Lysosomal Disorders

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.

Three participants with ATTR amyloidosis have received follow-on doses of NTLA-2001 with no serious complications for up to 3 years of follow-up.

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 21, 2024.

The company anticipates that it will be able to submit a BLA in late 2024 or early 2025.

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

An investigator noted challenging side effects related to immunosuppressant treatment.

The scientist from Krembil Research Institute shared takeaways from the session she chaired.

There was a 98% (2.7% SD) mean reduction in monthly HAE attack rate through the patients’ most recent assessment.

Review top news and interview highlights from the week ending June 14, 2024.

The pediatric neurologist at Nemours Children’s Health discussed next steps in the field to fully enable the benefits of gene therapy.

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

The FDA granted the designation after reviewing safety and efficacy data from phase 1 of the trial.

Ineka Whiteman, PhD, head of research and medical affairs, BDSRA Australia, discussed what has occurred in the field of Batten disease gene therapy in the past year.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison shared highlights from the discussion.

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

Review top news and interview highlights from the week ending June 7, 2024.

The associate professor of clinical pediatrics at Cincinnati Children's discussed further investigations into novel therapies and new research.

Other of the company’s RNAi therapies have recently shown promising data in hyperlipidemia and asthma.