Voyager Therapeutics Inc. has entered into a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG, to advance potential gene therapies for Huntington’s disease and spinal muscular atrophy (SMA).
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy. Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy (SMA). Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique. “This base editing approach to treating SMA should be applicable to all SMA patients, regardless of the specific mutation that caused their SMN1 loss,” the lead author David Liu, a professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of Harvard and MIT, told BioWorld.
Spinal muscular atrophy (SMA) has been surfacing more regularly in scientific journals lately, as drug developers – such names as Biohaven Inc., Roche Holding AG and Scholar Rock Inc. – continue to search for improved therapies directed at the condition, one that takes in a group of hereditary, motor neuron-destroying diseases.
New top-line data from Scholar Rock Holding Group’s phase II Topaz trial of apitegromab (SRK-015) in patients with type 2 and type 3 spinal muscular atrophy generated enough proof-of-concept results for the company to plan on initiating a phase III for later this year. But the 12-month data didn’t stop the Cambridge, Mass.-based company’s stock (NASDAQ:SRRK) from struggling mightily on April 6 as shares closed 20.3% lower at $35.97 each.
Shares of Cambridge, Mass.-based Scholar Rock Holding Corp. closed Oct. 27 at $30.02, up $16.30, or 119%, on positive six-month interim analysis results from the Topaz phase II trial with inhibitor of myostatin activator SRK-015 in type 2 and type 3 spinal muscular atrophy, and CEO Tony Kingsley pointed to “a rich cascade of data ahead of us.”
More than two weeks ahead of its expected PDUFA date, PTC Therapeutics Inc.’s spinal muscular atrophy (SM) drug, risdiplam, gained FDA approval, making it the first at-home, oral treatment intended for use in adults and children 2 months and older.