BBS1 Gene Therapy Clinical Trial Receives MHRA Approval: A Major Milestone for the BBS Community

We are proud to share an important milestone for the Bardet-Biedl Syndrome community. The clinical trial that A Race Against Blindness has been fundraising to support has officially received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) and is now publicly listed on ClinicalTrials.gov . This marks a significant step forward for research focused on BBS1-related retinal degeneration and represents years of work by researchers, clinicians, and advocates committed to finding meaningful treatment options. MHRA Approval: Why This Matters MHRA approval is a rigorous regulatory process that evaluates whether a clinical trial meets strict standards for: Patient safety Ethical oversight Scientific and clinical trial design For rare diseases like Bardet-Biedl Syndrome, reaching this stage is a meaningful achievement. It signals that a potential therapeutic approach has advanced from laboratory research into human clinical evaluation . About the Clinical Trial Study Title: First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration (AXIS)