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Rare genetic diseases

MeiraGTx Announces Positive 6-Month Data from Phase 1/2 Trial of Investigational Gene Therapy AAV-RPE65 for RPE65-Deficiency

AAV-RPE65 met the primary endpoint of safety and tolerabilityStatistically significant improvement demonstrated in vision-guided mobility and visual function in treated eyes compared to untreated eyesDose selected for pivotal study; Company expects to meet with global regulatory authorities in the second half of 2019 MeiraGTx Holdings plc announced positive data from a Phase 1/2 dose escalation trial of AAV-RPE65, the Company’s investigational gene therapy for the treatment of RPE65-deficiency, a condition that ...

Audentes Therapeutics Presents Positive Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 for X-linked Myotubular Myopathy

At the 22nd Annual Meeting of the American Society of Gene and Cell Therapy, promising results for the AT132 genetic therapy for X-linked Myotubular Myopathy ( XLMTM ) were presented. XLMTM is a rare neuromusclular condition that is caused by mutations in the MTM1 gene. This results in extreme muscle weakness, and a majority of patients require a ventilator to continue breathing, as well as a gastrostomy tube for nutritional support. The average lifespan...

Orchard Therapeutics Begins Early Trials for Genetic Treatments of Wiskott-Aldrich Syndrome and Beta-Thalassemia

Orchard Therapeutics has begin proof of concept trials for two separate rare diseases for which the current standard of care is quite poor. The first therapy is called OTL-103, and it is an autologous hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome (WAS). WAS is a life threatening immune disorder and the current standard of care is a bone marrow transplant, which carries a high risk for mortality. Orchard Therapeutics...