Prevail Signs an Agreement with Lonza to Develop and Manufacture AAV9 Gene Therapy Programs for Patients with Neurodegenerative Diseases

Shots:

• Pevail and Lonza to work collaboratively for process and analytical development plus large-scale production using the baculovirus/Sf9 process for late-stage clinical and commercial supply at Lonza’s GMP facility in Houston. Lonza to manufacture Prevail’s pipeline of novel AAV-based gene therapy programs for neurodegenerative diseases at its gene therapy center of excellence in Houston, TX
• The companies will work on process development and scaling up production of Prevail’s PR001 candidate targeted for Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease plus will also support PR006 gene therapy programs for patients with frontotemporal dementia with GRN mutations (FTD-GRN)
• PR001 is an AAV9-based gene therapy delivering GBA1, targeted for PD-GBA and neuronopathic Gaucher disease (nGD) with the onset of its P-I/II trial in PD-GBA in 2019. PR006 is an AAV9-based gene therapy delivering GRN for patents with FTD-GRN and will enter the clinic in 2020

Click here to­ read full press release/ article | Ref: Lonza | Image: Twitter