Lysosomal Disorders

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

Forge Biologics’ FBX-101 previously demonstrated promising safety and efficacy in early data from the phase 1/2 RESKUE clinical trial.

Review top news and interview highlights from the week ending January 13, 2023.

The assistant member of the bone marrow transplantation and cellular therapy department at St. Jude Children’s Hospital discussed preclinical research presented at ASH 2022.

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

The assistant member at St. Jude Children’s Research Hospital discussed the ASH 2022 basic and translational science session she moderated.

Some patients previously treated with GNT-003 were able to maintain the stoppage of phototherapy for at least 1 year.

No serious adverse events related to the gene therapy have been reported.

Promising results were previously reported from a proof-of-concept study involving 8 patients.

Review top news and interview highlights from the week ending January 6, 2023.

The company is kicking off 2023 by announcing planned advancements for several gene therapies in its pipeline.

Initial data from the trials of HMI-103 and HMI-203 are expected in 2023.

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.