Genable Technologies Ltd Moving GT038 Into Full Development

Dublin - The Genable team recently attended a Pre-IND meeting with the Food and Drug Administration (FDA) in Washington USA, to discuss the development of GT038 for the treatment of Rhodopsin linked autosomal dominant retinitis pigmentosa (Rho-AdRP). It was a very informative meeting during which the FDA provided feedback and advice on the preclinical and clinical development plans for GT038.

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.


Background Information

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 & Professor Peter Humphries at the Smurfit Institute for Genetics in Trinity College Dublin, Ireland. Genable has received significant financial backing to date from venture capital companies including, Fountain Healthcare Partners, Delta Partners and the patient-led research charities, Fighting Blindness Ireland and Foundation Fighting Blindness Clinical Research Institute.

Genable's approach is innovative and unique, utilising well-established, clinically safe & effective AAV vectors to obtain expression of RNA interference (RNAi) molecules, which suppress the expression of both copies of the disease gene, normal and faulty and a replacement gene which is genetically modified and is refractory to suppression but still encodes a normal wild-type protein. The combination of suppression and replacement overcomes the significant hurdle in dominant disease of mutation variability by eliminating the need to target specific mutations and is applicable to a wide range of disorders

Genable's lead product, GT038, is designed for the treatment of patients with rhodopsin linked autosomal dominant retinitis pigmentosa (Rho-adRP), a debilitating, progressive form of inherited blindness resulting from a diverse array of mutations in the RHO gene. Genable's technology is protected by a broad suite of granted patents and patent applications in the USA, EU and worldwide.

This sub-type of adRP affects approximately 1 in 30,000 people and represents an already identified and potentially treatable population of around 30,000 patients in the US and Europe. Genable will employ the same approach to develop other gene medicines for a number of other sub-types of adRP. The company has received Orphan Drug Designation for GT038 in both the EU and US.

Agreements were executed in 2014 with Spark Therapeutics for the supply of GLP & GMP grade GT038 for the completion of preclinical development as well as the clinical trial programme.

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