#Usher syndrome coalition trial#

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#Usher syndrome coalition trial#

There is a second trial that is following the long-term effects of those who have participated in the terminated SAR421869 trial.Ĭ listing: Study of SAR421869 in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1BĬ listing: A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B In the February 2019, press release they have announced they are seeking an out-licensing partner and/or co-sponsor. However, since December 2018 clinical trials have been on hold in Phase 1/2 after Sanofi removed it from their pipeline. It is gene replacement therapy where a correct copy of the MYO7A gene is injected into the retina through a lentiviral vector, which has the capacity to carry a large gene like MYO7A. Due to review of clinical development plans and priorities, Sponsor decided to stop development of the product.)Ĭlinical trials to evaluate the safety and the tolerability of ascending doses of subretinal injections of SAR421869 (UshStat) in USH1B patients are taking place in Paris, France at the Hopital Nationale des Quinze-Vingt and in Portland, Oregon at the Casey Eye Institute. (TERMINATED: Study stopped not for safety reasons. The use of dual AAV vectors could also hopefully be applied to other conditions that require large genes.Ĭreate a Nonhuman Primate Model of Usher Syndrome

The hope is that this study will create the basis for a cure for USH1B retinitis pigmentosa (RP). Twelve USH1B patients will be given the dual AAV vectors for the study. This study is the first time the dual AAV vector has been used in humans. The objective of the study USHTher is to test the safety and efficacy of an innovative gene therapy approach that uses dual AAV in retinas of USH1B patients. Additionally, a phase 1/2 clinical trial to investigate the safety and efficacy of dual AAV-MYO7A vectors in humans has been designed. Currently the safety and expression of dual AAV vectors is being assessed in primates. A consortium of researchers around Alberto Aurrichio has worked on dual AAV vectors for non-clinical studies and a clinical trial.