Andelyn Biosciences Expands AAV Curator™ Platform with Broad Institute's MyoAAV Plasmids

Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has entered into a License Agreement with the Broad Institute to expand its AAV Curator™ Platform offering to include MyoAAV plasmids. Developed by researchers at the Broad Institute of MIT and Harvard, this new family of plasmids produce adeno-associated viruses (AAVs) that are ten times more efficient in reaching muscle tissue than wild-type vectors, while also de-targeting the liver. With more precise targeting of muscle tissue, research shows MyoAAV-delivered therapies may require lower dosing.

The License Agreement allows Andelyn to use the MyoAAV plasmids to perform research and development services for its clients who are developing gene therapies to screen potential candidates at Andelyn and to perform scale-up and pre-clinical development work in preparation for IND-enabling studies. The Agreement also permits Andelyn to sublicense the MyoAAV plasmids to its clients for internal research purposes.

Andelyn's expertise in translational drug development will bring Broad's new class of engineered AAVs to biotech organizations targeting diseases in these therapeutic areas where there is still a significant unmet need. This capability will ultimately reduce the effective dose demand and improve product safety. Incorporating MyoAAVs into Andelyn’s AAV Curator™ Platform will drive sustainable manufacturing practices and continue to bring more accessible treatments to patients.

Matt Niloff, Chief Commercial Officer of Andelyn, said, ”Our license from the Broad Institute furthers our goal of providing the industry access to critical tools and capabilities that facilitate the development of innovative therapies to bring more treatments to more patients. With access to the increased specificity of MyoAAVs, our clients now have the opportunity to maximize efficiency in their gene therapy processes and drive down the cost per patient.”

With the highest quality standards and scalable end-to-end development and manufacturing capabilities across its Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases