Contract win sees Lonza develop and manufacture gene therapies for Prevail

By Murielle Gonzalez 9-Oct-2019

New York-based Prevail Therapeutics has chosen the Swiss CDMO for GMP manufacturing of the AAV9-based gene therapy for patients with neurodegenerative diseases

Lonza Houston site

Lonza has entered into a strategic collaboration with US-based biopharmaceutical Prevail Therapeutics to develop and manufacture its novel AAV9 gene therapy for patients with neurodegenerative diseases.

Headquartered in New York, Prevail has been working with the Swiss CDMO since 2018, with an initial focus on process development to GMP manufacturing of Prevail’s two lead programmes, PR001 and PR006.

Under this collaboration, focused on the baculovirus/Sf9 production system for gene therapies, Lonza will manufacture Prevail’s pipeline of novel AAV-based gene therapy for patients with neurodegenerative diseases. Lonza will carry out this work at its gene therapy centre of excellence in Houston, Texas. The site entered operation in April 2018, as shown in the video below.

Gene therapy pipeline

Prevail’s pipeline includes PR001, an AAV9-based gene therapy delivering GBA1, in development for Parkinson's disease patients with GBA1 mutations (PD-GBA), and neuronopathic Gaucher disease patients (nGD). The biopharma company plans to initiate a Phase 1/2 clinical trial of PR001 in PD-GBA in 2019.

The company is also developing PR006, an AAV9-based gene therapy delivering GRN, for frontotemporal dementia patients with a GRN 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 of AAV-based gene therapy programs.

Prevail has built in-house process and analytical development capabilities utilising both HEK293 and baculovirus/Sf9 AAV expression systems.

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Under this 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.