ReNeuron said its hRPC clinical trial's data safety monitoring board has given approval for the study to progress into its Phase II element. That decision was said to be based on short term data from the nine RP patients treated in the Phase I part of the study, which indicated that the hRPC cell therapy was “safe and well tolerated” at the three doses tested.
One study altered the order of atoms in DNA to rewrite the human genetic code and the instructions for life. The other edited RNA, which is a chemical cousin of DNA and unlocks the information in the genetic code.
Spark Therapeutics, the gene therapy company, announced today that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended approval of Luxturna (voretigene neparvovec), an investigational, potential one-time gene therapy.
A French biotech company based in Paris and Nantes, which specialises in gene therapy for the treatment of rare, inherited retinal diseases, has announced that it has been authorised by the French National Agency for Medicines and Health Products Safety (ANSM) to launch a Phase I/II clinical trial for HORA-PDE6B, in the treatment of a retinitis pigmentosa.
Researchers managed to restore sight to mice affected by retinitis pigmentosa after reprogramming their remaining retinal nerve cells.
National sight loss charities RP Fighting Blindness and the Macular Society, have announced a new collaboration to fund talented young researchers in the field of genetic macular diseases.
Chemical changes in the eye that can lead to blindness have been identified by scientists.
Manchester Royal Eye Hospital (MREH) achieves a historical milestone, as it delivers gene therapy at this hospital for the first time. MREH is participating in a gene therapy clinical trial, in collaboration with Saint Mary’s Hospital, for patients with a rare genetic eye condition, x-linked retinitis pigmentosa (XLRP).
Researchers at the University of Louisville have discovered a way to revitalize cone receptors that have deteriorated as a result of retinitis pigmentosa.
The Food and Drug Administration in the US has given orphan drug designation to a gene therapy product intended for treatment of X-linked retinitis pigmentosa.