Sarepta In-Licenses Next-Gen AAV Tech Covering Five Indications
Sarepta Therapeutics Inc (NASDAQ: SRPT) will license a new group of adeno-associated viruses from the institute for undisclosed upfront payment and milestone promises.
Under the agreement, Sarepta will have the rights to five neuromuscular and cardiac indications, including Duchenne muscular dystrophy.
According to Sarepta, the deal covers the MyoAAV program that aims to deliver more efficient gene therapies and at lower doses using modified capsids.
Also Read: Sarepta's Duchenne Gene Therapy Update Gets Bullish Stance From Analysts.
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% and showed lower accumulation in the liver.
Due to increased efficiency, it can be used at up to a log lower dose than traditional AAV vectors.
Full research for MyoAAV in a Duchenne mouse model was published in Cell in 2021. Sarepta CEO Doug Ingram said the viruses could "substantially reduce viral load," while CSO Louise Rodino-Klapac touted the platform's "broad applicability."
Price Action: SRPT shares are up 3.17% at $109.38 during the market session on the last check Monday.
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