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French biotechnology company GenSight receives orphan designation for retinitis pigmentosa gene therapy treatment in Europe.

GenSight Biologics S.A. (Euronext: SIGHT) has announced the receipt of Orphan Drug Designation in Europe for its experimental retinitis pigmentosa (RP) treatment, “GS030”. According to the company, GS030, is an optogenetic therapy delivered using adeno-associated viral (AAV) vectors to introduce the genetic sequence of a photosensitive protein into the nucleus of target cells. Once expressed, target cells should be able to function in a similar manner to photoreceptor cells enabling the restoration of vision in patients who are either blind due to RP or have too few remaining photoreceptor cells to benefit from other potential therapeutic options. As the light sensistivity delivered to such cells is below the capacity of normal photoreceptors, the GS030 treatment additionally employs “biomimetic goggles” which, according to the company, “amplify the light signal at the appropriate wavelength to enable vision restoration”.


According to GenSight, orphan drug designation, granted by the European Commission, provides incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect up to five in 10,000 persons in the European Union (EU). The designation is understood to provide commercial sponsors with financial incentives and benefits in the EU, including a period of market exclusivity if GS030 is approved for the treatment of retinitis pigmentosa patients. In the same announcement, GenSight aslo stated that the EMA have granted “Advanced Therapy Medicinal Product” (ATMP) classification to GS030. Such classifications offer groundbreaking innovative opportunities for the treatment of certain conditions with high unmet needs. The classification provides GenSight with scientific regulatory guidance from the EMA, notably on the applicable regulatory framework and pathway, as well as a centralized approval procedure in Europe.


Commenting on the achievement, Jean-Philippe Combal, Chief Operating Officer of GenSight Biologics stated, “We are very excited that GS030 has received both Orphan Drug Designation and Advanced Therapy Medicinal Product classification in Europe, recognizing the urgent and unmet medical need for a safe and effective treatment for retinitis pigmentosa patients, and the potential of GS030 to address it. This will allow us to optimize future steps to further advance GS030 toward regulatory approval”. In August, 2016, the company raised over €40M on the Euronext exchange following a venture capital fundraising of €36M approximately 12 months prior. Gensight was co-founded by Bernard Gilly, former CEO of both Fovea and Transgene, Jose-Alain Sahel and Serge Picaud, both from the Institut de la Vision at INSERM, Jean Bennett from the University of Pennsylvania, Luk Vandenberghe and Connie Cepko from Harvard Medical School and Botond Roska of the Freidrich Miescher Institute of Biomedical Research.