Gene Therapy

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 the first approval of gene therapy product in the Western world. In late 2017, the solution developed by Spark Therapeutics US became the first approved gene therapy in the US.

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.

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.