Noah Stansfield

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.

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.

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.

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.

Prerna Mewawalla, MD, the medical director of Apheresis and a hematologist-oncologist in the Division of Hematology and Cellular Therapy at Allegheny Health Network, spoke on unmet needs in patients who receive early-line CAR-T for R/R MM.

Notably, the FDA granted Papzimeos a full approval, without a requirement for a confirmatory trial.

Ultragenyx anticipates that the full BLA will have been submitted in the fourth quarter of this year.

The new PDUFA date is set for February 8, 2026.

Surbhi Sidana, MD, the assistant professor of medicine, bone marrow transplantation, and cellular therapy at Stanford discussed the benefit of second-line cilta-cel in PFS and OS.

Review top news and interview highlights from the week ending August 22, 2025.

The hold was originally placed on the trial by the FDA on May 23, 2025, following the death of a patient treated in the study.

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

Damien Maura, PhD, a senior scientist at Voyager, discussed the company’s work on developing AAV capsids capable of overcoming patients’ preexisting immunity.