Lysosomal Disorders

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

The chief medical officer at Creyon Bio discussed findings from a treated patient with a TNP02 missense mutation.

Review top news and interview highlights from the week ending August 2, 2024.

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

The chief medical officer and head of translational research at Ring Therapeutics discussed research presented at ASGCT 2024.

The company acquired the program, along with Spark Therapeutics’ pipeline, in December 2019.

Review top news and interview highlights from the week ending July 26, 2024.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison discussed the state of cell and gene therapy development.

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

Both bluebird bio and Vertex Pharmaceuticals have received unfavorable opinions from the Office of the Inspector General on the matter.

Review top news and interview highlights from the week ending July 19, 2024.

The chief medical officer at Creyon Bio overviewed the development of an allele-selective TNPO2 protein for a single patient with a de novo pathology.

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

The FDA announced its START Pilot Program in October 2023 with a similar goal.

Review top news and interview highlights from the week ending July 12, 2024.

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

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.

The phase 1/2 GALILEO-1 trial has completed enrollment and a phase 3 trial is planned for 2025.

Review top news and interview highlights from the week ending July 5, 2024.

The therapy, rAAV-Olig001-ASPA, has shown positive clinical benefits in several interim updates from an ongoing, first-in-human phase 1/2 clinical trial (NCT04833907).

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

CGTLive takes a look at the path gene therapies have blazed in leukodystrophies, the challenges they’ve faced, and the road they’ve paved.

Review top news and interview highlights from the week ending June 28, 2024.

The FDA has requested limited additional CMC information.