Asa Abeliovich, MD, PhD, Prevail Therapeutics founder and CEO.

Lonza has agreed to manufacture Prevail Therapeutics’ pipeline of novel adeno-associated virus (AAV)-based gene therapies for patients with neurodegenerative diseases at its Lonza Houston Center of Excellence in the Houston suburb of Pearland, TX, the companies said today, through a collaboration whose value was not disclosed.

Prevail and Lonza said they 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. Lonza opened the 300,000-square-foot cell and gene therapy manufacturing facility last year, with the company calling it the largest dedicated cell-and-gene-therapy manufacturing facility in the world, with the ability to address process development from concept through preclinical, clinical, and commercialization phases, to patient dosing.

“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,” Alberto Santagostino, SVP head of cell & gene technologies, Lonza Pharma & Biotech, said in a statement. “The partnership combines Prevail’s frontier science with Lonza’s operational expertise to support the development and commercialization of potentially life-changing treatments.

Baculovirus/Sf9 is one of two expression systems upon which Prevail has built in-house process and analytical development capabilities; the other is HEK293. In the baculovirus production system, AAV vectors are produced by infection of insect cells with recombinant baculoviruses.

“This scalable suspension production system, using single-use bioreactors, is designed to produce higher yields of vectors more cost-effectively and efficiently than current mammalian cell-based approaches,” Prevail stated in its Prospectus for its initial public offering (IPO), which raised $113.2 million in aggregate net proceeds in June. “We plan to have the baculovirus-based process developed and producing cGMP-compliant batches in time to support our pivotal clinical trials for each of our programs.

“Cost-efficient, safe, and scalable”

“We believe the baculovirus production system will maximize our ability to ensure cost-efficient, safe, and scalable supply at the higher quantities required for late-stage clinical development and commercialization,” Prevail added.

The companies are building on a year-old partnership that was focused initially on process development, with the goal of achieving the GMP manufacturing of Prevail’s two lead programs, PR001 and PR006.

PR001 is an AAV9-based gene therapy designed to deliver GBA1, and is being developed for Parkinson’s disease patients with GBA1 mutations (PD-GBA) as well as neuronopathic Gaucher disease patients (nGD). Prevail said it plans to launch a Phase I/II clinical trial of PR001 in PD-GBA later this year.

Prevail 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 begin clinical trials in 2020.

Both gene therapy candidates apply REGENXBIO’s Novel AAV Vector (NAV) AAV9 vector technology platform, which consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. In 2009 REGENXBIO exclusively licensed key intellectual property covering novel recombinant AAVs discovered in the lab of James M. Wilson, MD, PhD, director of the gene therapy program at the University of Pennsylvania.

Prevail’s gene therapies aim to restore healthy lysosomal function in the cells of a patient’s central nervous system, in order to slow or stop the progression of neurodegenerative disease.

“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,” stated Asa Abeliovich, MD, PhD, Prevail’s founder and CEO.

“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 Abeliovich, who partnered with the Silverstein Foundation for Parkinson’s with GBA and investment firm OrbiMed to launch Prevail in 2017. The company is headquartered in New York at the Alexandria Center for Life Science–New York City.

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