Lysosomal Disorders

RGX-121 was well-tolerated across all 3 of the dose cohorts with no treatment-related serious adverse events reported.

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

Updated data from the STAAR clinical trial were presented at the SSIEM Annual Meeting.

Review top news and interview highlights from the week ending August 26, 2022.

iECURE and the University of Pennsylvania Gene Therapy Program have collaborated to develop the GTP-506 program.

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 August 19, 2022.

The pheEDIT clinical trial initiated in June 2022.

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

Preclinical data showed a reduction of Gys1 mRNA and protein of more than 80% in multiple skeletal tissues.

Preclinical data showed hARSA activity levels in the brain that are predictive of functional improvements.

Review top news and interview highlights from the week ending August 12, 2022.

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 August 5, 2022.

The trial is being launched by the University of Florida Center for Regenerative Medicine in collaboration with RESTEM.

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

The FDA will consider RGX-121's BLA in an accelerated approval pathway.

Review top news and interview highlights from the week ending July 29, 2022.

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

Mustang Bio plans to add more clinical trial sites in the near future.

Review top news and interview highlights from the week ending July 22, 2022.

Upstaza has been granted marketing authorization by the European Commission.

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

The associate professor from UC San Diego discussed promising efficacy and safety data from a phase 1/2 trial.

Review top news and interview highlights from the week ending July 15, 2022.