Prevail and Lonza Deepen Partnership with New Collaboration

Working Together

Prevail Therapeutics and Switzerland-based Lonza entered into a strategic collaboration focused on the development of the baculovirus/Sf9 expression system for Prevail's pipeline of gene therapy programs aimed at neurodegenerative diseases.

The two companies will partner on the process development, analytical development and large-scale manufacturing for late-stage clinical and commercial supply. The process development and manufacturing of the gene therapy programs under the baculovirus/Sf9 expression system will be located in Lonza's cGMP facility in Houston. The latest collaboration builds on the existing relationship the two companies established in 2018. The companies initially joined together to focus on process development for Prevail’s two lead programs, PR001 and PR006, gene therapies for Parkinson’s disease and dementia, respectively. Financial terms of the updated partnership were not disclosed.

PR001 is an AAV9-based gene therapy is in development for Parkinson’s disease patients with GBA1 mutations and neuronopathic Gaucher disease patients. Prevail plans to initiate a Phase I/II clinical trial of PR001 in Parkinson’s disease later this year after the U.S. Food and Drug Administration approved its Investigational New Drug Application in June. PR006 is also an AAV9-based gene therapy for frontotemporal dementia patients with a GRN mutation. 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.

Alberto Santagostino, head of Cell & Gene Technologies at Lonza Pharma & Biotech, called Prevail a “brilliant trailblazer” and called it a privilege to work with the company.

“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,” Santagostino said in a statement.

He added that Lonza’s cell and gene therapy center in Houston will support Prevail in its efforts to develop AAV-based gene therapies for these different indications.

Asa Abeliovich, founder and chief executive officer of Prevail, touted Lonza’s expertise as a leader in the manufacture of AAV gene therapy. Abeliovich said that expertise will enable large-scale production of his company’s novel gene therapies. He said Prevail believes its candidates have the 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,” Abeliovich said in a statement.

Prevail, which launched in 2017, has built in-house process and analytical development capabilities utilizing both HEK293 and baculovirus/Sf9 AAV expression systems.

In March, Prevail secured $50 million in a Series B financing round to advance development of Prevail’s pipeline of novel gene therapy programs. The year prior, Prevail grabbed $75 million in a Series A financing round.

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