Date: 2014-10-14
Type of information: Initiation of development program
phase:
Announcement: initiation of development program
Company: Genable Technologies (Ireland)
Product: RhoNova™ (GT038) adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing DNA encoding a rhodopsin gene
Action
mechanism: gene therapy. GT038 is an adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing a rhodopsin gene for the treatment of rhodopsin-linked retinitis pigmentosa. Development of GT038 began 10 years ago and was performed by Jane Farrar, Ph.D., Paul Kenna, Ph.D., and their colleagues at Trinity College Dublin. The Foundation Fighting Blindness funded early GT038 research, and, today, its chief researcher officer, Stephen Rose, Ph.D., serves Genable in a consultative capacity. Patricia Zilliox, Ph.D., the chief drug development officer at the Foundation’s Clinical Research Institute, also provides the company with insights into the clinical development of GT038.
Disease: retinitis pigmentosa
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Country:
Trial details:
Latest
news: * On October 10, 2014, Genable Technologies announced that the company is on course to launch the first gene therapy clinical trial with GT038 for people with an autosomal dominant retinal disease. The condition is retinitis pigmentosa caused by mutations in the gene rhodopsin (RHO), which affects about 30,000 people in the United States and Europe and thousands more around the world. Before its gene therapy is used in a clinical trial, Genable will also have to meet good manufacturing practices, or GMP, to ensure that the treatment qualifies for use in humans. The company has partnered with Spark Therapeutics, which is currently conducting a Phase III clinical trial of a gene therapy for Leber congenital amaurosis (LCA) caused by RPE65 mutations. Genable will use Spark’s manufacturing system to produce an adeno-associated virus, or AAV, for gene delivery into the retinal cells. AAVs have performed safely and effectively over the last six years in a number of clinical trials for retinal-disease treatments.
