GenSight Receives UK Approval to Start Phase I/II Trial of Gene Therapy for Retinitis Pigmentosa
GenSight Biologics announced Jan. 10 that the UK’s Medicines and Healthcare Regulatory Agency (MHRA) has given the company approval to initiate its PIONEER Phase I/II study of GS030 for patients with retinitis pigmentosa (RP).
GenSight Biologics announced Jan. 10 that the UK’s Medicines and Healthcare Regulatory Agency (MHRA) has given the company approval to initiate its PIONEER Phase I/II study of GS030 for patients with retinitis pigmentosa (RP).
GS030 is the combination of a gene therapy (GS030-DP) administered through a single intravitreal injection and a wearable optronic visual stimulation device (GS030-MD), France-based GenSight said.
PIONEER is a first-in-human, multi-center, open label dose-escalation study to evaluate safety and tolerability, GenSight said. Eligible patients are those affected by end-stage non-syndromic RP with vision “not better than counting fingers,” the company said.
In the study, three cohorts of three subjects each will be administered an increasing dose of GS030-DP through a single injection in their worse affected eye, GenSight said. A fourth extension cohort will receive the highest tolerated dose. An independent data safety monitoring board will review safety data of all treated subjects in each cohort and make recommendations before moving to the next higher dose, GenSight said.
The primary outcome analysis will be the safety and tolerability at one year after injection. GenSight expects to treat the first patient in the UK in the first quarter of 2018, the company said.
GS030 was granted orphan drug designation in the US and Europe. GenSight intends to file investigational new drug and clinical trial submissions in 2018, the company said.
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