

Bio-Techne and US Pharmacopeia (USP) have entered a distribution agreement in which the former company gains the ability to sell the latter's recombinant AA reference standards alongside its analytical solutions, a move aimed at aiding gene therapy development. "We are thrilled to engage Bio-Techne as an authorized distributor, which enables USP to expand access of our solutions within the scientific community and beyond, ensuring that safe and quality therapeutic products reach the market for the benefit of patients worldwide," Fouad Atouf, PhD, the senior vice president of global biologics at USP said in a statement.

7. Nicole Verdun, MD, Placed on Administrative Leave by FDA

Nicole Verdun, MD, the director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, has been placed on leave by the FDA, along with her deputy Rachel Anatol, PhD. Notably, advocacy organization Alliance for Regenerative Medicine put out a statement expressing disappointment at the agency's decision: "Over the past two years, Nicole and Rachel have modernized the FDA’s regulatory approach to cell and gene therapy (CGT), earning the trust and respect of the CGT community and helping to ensure that the FDA was the global leader in this evolving field."

8. Anixa Continues Progress on Ovarian Cancer CER-T

Anixa Biosciences stated that it has begun dosing of patients in the fourth dose-escalation cohort of its phase 1 clinical trial (NCT05316129) evaluating its novel follicle stimulating hormone receptor (FSHR)-targeting chimeric endocrine receptor (CER) T-cell therapy for the treatment of ovarian cancer. "With no dose-limiting safety issues observed in the third cohort, we have advanced to a higher dose level that is thirty times greater than the starting dose," Amit Kumar, PhD, the chairman and CEO of Anixa, said in a statement. "Although the study is primarily focused on safety at these early, low-dose levels, we have seen promising signs of potential efficacy. "