Lenadogene nolparvovec is under clinical development by GenSight Biologics and currently in the Phase III in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.
According to GlobalData, the latest event to affect Lenadogene nolparvovec’s likelihood of approval (LoA) and phase transition for Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) took place on 29 Jul 2022, which decreased the likelihood that the drug progresses to the next phase in its clinical pathway and decreased the likelihood of final approval for this indication.
GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Lenadogene nolparvovec Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.
Lenadogene nolparvovec overview
lenadogene nolparvovec (GS-010) is under development for the treatment of Leber hereditary optic neuropathy due to mutation of ND4 gene. It is administered through intravitreal route. lenadogene nolparvovec (GS-010) is a recombinant Adeno-associated virus vector serotype 2 (AAV2) containing the human wild-type mitochondrial ND4 gene (rAAV2-ND4 vector). It is based on MTS technology platform, that enables efficient expression of a mitochondrial gene by nuclear deoxyribonucleic acid, or DNA, and delivery of messenger ribonucleic acid, or mRNA, to polysomes located at the mitochondrial surface.
GenSight Biologics overview
GenSight Biologics (GenSight) operates as a clinical stage biotechnology company. It discovers and develops novel therapies for mitochondrial and neurodegenerative diseases. The company’s product pipeline comprises GS010 and GS030 developed for the treatment of retinal degenerative diseases. It develops solutions for preserving or restoring vision in patients suffering from very low vision or blindness due to degenerative retinal and inherited ophthalmologic diseases. GenSight’s drugs target against gene replacement therapy for vision loss in leber hereditary optic neuropathy. It also develops products for retinitis pigmentosa and other hereditary diseases of retina. GenSight is headquartered in Paris, France.
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