BEDFORD, Mass. – Stoke Therapeutics, Inc, a new company working to increase gene expression to treat a wide array of diseases caused by genetic insufficiency, announced that it has completed a $40 million Series A financing to support the further advancement of multiple pre-clinical development programs. The Series A round was provided by founding investor Apple Tree Partners.
Edward M. Kaye, M.D., an industry leader in the development of gene-based medicines for rare diseases and most recently the Chief Executive Officer of Sarepta Therapeutics, has joined Stoke as Chief Executive Officer. He leads a deeply experienced management team with a track record of successfully translating novel biology into groundbreaking new therapies for patients.
Stoke is pioneering a unique therapeutic approach using antisense oligonucleotides to increase the expression of proteins whose function is reduced in genetic diseases. The company 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.
“Stoke Therapeutics represents a bold step forward in opening up a vast new area of drug development focused on up-regulation of gene expression,” said Edward M. Kaye, Stoke Therapeutics Chief Executive Officer. “By restoring gene dosage using target-specific antisense approaches, we have the opportunity to create a new way of treating diseases that are not amenable to enzyme replacement, gene therapy or other existing modalities.”
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, Stoke 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.