Technology

Genable is developing unique gene-based therapies for the treatment of an entire class of incurable genetic disorders.

Genable's technology consists of a suppression and replacement approach for inherited disorders using Ribonucleic Acid Interference (RNAi) as the suppression tool in concert with an altered replacement gene protected from RNAi attack (Millington-Ward et al Hum Mol Genet. 1997 Sep;6(9):1415-26).

This simple combination eliminates the need to target specific mutations in a wide range of disorders while presenting an effective strategy to overcome dominant negative pathology. The simultaneous delivery of a replacement gene is protected from RNAi attack through alteration of the nucleotide sequence while maintaining the normal amino acid sequence.

Products

Name	Phase	Indication	Milestone
GT015	Pre-clinical	Retinitis pigmentosa	Ongoing
GT025	Pre-clinical	Retinitis pigmentosa	Ongoing
GT012	Pre-clinical	Osteogenesis imperfecta	Ongoing