Regenxbio to Develop Antibody-based Gene Therapy for Hereditary Angioedema

Regenxbio is exploring a new hereditary angioedema (HAE) treatment that harnesses viral vectors to deliver an antibody-based gene therapy, the company announced.

The treatment will deliver the genetic sequence for an antibody that targets plasma kallikrein, a protein involved in swelling attacks in HAE patients, preventing it from exceeding healthy levels.

The therapy uses a safe adeno-associated virus (AAV) to take the genetic information into the nucleus of target cells. Compared with earlier AAVs, the newer AAV viruses (NAV vectors) are thought to transport genes more effectively and provide greater and longer gene expression. This is thus believed to be a one-time treatment that provides significant, long-lasting benefits.

“We are thrilled to announce our research program for the treatment of HAE, a chronic, life-threatening disease for which a one-time AAV-mediated antibody delivery approach may provide significant benefit for patients,” Kenneth T. Mills, president and CEO of Regenxbio, said in a press release.

Regenxbio is also using its proprietary NAV Technology platform to deliver therapeutic antibodies in neurodegenerative diseases, including Alzheimer’s disease. This is part of a collaboration and marketing agreement with Neurimmune.

The two companies will be jointly responsible for the design and development of vectorized antibody therapies. After a research and discovery phase, each company will have the option to continue the collaborative developmental work.

“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,” Mills said. “Using NAV vectors to deliver therapeutic antibodies has enormous potential for patients who lack treatments or who are currently underserved by existing therapies.”

In addition to hereditary angioedema and neurodegenerative diseases, the company is also conducting preclinical tests in diabetic retinopathy, and has four gene therapies already in clinical testing, including RGX-314 for wet age-related macular degeneration, RGX-121 for mucopolysaccharidosis (MPS) type II, RGX-111 for MPS type 1, and RGX-501 for homozygous familial hypercholesterolemia.

“Regenxbio is at the forefront of research and development of AAV-mediated antibody gene therapies,” said Olivier Danos, PhD, chief scientific officer of Regenxbio. “Our pipeline expansion through our research for the treatment of HAE and our … 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 age-related macular degeneration.”