Stoke Receives $40M to Fund New Therapies for MD, Other Genetic Diseases

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by Alice Melão |

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Stoke Therapeutics

Apple Tree Partners has invested $40 million in Stoke Therapeutics to come up with gene-expression treatments for muscular dystrophy and other genetic disorders.

Stoke aims to develop antisense oligonucleotides to promote targeted augmentation of nuclear gene output (TANGO). The goal is to reverse the features of rare diseases caused by a single mutated gene that affect the central nervous system, eye and liver. This strategy could potentially reverse or prevent progression of diseases that benefit from increased amounts of a non-mutated gene.

Small synthetic RNA oligonucleotides can work as a bridge to modulate the way RNA, the product of genes’ transcription, is read and processed. The idea is to encourage production of a functional version of the missing protein.

Dr. Edward M. Kaye will serve as CEO of Stoke, which is headquartered in Bedford, Massachusetts.

“Stoke Therapeutics represents a bold step forward in opening up a vast new area of drug development focused on up-regulation of gene expression,” Kaye said in a press release. “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.”

Kaye developed this therapeutic strategy in collaboration with Dr. Adrian Krainer of Cold Spring Harbor Laboratory, an expert in RNA processing. Kainer helped develop the recently approved antisense therapy Spinraza (nusinersen) to treat children with spinal muscular atrophy (SMA).

Added Sam Hall, principal of Apple Tree Partners: “We are tremendously impressed by the broad potential of this approach to address so many debilitating diseases and have assembled the team, platform and funding to thoroughly exploit this opportunity.”