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According to REGENXBIO, HS D2S6 is “a key biomarker of MPS II brain disease that is reasonably likely to predict clinical benefit.”

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

The findings come from patients with DMD44 treated in the phase 1/2 EXPLORE44 clinical trial and the phase 2 EXPLORE44OLE clinical trial.

Capricor stated that it was not notified in advance by the FDA that the latter would be publishing the CRL publicly on its website.

Robert Alexander Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, expressed optimism for RNA therapy's future.

The director of RNA therapeutics at MGB’s Gene and Cell Therapy Institute expressed optimism for RNA’s potential role in CAR T-cell therapy, gene editing, and more.

Mind Moments, a podcast from our sister site Neurology Live, held an exclusive interview with Barry Byrne, MD, PhD.

Alex Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, discussed the Institute's work in exploring the new modality.

Review top news and interview highlights from the week ending September 5, 2025.

Notably, the European Medicines Agency also granted MCO-010 orphan drug designation for 5 retinal dystrophy categories.

John Murphy, PhD, and Tia DiTommaso, PhD, of Arbor Biotechnologies, discussed ABO-101, the company’s gene therapy for primary hyperoxaluria type 1.

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

The chief scientific officer and senior director of preclinical pharmacology Arbor Biotechnologies discussed ABO-101, the company’s gene therapy for primary hyperoxaluria type 1.