GNT-0003 is under clinical development by Genethon and currently in Phase II for Crigler-Najjar Syndrome. According to GlobalData, Phase II drugs for Crigler-Najjar Syndrome does not have sufficient historical data to build an indication benchmark PTSR for Phase II. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the GNT-0003 LoA Report. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
GNT-0003 overview
GNT-0003 is under development for the treatment of crigler-najjar syndrome. The drug candidate is administered through intravenous route. Direct administration of an AAV (adeno-associated virus) vector carrying a normal copy of UGT1A1.
Genethon overview
Genethon, a subsidiary of The French Muscular Dystrophy Association, is a non-profit organization that designs preclinical and clinical development of gene therapy drugs for rare genetic diseases. The organization’s pipeline products include GNT 0004, GNT 0006, GNT 0007, GNT 0008, Zolgensma and Lumevoq. Its gene therapy drugs used for treating Duchenne muscular dystrophy, infant spinal muscular atrophy, SMA-PME (spinal muscular atrophy with progressive myoclonic epilepsy), Farber’s disease, Leber hereditary optic neuropathy, myotubular myopathy, limb-girdle muscular dystrophies, wiskott-aldrich syndrome, Fanconi’s anemia, sickle cell disease, chronic septic granulomatosis, Crigler-Najjar syndrome, Pompe disease, cori Forbes disease and glycogen storage disease type 1a (GSD1a). The organization works in partnership with Hansa Biopharma, Avexis, Samabriva and Sanofi, among others. Genethon is headquartered in Ile-de-France, Paris, France.
For a complete picture of GNT-0003’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
