News

Review top news and interview highlights from the week ending May 16, 2025.

The physician-scientists at CHOP discussed what lies on the horizon after KJ’s treatment with a CRISPR-based gene editing therapy for CPS1 deficiency.

The director of the Mass General Brigham Gene and Cell Therapy Institute also discussed the Institute’s presentations at ASGCT’s 2025 conference.

The PDUFA action date for the BLA has been set as November 9, 2025.

The physician-scientists at CHOP discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

The senior principal scientist at Regeneron discussed an advanced approach to enabling gene therapy redosing.

The head of research at Mass General Brigham’s Gene and Cell Therapy Institute discussed how innovative research can be held back by financial considerations.

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

The new data were presented in a poster at the American Society of Gene and Cell Therapy’s 2025 meeting.

James Beck, PhD, chief scientific officer of the Parkinson’s Foundation, discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed treatment decisions in newly diagnosed SMA.

In observance of World Lupus Day, held annually on May 10, we took a look back at recent progress in bringing cell therapy to this autoimmune disease.

Review top news and interview highlights from the week ending May 9, 2025.

In a mid-cycle review meeting, the agency also noted that the review committee found no major deficiencies with the BLA package.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed the evolving SMA treatment landscape.

In observance of World Ovarian Cancer Day, held annually on May 8, we took a look back at news in cell therapy for ovarian cancer indications.

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

J&J purchased the full rights to bota-vec in a 2023 deal with MeiraGTx

The company’s CEO announced via LinkedIn that the company is shutting down its operations, citing a “difficult funding environment.”

The chief scientific officer of the Parkinson’s Foundation discussed how broad genetic testing through PD GENEration is uncovering valuable prognostic and diagnostic insights in Parkinson disease.

For Healthy Vision Month, observed annually in May by the patient and clinician communities, CGTLive® has decided to take a closer look at this ongoing clinical trial.

The chief scientific officer of the Parkinson’s Foundation discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations.

Linda Marbán, PhD, the CEO of Capricor Therapeutics, discussed future plans for the product after the promising data presented at MDA’s 2025 conference.

Review top news and interview highlights from the week ending May 2, 2025.

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

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

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

Matthew Alexander, PhD, a neuromuscular expert, discussed the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.