Lysosomal Disorders

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.

The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.

The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.

The interim study results presented at ASGCT 2022 likely represent the longest follow-up data to date for the largest cohort of infants with XSCID treated with lentiviral vector gene therapy.

Catch up on the latest news and announcements in cell and gene therapies presented at the annual ASGCT meeting.

Multiple insertions were observed in affected participants, including insertions in the MECOM and PRDM16 genes.

Data from 2 patients dosed in the Imagine-1 clinical trial were presented in a late breaking session at ASGCT 2022.

Principal investigator Barry Greenberg, MD, FHFSA, shares extended results from the phase 1 trial and also preliminary pediatric safety results in a presentation at ASGCT 2022.

Review top news and interview highlights from the week ending May 13, 2022.

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

The hold was originally placed after reports of 2 serious adverse events in pediatric patients.

Review top news and interview highlights from the week ending May 6, 2022.