Around the Helix: Cell and Gene Therapy Company Updates – February 25, 2026

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. Robin E. Miller, MD, on Integrating Gene Therapy into Clinical Care for Sickle Cell Disease

CGTLive® spoke with Robin E. Miller, MD, the director of the Sickle Cell Disease Program at Nemours Children’s Hospital, to learn more about the practical considerations for treatment centers that are integrating or considering integrating gene therapy into their clinical care for sickle cell disease.

2. Can we omit chemotherapy in breast cancer treatment?

Julia C. Tchou, MD, PhD, told the ImmunoLogic cohosts about a study that will allow patients to receive breast cancer treatment that does not include chemotherapy.

3. DMD Gene Therapy Elevidys Shows Functional Benefit at 2 Years in Phase 3 EMBARK Trial

Two-year data from the phase 3 EMBARK clinical trial (NCT05096221) suggest that treatment with delandistrogene moxeparvovec (marketed as Elevidys) was associated with stabilization or slowing of functional decline in ambulatory children with Duchenne muscular dystrophy (DMD), compared with a matched external control cohort receiving standard-of-care corticosteroids.

4. MDA’s Upcoming 2026 Clinical & Scientific Conference

In the lead up to this year's meeting, which will be from March 8 to 11, 2025, in Orlando, Florida, CGTLive™ sat down with Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida, to learn more about the tracks at this year’s conference.

5. Ultragenyx’s GSDIa Gene Therapy Gets PDUFA

The FDA has accepted Ultragenyx’s biologics license application (BLA) for DTX401, an investigational adeno-associated virus serotype 8 (AAV8) vector gene therapy expressing the human G6PC gene that is intended to treat Glycogen Storage Disease Type Ia (GSDIa). The agency has set the Prescription Drug User Fee Act (PDUFA) action date for August 23, 2026, and granted the BLA priority review. “Current dietary approaches to managing GSDIa place an extraordinary burden on individuals and families while still leaving patients with significant medical needs, including the risk of potentially life-threatening episodes of acute hypoglycemia and accumulation of long-term complications over their lifetime,” Eric Crombez, MD, the chief medical officer at Ultragenyx, said in a statement. “If approved, DTX401 would be the first treatment to address the disease at its root cause.”

6. Elevidys Hits Markets in Japan

Sarepta announced that Elevidys has been commercially launched in Japan by Chugai Pharmaceutical. The product, which is the first gene therapy for DMD to be approved in the country, is indicated for patients with DMD who are ambulatory, aged from 3 years to less than 8 years, have a deletion in or of exon 8 and/or exon 9 in the DMD gene, and who test negative for the presence of antiAAVrh74 antibodies. Japan’s approval of the therapy was based on data from clinical trials, including EMBARK. “The commercial launch of Elevidys in Japan marks an important step in expanding access to this therapy, where early intervention may offer the greatest opportunity to preserve muscle function,” Louise Rodino-Klapac‑, PhD, the president of research & development and technical operations at Sarepta, said in a statement.