GenSight Biologics Inc., (EURONEXT: SIGHT), Paris, France, has announced the results from the final analysis of the REALITY natural history study, which reaffirms the poor… Read More »GenSight Biologics has reported a natural history for LHON (with ND4 mutations) showing that a novel treatment could provide a convincing benefit.
Kubota Vision Inc. has announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has awarded an orphan products clinical… Read More »Kubota Vision Inc., (Seattle, US) has received a $1.6M orphan products clinical trial support for Stargardt disease.
AsciepiX Therapeutics Inc., has announced the receipt of $35 million Series A financing led by Perceptive Xontogeny Venture Fund. The proceeds of the funding will… Read More »AsciepiX Therapeutics Inc., (New Jersey, US) has announced a $35M investment to be used for the treatment of retinal diseases through Phase 1/2a clinical trials
MeiraGTx (Nasdaq: MGTX), has announced six-month data from an ongoing phase 1/2 clinical trial of AAV-RPGR for the treatment of patients with X-linked retinitis pigmentosa (XLRP).… Read More »A UK gene therapy company (MeiraGTx) has announced positive clinical data for the treatment of X-linked retinitis pigmentosa (XLRP)
A Reuters and Washington Post article have confirmed that Novartis AG, and a former subsidiary (Alcon), have agreed to pay $347 million fines to “resolve… Read More »Novartis and Alcon settled bribery fines of $347M related to Lucentis drug and artificial lenses
Alkahest Inc., a clinical stage biopharmaceutical company, announced the initiation of a Phase 2b clinical trial of its orally administered small molecule CCR3 inhibitor, AKST4290.… Read More »Phase 2b study for AKST4290, sponsored by Alkahest Inc. aims to pursue CCR3 inhibitor for neovascular AMD
Applied Genetic Technologies Corporation Inc. (Nasdaq: AGTC) has announced the publication of positive preclinical data for X-linked retinitis pigmentosa. The research study, entitled, “Dose Range Finding… Read More »Applied Genetic Technologies Corporation (NASDAQ: AGTC; Florida, USA) announced the publication of positive preclinical data for X-linked retinitis pigmentosa
Editas Medicine, Inc. (Nasdaq: EDIT), in collaboration with Allergan plc (NYSE: AGN), has announced the dosing of the first patient for LCA of a novel gene… Read More »Leber congenital amaurosis (LCA) initiates a Phase 1/2 trial for CRISPR gene therapy study following an earlier commercial success in gene therapy
MeiraGTx, a gene therapy company based in New York, USA, has announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Advanced… Read More »MeiraGTx (Nasdaq: MGTX) has announced the receipt of Priority Medicines (PRIME) and Advanced Therapy Medicinal Product (ATMP) designations from the EMA
Neuropath Therapeutics, a start-up company based in Wuhan Optics Valley Biological City, China, has announced a Series A funding round on April 8th for €16.9M… Read More »Neuropath Therapeutics in Wuhan, China has announced a Series A investment of €16.9M for the development of Leber’s Hereditary Optic Neuropathy (LHON)