Lonza, Prevail Therapeutics Partner on Novel AAV9 Gene Therapy Programs

Published on: 

The collaboration focuses on baculovirus/Sf9 expression system for Prevail's pipeline of novel AAV9-based gene therapy programs neurodegenerative diseases.

Lonza and Prevail Therapeutics, a biotechnology company specializing in AAV-based gene therapies for neurodegenerative disorders, announced on Oct. 8, 2019 that they have entered into a strategic collaboration to develop and manufacture Prevail's novel adeno-associated virus 9 (AAV9) gene therapy programs for patients with neurodegenerative diseases.

The two companies 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, the focus will be on the baculovirus/Sf9 production system for gene therapies. Lonza will manufacture Prevail's AAV-based gene therapy pipeline at its gene therapy center of excellence in Houston, TX.

PR001, an AAV9-based gene therapy delivering the GBA1 gene, is in development for Parkinson’s disease in patients with GBA1 mutations (PD-GBA) and in patients with neuronopathic Gaucher disease (nGD). Prevail plans to initiate a Phase I/II clinical trial of PR001 in PD-GBA in 2019. The company is also developing PR006, an AAV9-based gene therapy delivering the GRN gene, for patients with frontotemporal dementia s with a GRN mutation (FTD-GRN). Prevail anticipates PR006 will enter the clinic in 2020. The collaboration with Lonza also has the potential to extend to Prevail's future pipeline of AAV-based gene therapy programs.

Prevail has built in-house process and analytical development capabilities utilizing both HEK293 and baculovirus/Sf9 AAV expression systems. Under the collaboration, Prevail and Lonza will work closely together on process development, analytical development, and large-scale production using the baculovirus/Sf9 process for late-stage clinical and commercial supply at Lonza’s GMP facility in Houston.


"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 market AAV-based gene therapies for patients suffering from debilitating neurodegenerative diseases," said Alberto Santagostino, senior vice-president head of Cell & Gene Technologies, Lonza Pharma & Biotech, in a company press release.

"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. Lonza and Prevail will work together closely on process development and scaling up production of PR001, our gene therapy for Parkinson’s disease with GBA1 mutations and neuronopathic Gaucher disease, to supply late-stage clinical trials and for commercial production. In addition, we look forward to a collaborative relationship to support our PR006 gene therapy program for patients with frontotemporal dementia with GRN mutations, and for future gene therapy programs in our pipeline," added Asa Abeliovich, MD, PhD, founder and CEO, Prevail Therapeutics, in the press release.

Source: Lonza