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

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