Lysosomal Disorders

David-Alexandre Gros, MD, the chief executive officer of Eledon Pharmaceuticals, discussed the company’s collaboration with EGenesis.

In Episode 2 of ImmunoLogic, Bruce Levine, PhD, discussed the current the risk-benefit-ratio for CAR-T therapy.

Review top news and interview highlights from the week ending March 7, 2025.

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 areas of interest for future study for the Gaucher disease gene therapy product.

Review top news and interview highlights from the week ending February 28, 2025.

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

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.