Axxam S.p.A. collaborates with GenSight Biologics S.A. to support Clinical Trials of an optogenetic-based gene therapy drug for sight-threatening diseases

April 24th 2018 – Milan (Italy) – Axxam S.p.A. announced today a collaboration with GenSight Biologics S.A. in the field of optogenetic gene-therapy drug development, with the goal to support GenSight effort of establishing innovative therapies to preserve or restore vision in patients suffering from sight-threatening ophthalmic diseases, such as Retinitis Pigmentosa.

GenSight is an innovative clinical-stage gene therapy company with an initial focus on discovering, developing and commercializing novel therapies for severe retinal neurodegenerative diseases.

Its unique know-how in using optically controlled gene products for orchestrating cellular functions in miniaturized HTS-compatible functional assays, made Axxam the research partner of choice for the development of a cell-based potency assay to be used to validate GenSight GS030 drug product batches for the use in clinical-stage experimentation.

Axxam started the collaboration with GenSight with the establishment of a miniaturized in-vitro assay aimed at robustly and quantitatively measuring the biological effects linked to GS030 therapeutic properties, namely photoactivated cellular excitability. The potency assay, specially developed by Axxam for GenSight, is now ready to process for the method qualification in order to meet the regulatory requirements for clinical and further commercial stages.

Open PDF document