Prevail and Lonza Join Forces to Develop PR001 Gene Therapy for Gaucher Type 2

Prevail and Lonza Join Forces to Develop PR001 Gene Therapy for Gaucher Type 2

Prevail Therapeutics and Lonza Pharma & Biotech will collaborate in the development and production of Prevail’s PR001 gene therapy for children with type 2 Gaucher disease and for select patients with Parkinson’s disease, the companies announced.

Prevail is looking to start recruiting patients with type 2, or pediatric neuronopathic, Gaucher for a Phase 1/2 trial of PR001 in the first half of 2020. The company aims to test the therapy at higher doses than initially planned, and is waiting on a decision by the U.S. Food and Drug Administration (FDA) before starting the trial.

PR001 uses a modified, harmless version of an adeno-associated virus (AAV9) to deliver a fully working version of the GBA1 gene to nerve cells. Mutations in this gene cause Gaucher disease and also lead to Parkinson’s, as they result in a defective enzyme called beta-glucocerebrosidase and subsequent accumulation of fatty molecules inside cells. These toxic fatty molecules build up in the brain of patients with type 2 Gaucher disease from early infancy, leading to neurological symptoms.

The gene therapy is expected to restore production of normal beta-glucocerebrosidase in affected nerve cells. A single administration of PR001 is believed to have the potential to ease symptoms and modify disease course with long-lasting effect.

Studies in mice and monkeys indicated that PR001 is well-tolerated and can effectively lead to the production of a functional enzyme in nerve cells, reducing the accumulation of fatty molecules and improving motor function.

The FDA has accepted Prevail’s investigational new drug application to start a Phase 1/2 trial of PR001 in up to 16 Parkinson’s patients with a GBA mutation. Dosing is expected to start this year. PR001 has been granted FDA’s fast-track designation as a treatment for this indication.

Prevail is also developing PR006, an AAV9-based gene therapy that aims to deliver a correct version of the GRN gene to people with frontotemporal dementia who carry GRN mutations. The company anticipates clinical studies of PR006 to start in 2020.

Prevail and Lonza have been working together since 2018. Under the newly established collaboration, Lonza will manufacture PR001 and PR006 at a large scale, and possibly other future gene therapies.

“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,” Alberto Santagostino, senior vice president and head of cell and gene technologies at Lonza, said in a news release.

Asa Abeliovich, MD, PhD, Prevail’s founder and CEO, added: “We are pleased to partner with Lonza, a leader in the manufacturing of AAV gene therapy vectors, to enable large-scale production of our novel gene therapies that we believe hold potential to halt the course of neurodegenerative diseases for patient populations with urgent unmet needs.”

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