News

SAR402663 comprises a one-time intravitreal treatment and is intended to inhibit VEGF by providing a gene for soluble FLT01.

The chief scientific officer of Solvias discussed potential uses for the company’s platform.

With regard to safety, DTX401’s safety profile was characterized as “acceptable and expected.”

In Episode 6 of ImmunoLogic, Stephen Schoenberger, PhD, discussed his research on customized cancer vaccines.

For World Lymphoma Awareness Day, held annually on September 15, take a look at the stories that have stood out in lymphoma cell therapy in recent months.

In observance of Duchenne Action Month and World Duchenne Day, held annually in September, we took a look back at recent news in gene and cell therapy for DMD.

Review top news and interview highlights from the week ending September 12, 2025.

Capsida has made the move to pause the trial voluntarily.

The company specifically highlighted that one patient who was treated in the trial’s first cohort is still alive at 28 months.

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.

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

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.

The rAAVrh8 gene therapy delivers HEXA and HEXB genes and has posted positive phase 1/2 results in infantile GM2 gangliosidosis, with enhancements in enzymatic activity and neurological outcomes.

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

Review top news and interview highlights from the week ending August 29, 2025.

Annaiz Grimm, BS, a research scientist at Seattle Children's Research Institute, discussed the potential use of EngTregs to treat autoimmune disease.

The research scientist at Seattle Children's Research Institute discussed a potential alternative to standard of care immunosuppressive therapy.

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