Most patients with RP treated with MCO-010 had clinically meaningful improvements in vision.

The Study Safety Committee has recommended that the trial proceed to enroll patients in the highest-dose cohort.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about preclinical research with an iPS cell-derived product.

The gene-edited cell therapy had previously been under a partial clinical hold restricting pediatric enrollment to the clinical trial.

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

Intellia Therapeutics received clearance to enroll patients from the US in the global phase 2 portion of its clinical trial earlier this month.

The associate professor of neurology at Columbia University spoke about the current and future treatment landscape in ALS.

OCU400 is also being evaluated in other retinal degeneration indications.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about the session she chaired at the 2023 MDA Clinical and Scientific Conference.

The FDA will determine the acceptability of the BLA within the next 60 days.

Krystal Biotech's investigational topical gene therapy has shown promise in DEB. It is designed to deliver COL7A1 and restore C7 protein in patients with the rare disorder.

The co-CEO of BrainStorm Cell Therapeutics spoke on the supporting data for the investigational ALS cell therapy that will be discussed at the meeting, and expectations for it.

The company's persistence in bringing NurOwn to the market amidst negative regulatory feedback has garnered an AdComm meeting.

The nurse practitioner from Washington University in St. Louis discussed the practical considerations in gene therapy track she chaired at the 2023 MDA Conference.

The child neurologist and neuromuscular physician at Virginia Commonwealth University also discussed the session she chaired at the 2023 MDA Clinical and Scientific Conference.

Review top news and interview highlights from the week ending March 24, 2023.

The Delphi panel considerations were informed by safety data from 3 clinical trials of the therapy, and included vomiting, myocarditis, acute liver injury, and immune-mediated myositis.

Direct Biologics’ ExoFlo is also being evaluated for the treatment of COVID-19 acute respiratory distress syndrome.

The patient was treated at the new fifth dose level and had concomitant metapneumovirus infection.

The co-founder and chief executive officer of MyoGene Bio spoke about the company's gene editing therapy for Duchenne muscular dystrophy at MDA’s 2023 conference.

The Amplo Biotechnology gene therapy showed positive findings in mouse models, suggestive of the therapy’s development for use in a clinical setting. The AAV therapy delivers DOK7 gene, which has shown benefit in neuromuscular disorders.

Data from the HOPE-2-OLE were presented in a late-breaking session at the 2023 MDA Conference.

All children included in the analysis achieved swallowing, oral nutrition, and airway protection outcomes.

Investigators plan to present full results from the OS analysis of the phase 3 ZUMA-7 trial at an upcoming medical meeting.

Presented at MDA 2023, the gene therapy (also known as delandistrogene moxeparvovec) showed significant improvements on NSAA total score, 10-meter walk/run scores, and time to rise scores relative to an external control cohort.