Sarepta Licenses MyoAAV Next-Gen Capsids from Broad Institute for Rare Genetic Diseases

Sarepta Therapeutics reported that following progress on its sponsored research agreement on the MyoAAV program, it has executed a license agreement with the Broad Institute of MIT and Harvard for MyoAAV in Duchenne muscular dystrophy and certain other neuromuscular and cardiac indications.

Photo: Louise Rodino-Klapac, executive vice president, head of research and development and chief scientific officer for Sarepta

The announcement follows confirmation by Sarepta’s internal research and manufacturing teams of earlier published research from Broad Institute, which was conducted under a research agreement that began in 2020.

MyoAAV is a new group of adeno-associated viruses (AAV) that use a modified outer protein shell of AAV, known as the capsid, to deliver genetic therapies with greater efficiency and at lower doses. Data published in the journal Cell in 2021 found that, in mouse models of Duchenne muscular dystrophy and X-linked myotubular myopathy, MyoAAV demonstrated more efficient delivery of gene therapy and gene editing payloads, resulting in complete restoration of muscle function and improved survival.

In preclinical data from non-human primates, compared to natural AAV serotypes, MyoAAV delivered 25-50 times greater gene expression in multiple skeletal muscles and 10-15 times greater gene expression in cardiac muscle; demonstrated reduced delivery to the liver by 50 percent and showed lower accumulation in the liver; and can be used at up to a log lower dose than traditional AAV vectors, due to increased efficiency.

“Research published by Broad Institute, and so far, corroborated by Sarepta’s own internal research, reinforces the potential of MyoAAV as a breakthrough next-generation approach in genetic medicine delivery,” said Doug Ingram, president and CEO, Sarepta. “The significantly improved efficiency of MyoAAV may unlock the ability to effectively deliver genetic medicine at as much as a log lower doses when compared to current AAVs, which could substantially reduce viral load and cost of goods in the future.”

“The MyoAAV platform is noteworthy for its broad applicability across multiple disease states and will further advance the science of genetic medicine allowing for swift advancement of treatments into the clinic across a variety of conditions, including larger rare cardiac and neuromuscular indications,” said Louise Rodino-Klapac, executive vice president, head of research and development and chief scientific officer for Sarepta.

Under the terms of the agreement, Sarepta will receive worldwide commercial license grants for five neuromuscular and cardiac indications, including Duchenne muscular dystrophy, plus exclusive options on additional targets. In addition to an upfront payment, Broad Institute is entitled to future royalties and milestone payments, details of which were not disclosed.

Author: Rare Daily Staff