Exhibit 99.1
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Prevail Therapeutics and Lonza Establish Strategic Collaboration to Develop and Manufacture Prevail’s Novel AAV9 Gene Therapy Programs for Patients with Neurodegenerative Diseases
Strategic collaboration focuses on baculovirus/Sf9 expression system for Prevail’s pipeline of novel AAV9-based gene therapy programs for patients with neurodegenerative diseases
Companies to partner on process development, analytical development, and large-scale manufacturing for late-stage clinical and commercial supply
Process development and manufacturing to be located in Lonza’s cGMP facility in Houston, Texas
Basel, Switzerland and New York, USA, October 8, 2019– Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifyingAAV-based gene therapies for patients with neurodegenerative disorders, and Lonza (SWX: LONN) today announced that the companies have entered into a strategic collaboration.
Lonza and Prevail have been working together since 2018, with an initial focus on process development, towards the GMP manufacturing of Prevail’s two lead programs, PR001 and PR006. Under this collaboration, focused on the baculovirus/Sf9 production system for gene therapies, Lonza will manufacture Prevail’s pipeline of novelAAV-based gene therapy programs for patients with neurodegenerative diseases at its gene therapy center of excellence in Houston, TX.
“Prevail is a brilliant trailblazer and it is a privilege to partner with them,” said Alberto Santagostino, SVP Head of Cell & Gene Technologies, Lonza Pharma & Biotech. “This strategic collaboration reflects both companies’ commitment to fueling the innovation of novel medicines. The partnership combines Prevail’s frontier science with Lonza’s operational expertise to support the development and commercialization of potentially life-changing treatments. Our cell and gene therapy center of excellence in Houston, TX, will support Prevail in their journey as they develop and potentially bring to marketAAV-based gene therapies for patients suffering from debilitating neurodegenerative diseases.”
Prevail’s pipeline includes PR001, an AAV9-based gene therapy deliveringGBA1, in development for Parkinson’s disease patients withGBA1 mutations(PD-GBA) and neuronopathic Gaucher disease patients (nGD). Prevail plans to initiate a Phase 1/2 clinical trial of PR001 inPD-GBA in 2019. The company is also developing PR006, an AAV9-based gene therapy deliveringGRN, for frontotemporal dementia patients with aGRN mutation(FTD-GRN). Prevail anticipates PR006 will enter the clinic in 2020. The collaboration also has the potential to extend to Prevail’s future pipeline ofAAV-based gene therapy programs.
“We are pleased to partner with Lonza, a leader in the manufacturing of AAV gene therapy vectors, to enable large-scale production of our novel gene therapies that we believe hold potential to halt the course of neurodegenerative diseases for patient populations with urgent unmet needs,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer, Prevail Therapeutics. “Lonza