REGENXBIO Expands Pipeline Using NAV Vectors to Deliver Therapeutic Antibodies for the Treatment of Hereditary Angioedema and Neurodegenerative Diseases

Thursday, July 25, 2019

REGENXBIO Inc., a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced an expansion of its pipeline to include novel treatments for hereditary angioedema (HAE) and neurodegenerative diseases, including tauopathies, using NAV Vectors to deliver therapeutic antibodies.

REGENXBIO's new gene therapy product candidate for the treatment of HAE utilizes NAV Vectors that are designed to deliver a gene encoding a therapeutic antibody that targets and binds to plasma kallikrein, a key protein left unregulated in patients with HAE.

In addition, REGENXBIO and Neurimmune AG announced earlier today an exclusive license, development and commercialization agreement to discover and develop novel AAV gene therapies using NAV Vectors to deliver human antibodies against targets implicated in chronic neurodegenerative diseases, including tauopathies.

"Using NAV Vectors to deliver therapeutic antibodies has enormous potential for patients who lack treatments or who are currently underserved by existing therapies, and provides a significant opportunity to expand our pipeline through the application of our AAV-mediated antibody delivery capabilities and expertise to a number of validated and new targets in multiple therapeutic areas and tissues," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We are thrilled to announce our research program for the treatment of HAE, a chronic, life-threatening disease characterized by recurring severe swelling, most commonly in the face, airway, intestines and limbs, for which a one-time AAV-mediated antibody delivery approach may provide significant benefit for patients. In addition, we are excited to partner with Neurimmune and we believe the combination of Neurimmune's human-derived antibodies with REGENXBIO's AAV expertise creates a unique opportunity to develop significant therapies for chronic neurodegenerative diseases."

"REGENXBIO is at the forefront of research and development of AAV-mediated antibody gene therapies," said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. "Our pipeline expansion through our research for the treatment of HAE and our previously announced partnership with Neurimmune builds on promising results with our clinical-stage RGX-314 program, where we are using our NAV Vectors to deliver a therapeutic antibody for the treatment of wet AMD."