Neurotech expects that the therapy will become available in the United States in June 2025.

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

According to Ocugen, the EMA has granted OCU410 and OCU410ST Advanced Therapy Medicinal Product classification.

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.

The chief executive officer of Eledon Pharmaceuticals discussed the company’s collaboration with EGenesis.

The agency has set the PDUFA action date for the BLA as August 31, 2025.

In observance of World Hearing Day, held annually on March 3, CGTLive® took a look back at the past year's news and expert insights in gene therapy for hearing loss indications.

Following up on Rare Disease Day, observed on February 28, Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA, shared insights on the advances and promise for treating spinal muscular atrophy.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

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

In honor of Rare Disease Day, observed this year on February 28, Amber Freed, the founder of SLC6A1 Connect, shared thoughts on advocacy and efforts to raise awareness for rare genetic disease SLC6A1.

The company intends to pursue a full IND application for troculeucel in FTD.

The chief executive officer of ASGCT broke down the highlights of the organization’s final report for 2024.

Out of 11 treated children who had at least 1 posttreatment assessment, 10 showed improved hearing at various decibel hearing levels.

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

Alfred L. Garfall, MD, MS, discussed data from the phase 2 BMT CTN 1902 trial.

The PDUFA action date for the BLA has been set at August 27, 2025.

The FDA’s decision was based on a review of findings from a phase 1b clinical trial.

The director of the Autologous Hematopoietic Cell Transplantation, Cell Therapy and Transplant Program at the Hospital of the University of Pennsylvania discussed data from the phase 2 BMT CTN 1902 trial.

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

The data monitoring committee reviewed safety data from the trial’s first cohort, which included 3 patients, and deemed the findings favorable.

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

The company made the decision based on a lack of interest from the patient and clinician communities.

BRTX-100 is currently being evaluated in a prospective, randomized, and controlled phase 2 clinical trial.

Nirav Shah, MD, an associate professor of medicine at Medical College of Wisconsin, discussed efficacy data presented at the 2025 Tandem Meetings.