Lysosomal Disorders

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

Review some of our most-viewed coverage of advancements in gene therapies, including study data and clinical trial updates.

Review some of our most-viewed interviews with clinicians, researchers, and biotech companies about expanding applications of cell and gene therapies.

Review top news and interview highlights from the week ending December 23, 2022.

Both REGENXBIO and Rocket Pharmaceuticals have announced trial updates in their gene therapy programs for rare diseases.

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

With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.

No treatment-related serious adverse events were reported.

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

REGENXBIO expects to provide updated interim data from the trial in the first half of next year.

AVROBIO plans to initiate a global phase 2/3 trial of AVR-RD-02 in GD3 in the second half of 2023.

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

The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach.

Review top news and interview highlights from the week ending December 2, 2022.

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

Lysogene’s MPS IIIA gene therapy program recently failed its primary endpoint in the phase 2/3 AAVANCE trial.

The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.

Review top news and interview highlights from the week ending November 25, 2022.

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

Statistically significant improvements in cognitive development were only seen in the cohort of patients younger than 30 months.

The gene therapy was approved in the European Union earlier this year.

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

The first 3 patients dosed have been attack-free for up to 10.5 months.

The Genomics and Epigenetic Guided Safe Harbor mapper will aid in the future design of gene-editing therapies.

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