Gene therapy involves the insertion of genes into a cell's genetic code to either replace defective genes that cause disease (e.g. because they fail to produce a functional protein) or to produce therapeutic proteins locally. Gene therapy offers the possibility to administer the treatment once, or a limited number of times, to achieve a long-term, durable benefit and potentially a cure.
In the last two decades, gene therapy has become a powerful, viable and safe treatment modality to address disease biology in a targeted and efficient way. The following recent elements have reinforced the likelihood that gene therapy will be an effective therapeutic approach to address unmet medical needs:
- Clinical data: Positive data from gene therapy have been reported in a variety of indications including several ophthalmic diseases such as Leber Congenital Amaurosis, or LCA, and wet-AMD.
- Product approval: In 2012, the European Medical Agency granted approval for Glybera (developed by uniQure N.V.) for the treatment of lipoprotein lipase deficiency. Glybera is the first gene therapy product approved in the Western world
- Guidance from regulatory agencies
- Pharmaceutical industry investment in gene therapy
We have chosen to focus first on severe retinal diseases, with the goal of preserving or restoring vision in patients suffering from sight-threatening ophthalmic diseases. Using our gene-therapy based approach, our product candidates are designed to be administered only once in order to provide patients with a long-lasting functional cure, potentially for the rest of their lives.
Adeno-Associated Virus (AAV)
Our technologies use AAV vectors for the treatment of retinal diseases. AAV vectors are the safest, most studied, and most widely used vectors in the field of gene therapy. The use of AAV vectors in the retina has proven to be an efficient way to transfer genes into cells and the resulting protein expression appears sustainable over the long term.
- Our GS010 product candidate uses an AAV2, containing the human wild-type mitochondrial ND4 gene, combined with our proprietary MTS technology.
- Our GS030 product candidate comprises a gene encoding a photoactivatable channelrhodopsin, delivered via a modified, known as AAV2 7m8.