Life Sciences Drug DevelopmentBedford, MA
ASOthera Pharmaceuticals is a privately-held biotechnology company focused on the development of first-in-class therapeutics that selectively increase gene expression to treat genetic diseases. ASOthera’s technology is based on research from the laboratory of Professor Adrian Krainer at Cold Spring Harbor Laboratory (CSHL), the inventor of Isis-SMNRx (in Phase 3 for Spinal Muscular Atrophy) and a repeat innovator in the fields of RNA splicing and oligonucleotide / antisense therapeutics. ASOthera’s approach constitutes an extremely novel application of antisense technology, a mature and FDA-approved therapeutic modality, and ASOthera is building a pipeline of drug candidates to treat a diverse set of human genetic diseases in tissues that are clinically proven to be addressable with antisense oligonucleotides, including CNS, eye, liver and kidney. ASOthera is located in Bedford, MA and is backed by Apple Tree Partners (www.appletreepartners.com), a $1.5B healthcare-focused venture capital firm based in New York City.