information: Submission of a clinical trial application
Announcement: submission of a clinical trial application
Company: Gensight Biologics (France)
- gene therapy. GS030 is based on optogenetics, a technology that makes cells responsive to light. GS030 consists of a gene therapy product comprising a gene encoding a photoactivatable channelrhodopsin protein, delivered via a modified AAV2 (AAV2 7m8) and biomimetic goggles that stimulate the engineered retinal cells. The images are projected by a light source that uses a specific wavelength onto the retina. Once this optogenetic and photoactivable protein is expressed, it confers a photoreceptor-like function to the target cell, enabling the restoration of vision in patients with extremely reduced vision or who are blind due to retinitis pigmentosa.
- GS030 was granted Orphan Drug Designation in the U.S. and Europe.
Disease: retinitis pigmentosa
area: Rare diseases - Genetic diseases - Ophtalmological diseases
- PIONEER is a first-in-man, multi-center, open label dose-escalation study to evaluate the safety and tolerability of GS030 in subjects with retinitis pigmentosa. GS030 is the combination of a gene therapy (GS030-DP) administered via a single intravitreal injection and a wearable optronic visual stimulation device (GS030-MD). Eligible patients will be those affected by end-stage non-syndromic retinitis pigmentosa with vision not better than “counting fingers”.
As per protocol, three cohorts of three subjects each will be administered an increasing dose of GS030- DP via a single intravitreal injection in their worse affected eye. A fourth extension cohort will receive the highest tolerated dose. An independent Data Safety Monitoring Board (DSMB) will review safety data of all treated subjects in each cohort and make recommendations before moving to the next higher dose. The primary outcome analysis will be the safety and tolerability at one year post-injection.
- • On January 10, 2018, GenSight Biologics announced UK Medicines and Healthcare Regulatory Agency (MHRA) acceptance of its Clinical Trial Application (CTA) to initiate the PIONEER Phase I/II study of GS030 in patients with retinitis
pigmentosa. GenSight expects to treat the first patient in the United Kingdom in the first quarter of 2018. GenSight is in active dialogue with global regulatory agencies and intends to file additional IND and CTA submissions in 2018.