Under the terms of the agreement, Pfizer will be able to use REGENXBIO’s proprietary NAV adeno-associated virus (AAV) system to deliver genes that could help resolve the underlying faulty mechanisms that cause the progressive nerve and muscle disease.
“We are excited to partner with REGENXBIO on the use of the NAV AAV9 vector for the treatment of Friedreich’s ataxia, a condition with significant unmet medical need,” Seng Cheng, PhD, senior vice president and chief scientific officer of Pfizer’s rare disease research unit, said in a press release.
“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,” he added.
Friedreich’s ataxia affects about one in 50,000 people in the U.S., and has no available cure.
Researchers have tried to develop gene therapies that could reverse the genetic deficiency that characterizes this disease. At issue is the expansion of the FXN gene, and consequent impaired production of functional frataxin protein.
However, to date, most of the attempted strategies have failed. In the majority of cases, this has been due to the poor efficacy of the delivery of the target gene. Scientists say improved delivery systems are necessary to achieve the production of an effective gene therapy for Friedreich’s ataxia.
“We applaud these efforts to develop new potential NAV Technology-based gene therapies for our under-served patient community,” said Ronald J. Bartek, president, director, and co-founder of the Friedreich’s Ataxia Research Alliance.
REGENXBIO’s NAV technology platform comprises more than 100 new AAV vectors that have been used to develop several investigational candidates for the treatment of disease in multiple therapeutic areas.
In return for the rights granted to Pfizer, REGENXBIO will receive an upfront payment, and has the potential to receive additional fees and development payments. The company also may receive future commercial milestone payments, and royalties on sales after regulatory approval of products that use the licensed NAV AAV technology.
“This license agreement further validates the strength of our intellectual property portfolio and the potential of NAV AAV9 for the treatment of systemic and central nervous system manifestations of movement disorders,” said Kenneth T. Mills, president and CEO of REGENXBIO.