A new WaVe in nucleic acid therapeutics

WaVe Life Sciences closes $18 million Series A financing to advance stereopure nucleic acid therapeutics

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BOSTON & OKINAWA, Japan—WaVe Life Sciences has closed an $18 million Series A financing, led by RA Capital Management, LLC and Kagoshima Shinsangyo Sosei Investment LP, joining founding investor SNBL Ltd. The proceeds will fund the advancement of the company’s pipeline of stereopure nucleic acid therapeutics, including antisense and exon-skipping drug candidates, which are being developed to treat diseases across multiple therapeutic areas. Peter Kolchinsky, Ph.D., managing general partner of RA Capital, has joined WaVe’s board of directors.
WaVe is building on the success of stereopure medicines (think back to left/right chirality in Chem 101) with the first and reputedly the only chemistry platform capable of precisely synthesizing stereopure nucleic acid therapeutics, designed to possess enhanced pharmacology when compared to conventional drugs and drug candidates in this class. This is an improvement from conventional nucleic acid therapeutics, such as antisense, siRNA, miRNA and mRNA, which are composed of complex mixtures containing hundreds or thousands of unique isomers with the exact same nucleotide sequence. WaVe’s novel chemistry enables direct control of RNA cleavage sites to increase antisense potency and can also be used to synthesize compounds that target allele specific diseases, an area of high unmet need.
WaVe’s drug discovery platform is capable of producing single, precisely defined oligonucleotide structures that provide enhanced pharmaceutical properties compared to those of conventional drugs in this class.

“Nucleic acid therapeutics are a rapidly growing area of next-generation drugs that promise to broadly and fundamentally improve the treatment of human disease. Until now, the class’ full potential has been dramatically limited by products containing complex mixtures of drug molecules. At WaVe, we have shown that single drug products demonstrate superior pharmaceutical properties, and we have developed the capability to design, synthesize and advance these superior products for a broad range of disease indications,” said Paul Bolno, M.D., president and CEO of WaVe Life Sciences. “Our chemistry intersects with all the major classes of nucleic acid therapeutics currently being developed, from antisense to siRNA to mRNA and microRNA, and we therefore anticipate having a broad and deep impact on the ability of the entire field to reach its full potential.”

Conventional antisense medicines containing a phosphorothioate backbone comprise mixtures of drug products that have as many as 500,000 or more individual components, each of which behaves differently, the company stated in a news release. WaVe’s transformative and commercially scalable chemistry enables programmable control over the structure of the drug product during its synthesis, affording precisely tailored drug molecules designed to possess optimized pharmacokinetic and therapeutic properties.

WaVe’s technology, when applied to antisense, directs cellular machinery to disable mRNA through cleavage at defined, predetermined sites, which enables targeting of specific disease-causing genetic variants, known as alleles. It offers a compelling new modality for treating diseases resulting from allelic variation, a major unsolved problem in personalized medicine and an area of particularly high unmet medical need.

“We are absolutely floored by the enhancements that WaVe’s advances represent for multiple platforms, including mRNA-targeted antisense, exon-skipping and other forms of epigenetic modulation. WaVe’s technology has the potential to both improve upon existing nucleic acid programs in development across the industry as well as solve new problems, opening doors to new disease areas,” said RA Capital’s Kolchinsky. “We love investing in companies with talented, driven people with whom we can engage in an idea-rich prioritization process, including determining which programs to develop independently, spin-out or partner, and WaVe is among the richest we have seen.”

Dr. Bolno added, “With the powerful financial and intellectual support of our existing and new investors and this additional funding in place, we will advance our programs through preclinical development and continue to strengthen our core technology capabilities.”

WaVe maintains R&D facilities in Boston and Japan. The company was founded by two scientific leaders Gregory Verdine (Harvard University) and Takeshi Wada (Tokyo University of Science).

WaVe Life Sciences was founded on the core principle that medicines should possess precisely controlled molecular structures. The company and its strategic partners are advancing a portfolio of stereopure drug candidates that target underlying biology across a wide range of human diseases.

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