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