French Biotech firm raises €19M for gene therapy trial RP
Posted on: Monday, November 13th, 2017
The funds will be used to finance the development of Horama’s lead candidate, HORA-PDE6B, which is about to enter clinical trials. Last month, the French ANSM authorized the start of a study at the Nantes University Hospital that will recruit 12 patients to test the gene therapy in humans for the first time.
HORA-PDE6B is intended to treat retinitis pigmentosa in patients that develop the disease because of a mutation in the PDE6β gene. The gene therapy consists of a recombinant adeno-associated virus (AAV) in which the viral DNA is replaced by a functional copy of the PDE6β gene.
This DNA is injected under the retina, right between the photoreceptors and the retinal pigment epithelium, where the cells use it to express the correct version of the PDE6β protein. And since these cells are not renewed over time, the therapy has the potential to prove effective for years after a single injection.