Stoke Therapeutics, Inc., a Bedford, MA-based new company working to increase gene expression to treat a wide array of diseases caused by genetic insufficiency, completed a $40m Series A financing.
The round was provided by founding investor Apple Tree Partners.
The company intends to use the funds to support the further advancement of multiple pre-clinical development programs.
Led by CEO Edward M. Kaye, M.D., Stoke a biotechnology company working to increase gene expression to treat a wide array of severe genetic diseases, including genetic conditions affecting the central nervous system, eye, and liver. The company is pioneering a therapeutic approach using antisense oligonucleotides to increase the expression of proteins whose function is reduced in genetic diseases.
It is focused specifically on modulating RNA splicing, a critical step in gene expression, to increase the production of messenger RNA that can be translated into protein.
The technological foundation of Stoke’s approach was developed in close collaboration with Scientific Founder Adrian Krainer, Ph.D., of Cold Spring Harbor Laboratory, an expert in RNA splicing and an inventor of the recently approved antisense therapy SPINRAZA® (nusinersen), a life-saving drug for children with Spinal Muscular Atrophy (SMA).
Since its inception, the company has validated hundreds of disease target genes that could be up-regulated with its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform and is prioritizing therapeutic programs that target antisense-addressable tissues including the central nervous system, eye, and liver. While Stoke’s initial focus is on inherited diseases caused by the reduced function of a single mutated gene, the TANGO approach is also applicable to diseases whereby targeted augmentation of a non-mutated gene could reverse or prevent disease progression.
Stoke Therapeutics is also led by:
– Huw M. Nash, Ph.D., Founding CEO, Chief Operating Officer and Chief Business Officer.
– Barry Ticho, M.D., Ph.D., FACC, Chief Medical Officer.
– Isabel Aznarez, Ph.D., co-founder and VP, Head of Biology.
– Charles R. Allerson, Ph.D., VP, Head of Chemistry.
FinSMEs
04/01/2018