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Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has expanded its AAV Curator™ Platform offering and License Agreement with the Broad Institute of MIT and Harvard to include the Stanton Lab CNS (Central Nervous System) capsids. Developed by researchers at the Broad Institute, these plasmids produce adeno-associated viruses (AAVs) that increase brain transduction and reduce tropism for the liver and other peripheral organs.
The expanded agreement is an extension of Andelyn’s MyoAAV plasmids License Agreement with Broad Institute announced in August 2024. The Agreement allows Andelyn to use the Stanton Lab CNS capsids to perform research and development services for its clients who are developing gene therapies. Such work includes screening potential candidates at Andelyn and performing scale-up and pre-clinical development work in preparation for IND-enabling studies. The Agreement also permits Andelyn to sublicense these capsids to its clients for internal research purposes.
Incorporating the Stanton Lab CNS capsids into Andelyn’s AAV Curator™ Platform will support the effort to improve gene therapies for nervous system disorders. Data suggests that transduction of the capsids can be as much as six times more effective at targeting most areas of the cerebrum and as much as 13 times more potent in transducing the neuroretina. Increased specificity will reduce off targeting effects as well.
Matt Niloff, Chief Commercial Officer at Andelyn, said,” Expanding our agreement with the Broad Institute to include these novel CNS capsids enables Andelyn to offer critical tools to our clients who are seeking to manufacture gene therapies for neurologic diseases. We’re excited by the potential to provide safer and more effective treatments for rare central nervous system diseases to patients in need of these critical treatments.”
Andelyn is enabling the progression of life-altering gene therapies for both rare and prevalent diseases with the highest quality standards and scalable end-to-end development and manufacturing capabilities at its Columbus, Ohio facilities.
