* NEWSFLASH *
Six-month extension in regulatory review of GenSight's gene therapy for LHON
SECOND CASE OF A PATIENT WHO RECOVERED VISION AFTER TREATMENT WITH gs030 OPTOGENETIC THERAPY
The case of a second patient with late-stage retinitis pigmentosa who experienced visual recovery after treatment with GS030 in the PIONEER trial was presented at the Retina Sub-Specialty Day of the 2021 AAO Annual Meeting.
Click on the video below to see her perform visual tasks that she had not been able to do before GS030 treatment.
GS010 , GENE THERAPY FOR THE TREATMENT OF LEBER HEREDITARY OPTIC NEUROPATHY (LHON)
GS010 targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus).
GenSight Biologics’ lead product candidate, GS010; (lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness.
Our products and pipeline
Clinical trials
GS010; (lenadogene nolparvovec) is completing its clinical development
while GS030 is currently running PIONEER Phase I/II
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