Overview

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. Recent developments have reinforced the likelihood that gene therapy will be used as 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, which made Glybera the first gene therapy product approved in the Western world. In late 2017, Luxturna (developed by Spark Therapeutics) became the first approved gene therapy in the US.
  • Guidance from regulatory agencies
  • Investment in gene therapy by the pharmaceutical industry

The technology

Using our gene-therapy based approach, our product candidates are designed to be administered in a single treatment to each eye by intravitreal, or IVT, injection and provide patients with a long-lasting functional cure, potentially for the rest of their lives.

Our technologies use Adeno-Associated Virus (AAV) vectors for the treatment of retinal diseases. AAV vectors are among 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 of transferring genes into cells; the resulting protein expression appears sustainable over time.

Product applications

  • Our GS010 product candidate uses an AAV2 vector, containing the human mitochondrial ND4 gene, combined with our proprietary MTS technology
  • Our GS030 product candidate includes a gene encoding a photoactivatable channelrhodopsin, delivered via a modified AAV vector, known as AAV2 7m8