Genable Technologies Move Therapy For Rhodopsin Linked Autosomal Dominant Retinitis Pigmentosa (Rho-AdRP) From Research Into Development

Fighting Blindness is delighted to announce that Genable Technologies recently attended a Pre-IND (Investigational New Drug) meeting with the Food and Drug Administration (FDA) in Washington USA, to discuss the development of their gene medicine, GT038, for the treatment of Rhodopsin linked autosomal dominant retinitis pigmentosa (Rho-AdRP).

Dr Jason Loveridge, Genable CEO, commented “Following a very informative and useful meeting with the US medicines regulatory authority, the FDA, we are delighted to confirm that GT038 has now moved from research to development. The FDA’s input, taken together with advice from our Medical Advisory Board and most importantly Genable’s close links with patient groups, increases the probability that we can progress GT038 into the clinic and subsequently provide benefit for RP patients.”

A positive outcome from this Pre-IND meeting represents an important step for Genable as it allows them to gain clarity and seek input from a key regulatory body that will ultimately approve the drug. It is a momentous move from the laboratory stage towards drug development and paves the way for human clinical trials in the near future.

Avril Daly, CEO of Fighting Blindness, commented on Genable’s progress saying “The news that GT0038 has moved from the research to the development phase represents a massive leap forward in the journey towards treatments and cures for inherited retinal disease, particularly for those living with dominantly inherited forms of retinitis pigmentosa. It is also an important example of how patient-led research can bring about innovative developments that will not only improve the quality of life for people affected by conditions with an unmet need but is also a shining example of scientific innovation leading to economic benefit.”


About Genable Technologies Ltd

Genable Technologies Ltd. is a privately held Irish bio-pharmaceutical company developing new gene medicines to treat autosomal dominant genetic diseases based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna and Professor Peter Humphries at the Smurfit Institute for Genetics in Trinity College Dublin. Fighting Blindness has made a substantial investment in Genable Technologies Ltd. Genable’s first gene medicine – GT038 is an adeno-associated viral vector that uses a patented suppression and replacement therapy to ‘suppress’ both copies of the gene (faulty and normal) and ‘replace’ it with a ‘correct’ version of the gene using GT083 will be used to treat patients with rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP), an inherited retinal dystrophy. This sub-type of adRP affects approximately 1 in 30,000 people and leads to blindness in most cases.