Topic: muscular dystrophy
Another gene therapy alliance shows that Sarepta won’t allow an issue with its lead gene therapy candidate to dampen its enthusiasm for the field.
The gene therapy suppresses expression of a mutated protein and encourages production of a functional version, thereby lessening muscular problems.
Sarepta licenses five new gene therapies to its muscular dystrophy portfolio from Myonexus—and takes an option on buying the company.
Sarepta is hoping to avoid the data-quality controversy that arose over its Duchenne muscular dystrophy drug Exondys51 last year with its next-generation drug golodirsen.
A University of Missouri-led team developed a new gene transfer method to treat Duchenne muscular dystrophy.
Scientists at Sanford Burnham have identified the mechanism that causes muscle cells to stop regenerating, which could lead to methods for slowing age-related muscle decline.
Ventec Life Systems, the maker of integrated respiratory systems, has received FDA 510(k) clearance for its portable life support device.
Israel's Bioblast Pharma has abandoned plans for a securities offering, blaming what it called "adverse market conditions" for the decision.