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Wave Life Sciences’ RNA therapy WVE-N531 generates sustained dystrophin and improves early function in exon 53–skipping Duchenne muscular dystrophy trial.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Robin E. Miller, MD, director of the Sickle Cell Disease Program at Nemours Children’s Hospital, discussed what clinics need in order to start administering gene therapy effectively.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed the tracks at the upcoming 2026 MDA Meeting.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Review top news and interview highlights from the week ending February 13, 2026.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

In episode 8 of ImmunoLogic, Julia C. Tchou, MD, PhD, and the cohosts discussed the potential of combining immunotherapy approaches and surgery in the treatment of breast cancer.

FDA objections centered on study population misclassification risk, external control validity, and surrogate end point evidentiary strength.

The new BLA includes “substantial longer-term data on multiple measures of neurologic benefit”, and is aimed at obtaining an accelerated approval for the gene therapy product.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Review top news and interview highlights from the week ending January 30, 2026.

Taysha also disclosed that it has received written alignment from the FDA on elements of a potential BLA strategy for TSHA-102.

The therapy provided a +6-letter gain in BCVA-evaluable patients.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, also discussed open questions that remain for the future of the hemophilia B gene therapy.

Review top news and interview highlights from the week ending January 23, 2026.

For Glaucoma Awareness Month, CGTLive® interviewed the chair of ophthalmology at the Byers Eye Institute at Stanford University about the potential of cell and gene therapy for the condition.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Ocugen's OCU410 gene therapy shows promising results in reducing lesion growth for geographic atrophy, potentially transforming treatment for retinal health.

Review top news and interview highlights from the week ending January 16, 2026.

We highlighted a few investigational cell and gene therapy candidates to keep an eye on in the first half of the new year.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive® team.

The CRL indicates that the ALLELE clinical trial does not sufficiently show efficacy of the product.

Review top news and interview highlights from the week ending January 9, 2026.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.