Stacy Grieve, PhD,  —

GenSight Biologics Reports GS010 Has Promise for Improving Long-term Vision in LHON Patients

GenSight Biologics has reported that a group of patients with Leber Hereditary Optic Neuropathy (LHON) who were treated with a single injection of its compound GS010, showed long-term sustained improvement in visual acuity when reassessed 2.5 years later. In a press release, Bernard Gilly, CEO and co-founder of GenSight,…

NeuroVive Gets Signal That EU Will Give Its Mitochondrial Therapy KL1333 Orphan Drug Status

NeuroVive Pharmaceutical has received a signal from European regulators that its mitochondrial disease therapy KL1333 is likely to receive Orphan Drug Designation, which could accelerate its development and approval. The company is seeking Orphan Drug Designation for KL1333 as treatment for a condition known as mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like…

Michael J. Fox Foundation Awards Mission Therapeutic, University of Oxford Grant To Test USP30 Inhibitor as Parkinson’s Treatment

The Michael J. Fox Foundation has awarded Great Britain’s Mission Therapeutics and the University of Oxford a grant to test the novel USP30 inhibitor on a range of disease models including Parkinson’s disease. The New York-based foundation announced its grant, whose amount was not disclosed, in a press release on…