Lysosomal Disorders

The trial is being launched by the University of Florida Center for Regenerative Medicine in collaboration with RESTEM.

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

The FDA will consider RGX-121's BLA in an accelerated approval pathway.

Review top news and interview highlights from the week ending July 29, 2022.

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

Mustang Bio plans to add more clinical trial sites in the near future.

Review top news and interview highlights from the week ending July 22, 2022.

Upstaza has been granted marketing authorization by the European Commission.

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

The associate professor from UC San Diego discussed promising efficacy and safety data from a phase 1/2 trial.

Review top news and interview highlights from the week ending July 15, 2022.

AVROBIO decided to deprioritize their Fabry disease gene therapy program in January 2022.

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

A 93% reduction in serum TTR remained consistent in 6 patients at 6 months.

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

Review top news and interview highlights from the week ending July 1, 2022.

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

Michael Parini, chief executive officer and director, Freeline Therapeutics, discussed the company’s pipeline and mission.

Review top news and interview highlights from the week ending June 24, 2022.

B-VEC recently showed efficacy in the phase 3 GEM-3 trial.

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

The chief scientific officer of the CMT Research Foundation discussed investigative cell and gene therapy approaches for treating CMT.

Review top news and interview highlights from the week ending June 17, 2022.

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

Takashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.

Review top news and interview highlights from the week ending June 10, 2022.

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted in favor of the benefits of bluebird bio's eli-cel in patients with cerebral andrenoleukodystrophy despite substantial safety concerns.

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

Review top news and interview highlights from the week ending June 3, 2022.

The cofounder, president, and chief executive officer of Forge Biologics discussed the company’s approach to gene therapy manufacturing.