Lysosomal Disorders

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.

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

The renowned professor from University of California Los Angeles reflected on receiving the Outstanding Achievement Award from the ASGCT.

Stefan Braam, PhD, chief executive officer of Ncardia and Cellistic discussed the companies’ work in the cell therapy space.

RP-L301 also improved hemolysis and eliminated the need for red blood cell transfusions for up to 1year after therapy.

Review top news and interview highlights from the week ending May 27, 2022.

The chief executive officer of Ring Therapeutics, Tuyen Ong, MD, discussed the potential of anellovirus vectors for use in gene therapy.

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

The positive opinion was based on findings from studies conducted in Taiwan.

The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.

In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.

Review top news and interview highlights from the week ending May 20, 2022.