Around the Helix: Cell and Gene Therapy Company Updates – November 26, 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 Approves Itvisma, a New Intrathecal Route for Novartis’ SMA Gene Therapy

The FDA has approved Novartis’ onasemnogene abeparvovec-brve (also known as OAV101 IT), an intrathecally-delivered version of the separately marketed gene therapy onasemnogene abeparvovec (Zolgensma), for the treatment of spinal muscular atrophy (SMA) in people 2 years of age and older who have a confirmed mutation in the survival motor neuron 1 (SMN1) gene. It is to be marketed under the name Itvisma.

2. Platform Approaches and Industry Investment in Cell and Gene Therapy

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, and Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center, discussed bottlenecks to cell and gene therapy development and how institutions and companies are trying to overcome them.

3. Carrie Haverty, MS, CGC, on Exploring Genetic Counseling's Growth and Challenges

Carrie Haverty, MS, CGC, the National Society of Genetic Counselors (NSGC)'s president elect, discussed the evolving role of genetic counselors, emphasizing personalized care, gene therapy advancements, and the need for multidisciplinary integration in healthcare.

4. Michelle Alabek, MS, CGC, on the Role of Genetic Counselors in Supporting Patients Receiving Gene Therapy

The genetic counselor in the Department of Ophthalmology at the University of Pittsburgh Medical Center spoke on the growing role of genetic counselors in guiding patients undergoing gene therapy.

5. Sarepta Launches New Trial Cohort to Test Elevidys Immunosuppression Regimen

The FDA has cleared Sarepta to initiate cohort 8 in the phase 1 ENDEAVOR clinical trial (NCT04626674) for Duchenne muscular dystrophy gene therapy delandistrogene moxeparvovec-rokl (marketed as Elevidys). The new cohort in the open-label clinical trial will test a new sirolimus immunosuppressive regimen aimed at reducing acute serious liver injury (ALI) and acute liver failure (ALF) risks with the hope of aligning with the FDA on a plan to allow for a return of eligibility of nonambulatory patients for treatment with the gene therapy.

6. FDA Gives Sangamo Green Light to Start Rolling BLA Submission for Isa-Vec

Following an October 2025 meeting with the FDA in which the agency confirmed its agreement that eGFR slope can be used as an end point for the support of an accelerated approval pathway for Sangamo's investigational Fabry disease gene therapy isaralgagene civaparvove (isa-vec, ST-920), Sangamo has received acceptance of a request to start a rolling submission and review of a biologics license application (BLA). Sangamo now plans to begin a rolling submission before the end of the year.