Shape Therapeutics, a Seattle preclinical stage biotech company developing RNA editing and gene therapy technologies, has raised $112 million.
The company’s RNA editing technologies are spun out of the lab of co-founder Prashant Mali, a bioengineer at the University of California, San Diego who is on Shape’s scientific advisory board. Shape is also improving methods to deliver genetic material into cells.
“We continue to harness the potential of RNA therapeutics to redefine the standard of care for genetic diseases,” said CEO and co-founder Francois Vigneault, former VP of research at Juno Therapeutics, a Seattle flagship cell therapy company that was acquired by Celgene in 2018. Other Juno veterans at the company include Adrian Briggs, head of platform technologies, and David Huss, vice president and head of research.
The company is working on improving AAV vectors, a gene therapy delivery system that is the backbone of FDA-approved gene therapies for spinal muscular atrophy and a rare vision disease. Shape Therapeutics is developing AAV vectors that deliver genetic material directly to the nervous system or muscle, according to a news release. Its vectors can be used to deliver a variety of genetic payloads, including components of its RNA editing technology.
Shape’s RNA editing technology could potentially be used to treat genetic conditions, and for other uses such as changing the amount of a key regulatory enzyme in the body.
The company envisions its technology being applied to a “wide range of therapeutic areas,” Vigneault said in the release. As examples, he named Parkinson’s disease, Alzheimer’s disease, alpha-1 antitrypsin deficiency and Rett syndrome.
One challenge to RNA-editing approaches is that they can yield only low levels of corrected protein, according to Nature.
But one key advantage of RNA editing is that, unlike DNA editing, changing an RNA sequence does not result in permanent effects on the genome, as RNA is only transiently made in the body. The approach may also minimize the possibility that the body will have an immune reaction to the all-human RNA editing components. “You really don’t need heavy machinery to target RNA,” Mali said in an interview with Nature.
The company’s scientific board also includes Don Cleveland, who in 2018 was awarded a Breakthrough Prize in Life Sciences for his work on RNA therapies in animal models of Huntington’s disease and Amyotrophic lateral sclerosis (ALS), also called Lou Gehrig’s disease.
Shape, founded in 2018, will be jostling with other companies working to improve AAV vectors and gene therapies including Affinia Therapeutics and Dyno Therapeutics. Biotech companies involved in the development of RNA-based therapeutics include Beam Therapeutics, Locana and Korro Bio.
The $112 million Series B financing round was co-led by Decheng Capital and Breton Capital, with participation from Willett Advisors, and continued participation from New Enterprise Associates, and Mission BioCapital. The financing builds on $35.5 million in Series A financing in 2019 with participation from CureDuchenne Ventures, the investment arm of CureDuchenne, a nonprofit dedicated to finding cures for Duchenne muscular dystrophy.