- 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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