Prevail Therapeutics Inc., a biotechnology company focused upon developing novel gene therapies for treatment of neurodegenerative diseases recently announced that the U.S. FDA has approved of the company’s application for trial of PR001, company’s IND (Investigational New Drug) for the treatment of nGD (Neuronopathic Gaucher disease). The FDA has shown a green signal to Prevail Therapeutics for initiating its proposed clinical trial of the IND, which had been previously put on hold. Prevail Therapeutics’ planned Phase 1/2 clinical trial for patients with Neuronopathic Gaucher disease will begin at a dose bit higher than what was originally proposed. The biotechnology company submitted nonclinical data in which no PR001-related adverse findings or safety events were observed, which supports the initiation of the Phase 1/2 clinical trial at a higher dose. Type 2 Gaucher disease is the more severe form of Neuronopathic Gaucher disease, which occurs in infants and involves rapidly progressing neurodegeneration, which generally leads to death in infancy or in early childhood. The company will be initiating a Phase 1/2 clinical trial for patients with Type 2 Gaucher disease and is looking forward to initiate patient dosing over the first half of 2020. Prevail is also planning to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients by the second half of 2020, with the same name nGD investigational drug. Type 3 Gaucher disease is a form of Neuronopathic Gaucher disease that typically occurs in childhood and involves multiple neurological manifestations. Asa Abeliovich, Founder & CEO of Prevail Therapeutics, Inc., was reportedly quoted saying that the company is pleased to receive an active IND for PR001 and is looking forward to initiating clinical trial of the drug in the first half of 2020,” Abeliovich added saying that PR001 has tremendous potential to ameliorate the suffering of patients suffering from this devastating disease. Source Credits: https://ir.prevailtherapeutics.com/news-releases/news-release-details/prevail-therapeutics-announces-ind-active-pr001-treatment