ABL, Inc. (ABL), a global contract research and manufacturing organization to the biopharmaceutical industry, announced agreements with Regenxbio, Inc., a leading biotechnology company, to manufacture its lead product, RGX-501, for human clinical trials. RGX-501 is a novel therapeutic for the treatment of homozygous familial hypercholesterolemia (HoFH), which uses the NAV rAAV8 vector to deliver the human low-density lipoprotein receptor (LDLR) gene to liver cells.

Under the agreements, ABL will continue process development work and initiate the transfer and scale-up of the RGX-501 manufacturing process at its Rockville, MD cGMP production facility. The site is designed as a multi-product facility utilizing single-use, disposable technologies for the development and production of gene therapies, vaccines, protein immunotherapeutics, and other biologics-based products for use in human clinical trials.

“We are delighted to be supporting Regenxbio and the RGX-501 programme,” said Thomas VanCott, Ph.D., president and CEO of ABL, Inc. “AAV-based vectors have demonstrated remarkable promise in addressing some of the most challenging human diseases, and Regenxbio is at the forefront of applying the next generation of AAV vectors through its NAV Technology Platform.  ABL has an established track record of cGMP virus vector manufacturing, and we are very excited to be applying our bulk production and fill/finish expertise to the RGX-501 programme.”

Regenxbio, Inc., a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy based on its proprietary NAV Technology Platform.