Lysosomal Disorders

Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.

The professor of pediatrics at Stanford Cancer Institute discussed recent data from clinical trials of the lentiviral gene therapy RP-L102.

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

NGN-401 is Neurogene’s second investigational gene therapy to enter clinical trials.

With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.

Review top news and interview highlights from the week ending January 20, 2023.

The FDA has lifted a clinical hold placed in the summer of 2022 due to a mild but medically significant case of peripheral sensory neuropathy.

The company is reprioritizing to focus on its clinical stage programs, which ran into a number of setbacks in 2022.

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

Forge Biologics’ FBX-101 previously demonstrated promising safety and efficacy in early data from the phase 1/2 RESKUE clinical trial.

Review top news and interview highlights from the week ending January 13, 2023.

The assistant member of the bone marrow transplantation and cellular therapy department at St. Jude Children’s Hospital discussed preclinical research presented at ASH 2022.

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

The assistant member at St. Jude Children’s Research Hospital discussed the ASH 2022 basic and translational science session she moderated.

Some patients previously treated with GNT-003 were able to maintain the stoppage of phototherapy for at least 1 year.

No serious adverse events related to the gene therapy have been reported.

Promising results were previously reported from a proof-of-concept study involving 8 patients.

Review top news and interview highlights from the week ending January 6, 2023.

The company is kicking off 2023 by announcing planned advancements for several gene therapies in its pipeline.

Initial data from the trials of HMI-103 and HMI-203 are expected in 2023.

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

Review some of our most-viewed coverage of advancements in gene therapies, including study data and clinical trial updates.

Review some of our most-viewed interviews with clinicians, researchers, and biotech companies about expanding applications of cell and gene therapies.

Review top news and interview highlights from the week ending December 23, 2022.

Both REGENXBIO and Rocket Pharmaceuticals have announced trial updates in their gene therapy programs for rare diseases.