Lysosomal Disorders

The adult metabolic consultant at Salford Royal Hospital discussed areas of interest for future study for the Gaucher disease gene therapy product.

Review top news and interview highlights from the week ending February 21, 2025.

The PDUFA action date for the BLA has been set at August 18, 2025, and no advisory committee meeting is currently planned.

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

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed results she presented at the 21st Annual WORLDSymposium.

Review top news and interview highlights from the week ending February 14, 2025.

The adult metabolic consultant at Salford Royal Hospital discussed results she presented at the 21st Annual WORLDSymposium.

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 February 7, 2025.

A positive mean eGFR slope was seen in 23 patients who reached 1 year of follow-up in the phase 1/2 STAAR trial.

With regard to safety, Ultragenyx characterized the gene therapy as “generally well-tolerated."

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

Among the 5 patients in the efficacy set, 4 patients ceased receiving their previous standard of care therapy.

Review top news and interview highlights from the week ending January 31, 2025.

In Episode 1 of ImmunoLogic, Stephen J. Schuster, MD, discussed the potential of treating lymphoma without chemotherapy.

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 January 24, 2025.

The company stated it expects to submit a BLA to the FDA next year.

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 January 17, 2025.

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 January 10, 2025.

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

Ultragenyx is seeking accelerated approval for the BLA based on the use of CSF heparan sulfate data from the phase 1/2/3 Transpher A clinical trial.

Arbor noted it has designed redePHine, a phase 1/2 clinical trial (NCT identifier pending) that will evaluate ABO-101 in adults and children with PH1.