- Regenxbio has agreed to grant Pfizer a non-exclusive license to its AAV9 vector technology for use in the pharma's gene therapy program targeting the neurodegenerative movement disorder Friedreich's ataxia.
- The companies did not disclose financial specifics of the deal. Regenxbio will get an upfront payment and potential fees, milestone payments and royalties; Pfizer also receives sublicensing rights as part of the agreement.
- "This license agreement further validates the strength of our intellectual property portfolio and the potential of NAV AAV9 for the treatment of systemic and CNS manifestations of movement disorders," Regenxbio CEO Kenneth Mills said in a company statement.
Pfizer might not be the first drugmaker associated with gene therapies, yet the pharma has steadily advanced research stemming from a 2016 acquisition of Bamboo Therapeutics.
Securing access to viral vectors, the technology used to deliver functional genes, is an important part of that push.
"We believe the AAV9 vector has the potential to have a profound impact on patients with severe and debilitating diseases where treatment options are limited today,” Seng Cheng, chief scientific officer of Pfizer's rare disease research unit, said in a statement.
The Friedreich's ataxia program is one of Pfizer's earlier-stage gene therapy efforts. The company is further along with treatments for Duchenne muscular dystrophy and for hemophilia A, which is part of a collaboration with Sangamo Therapeutics.
Rockville, Maryland-based Regenxbio both develops its own therapies and licenses out its viral vector technology, discovered by one of the company's founders, James Wilson, while working in a lab at the University of Pennsylvania.
The company says it has rights to more than 100 patents and patent applications covering more than 100 adeno-associated virus (AAV) vectors.
More than 20 therapies are under development at other companies using the company's so-called NAV platform, according to analysts at Raymond James.
Regenxbio, for example, receives royalties on sales of Novartis's Zolgensma (onasemnogene abeparvovec), a gene therapy for a rare inherited neuromuscular condition in children priced at a record $2.1 million per patient.
Friedreich's ataxia is a hereditary condition in which a genetic mutation causes limited production of a protein known as frataxin. Patients suffer from symptoms ranging from muscle weakness and loss of balance to impaired vision, hearing and speech.
The potentially life-shortening disease affects about 1 in 50,000 people in the U.S., according to the Friedreich's Ataxia Research Alliance.