Genable Technologies – a gene based medicines platform
Genable Technologies Ltd. is a privately held Irish bio-pharmaceutical company developing new gene medicines to treat aotosomal dominant genetic diseases based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna & Professor Peter Humphries. Genable has received significant financial backing to date from Fountain Healthcare Partners, Delta Partners, Fighting Blindness Ireland and Foundation Fighting Blindness Clinical Research Institute.
Genable's approach is innovative and unique, it utilises well-established, clinically safe & effective AAV vectors to obtain expression of RNA interference (RNAi) molecules which suppress the expression of faulty genes and replaces this a gene that 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 technology is protected by a broad suite of granted patents and patent applications in the USA, EU and worldwide.
Genable's lead product, GT038, is designed for the treatment of patients with rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP) - a debilitating, progressive form of inherited blindness resulting from a diverse array of mutations in the RHO gene.
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.
Genable has successfully completed proof of concept for GT038 in a number of animal models. Doses, volumes and suppression and replacement composition for the final GT038 formulation have been determined in a large animal model in preparation for the regulatory, biodistribution and toxicology programme.
Supply agreements have been executed with Spark Therapuetics for the supply of GLP & GMP grade GT038 for the completion of preclinical development as well as the commencement of clinical trials.