Gene Therapy News

Genethon receives approval from the UK medicine regulatory agency to start a new clinical trial for an inherited immune deficiency: chronic granulomatous disease

On January 10th, the British Medicine and Healthcare Regulatory Agency (MHRA) approved Genethon’s Phase I / II gene therapy clinical trial application in X-linked chronic granulomatous disease (XCGD) to start at Great Ormond Street Hospital in London. Genethon is also seeking approval for this multicenter trial in Germany, Switzerland and France. In total the trial will include 20 patients (5 per site) to be treated and followed up for 2 years.

Are There Limits to What Gene Therapy Can Achieve in Treating Retinal Blindness?

New Rochelle, NY, March 8, 2013— Follow-up results from one of several groundbreaking gene therapy studies reported in 2008 in patients with an inherited form of retinal blindness confirmed the earlier positive results of substantial improvement in vision that has now persisted for more than 4 years. However, the new study* also showed that while gene therapy was able to improve patients' vision, it did not slow or stop the underlying disease process.


RetroSense Therapeutics Announces Notice of Allowance for New U.S. Patent Application Broadly Covering Optogenetic Approaches to Vision Restoration

RetroSense Therapeutics, a biotechnology company dedicated to developing gene therapy approaches to vision restoration, announced today that the U.S. Patent and Trademark Office has issued a Notice of Allowance for U.S. patent application (No. 12/299,574), which broadly covers methods of restoring visual responses with a variety of optogenetic compounds.