Lysosomal Disorders

Among patients who received the pivotal dose level, a median reduction of 85% in HS D2S6 levels was seen in the CSF.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

Review top news and interview highlights from the week ending August 30, 2024.

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 August 23, 2024.

Review top news and interview highlights from the week ending August 16, 2024.

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

In light of the FDA’s decision, 4DMT plans to continue enrolling patients in INGLAXA before the end of 2024.

Review top news and interview highlights from the week ending August 9, 2024.

The chief medical officer at Creyon Bio discussed future applications for the company’s AI-guided discovery platform.

NGN-401 is being evaluated in an early phase clinical trial which has been selected for the FDA’s START Pilot Program.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

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.