Andelyn licenses Broad Institute plasmids to bolster gene-therapy research

Contract development and manufacturing organization (CDMO) Andelyn Biosciences has licensed MyoAAV plasmids from the Broad Institute. The Ohio-based cell and gene therapy (CGT) company will use them with its AAV Curator platform at its Columbus, Ohio facility. The agreement grants Andelyn’s clients access to MyoAAV plasmids for internal research purposes.

MyoAAV plasmids encode a new subgroup of adeno-associated viruses (AAVs) – the gene-delivering vectors of gene therapy – that improve targeting of the muscle tissue, which could be safer and more effective for patients with muscle diseases.

According to the firm, MyoAAV plasmids are at least ten times more efficient than other viral vectors, enabling them to deliver therapeutic genes at around 100 to 250 times lower doses than those used in other studies and trials, potentially reducing the risk of liver damage and other serious side effects.

“It is our goal to provide the industry access to critical tools and capabilities that facilitate the development of innovative therapies to bring more treatments to more patients,” Matt Niloff, CCO, Andelyn told BioProcess Insider.

“With access to the increased specificity of MyoAAVs, incorporating them into our portfolio of capsids provides us with an additional means to provide improved and efficient manufacturing regimes for critical gene therapies. The rigorous process and analytical development will still apply, which is why combining the MyoAAVs with the Curator platform has such remarkable potential.”

Andelyn's AAV Curator platform is built on data in AAV manufacturing and understanding that configurability across upstream and downstream technologies ensures the end process is “curated” to each program's specific needs, enabling yield optimization. According to the firm, the platform is built on the concept of modularity and configurability.

“Our approach uses configurable materials and methods optimized for each program’s unique process requirements and is reinforced with data and analytics. We’re constantly evaluating new technologies and analytical methods, and MyoAAVs have the potential to provide improved targeting to muscle tissue while also reducing off-targeting effects to the liver,” said Niloff.

The CDMO has three facilities in Columbus, Ohio to support gene therapy programs from research to commercialization. The firm’s headquarters, also known as Andelyn Corporate Center (ACC) is clinical and commercial production facility purpose-built for the rigors of gene-therapy manufacturing. The Andelyn Development Center (ADC) is where expert and precision-based process development, preclinical manufacturing, and analytical capabilities converge, and Andelyn’s Plasmid Center (APC) specializes in high-quality research, toxicology, and GMP-grade plasmids.