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Stoke Therapeutics Raises $90 Million Series B for Therapies to Restore Gene Expression

Antisense oligonucleotide therapies to increase gene expression for the treatment of severe genetic diseases. Lead candidate in Dravet Syndrome (Image: Stoke Therapeutics).

Dravet Syndrome is a rare, catastrophic, lifelong form of epilepsy that begins in a baby’s first year of life with frequent and/or prolonged seizures. About 1 in 16,000 people are affected by Dravet syndrome, according to the Dravet Syndrome Foundation, 80% of whom have a mutation in their sodium channel 1 alpha gene (SCN1A). While seizures persist, other problems such as developmental delay and abnormal EEGs are often not evident until the second or third year of life. Patients with Dravet syndrome face a 15% to 20% mortality rate due to sudden unexpected death in epilepsy (SUDEP), prolonged seizures, seizure-related accidents such as drowning, and infections.

Bedford, Massachusetts, based Stoke Therapeutics is developing antisense oligonucleotide (ASO) therapies that increase gene expression to treat a wide array of severe genetic diseases. Antisense oligonucleotides are short, synthetic, single-stranded polymers that can influence RNA processing, thereby modifying protein expression to have a specific effect. The company’s lead indication is Dravet Syndrome, for which it intends to be in human clinical trials by 2020. Stoke’s technology has the potential to provide the first gene-specific, disease-modifying approach for Dravet Syndrome to prevent seizures and reduce developmental deficits. The company is pursuing other genetic conditions affecting the central nervous system, eye, ear, liver and kidney.

Stoke announced today that it has raised $90 million in Series B funding led by RTW Investments with participation from founding investor Apple Tree Partners—who completed a $40 million Series A round in January 2018—and new investors RA Capital Management, Cormorant Asset Management, Perceptive Advisors, Janus Henderson Investors, Redmile Group, Sphera Funds Management and Alexandria Venture Investments. Ben Fidler writes in Xconomy that this latest financing “lays the foundation for an IPO that could happen in the first half of 2019,” according to Stoke Therapeutics CEO Edward Kaye, MD. Dr. Kaye is the former CEO of Sarepta Therapeutics SRPT, which he led through the approval of Exondys 51 (eteplirsen), an antisense oligonucleotide therapy that was the first FDA-approved treatment for Duchenne muscular dystrophy.

Source: Stoke Therapeutics

Stoke has identified thousands of genes that could be addressed by its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) technology, which targets non-productive RNA splicing, a critical step in gene expression, to address the root cause of monogenic diseases caused by loss or reduction of gene function.

Our technology is designed to address, for the first time, the genetic cause of diseases like Dravet Syndrome so we can do more than alleviate symptoms—we can potentially prevent the long-term disabling consequences of these diseases.

-Edward M. Kaye, MD, CEO, Stoke Therapeutics.

In addition to Dr. Kaye’s history with Sarepta’s Exondys 51, Stoke has another critical link to a groundbreaking antisense oligonucleotide drug. The company’s co-founder, Adrian Krainer, PhD, is the St. Giles Professor of Molecular Genetics and Program Chair of Cancer and Molecular Biology at Cold Spring Harbor Laboratory. Prof. Krainer’s fundamental and applied research on RNA splicing for over three decades directly led to the invention and development of Biogen’s BIIB blockbuster Spinraza (nusinersen), the first FDA-approved treatment for spinal muscular atrophy and the first antisense oligonucleotide drug to restore expression of a fully functional protein via splicing correction. Spinraza generated $1.42 billion in revenue for Biogen during the 12 months ended June 30, 2018.

Stoke Therapeutics, with its innovative scientific approach, strong preclinical data and seasoned leadership team, is well-positioned to be a leader in oligonucleotide therapeutics. Rare disease patients are waiting, and we look forward to supporting the Stoke team as it builds a great precision medicine company advancing disease-modifying programs into the clinic.

-Roderick Wong, MD, managing partner and chief investment officer, RTW Investments, and new Board observer, Stoke Therapeutics.

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