Chemistry-Based Biotech Start-Ups Secure Funding

Two chemistry-based biotech firms—both with high-profile founders—have each raised their first round of financing. WaVe Life Sciences has gathered $18 million to pursue “stereopure” nucleic acid therapies, and Lysosomal Therapeutics Inc. (LTI) has secured $20 million to support the development of small molecules that treat neurodegenerative diseases.

WaVe got its start in 2013, when Harvard University chemist Gregory L. Verdine and Tokyo University of Science’s Takeshi Wada combined their respective start-ups, Ontorii Pharmaceuticals and Chiralgen. WaVe is a nod to the two scientists’ names.

Pharmaceutical companies have long tried to dial up efficacy and dial down safety concerns by focusing on single enantiomers of their small-molecule drug candidates. Successful single-enantiomer drugs include the heartburn pill Nexium and the antidepressant Lexapro.

WaVe’s goal is to control the chirality of nucleic acid therapeutics. Its initial focus will be on oligonucleotides—nucleic acid strings in which each phosphorothioate modification introduces a new chiral center. As WaVe CEO Paul Bolno points out, an oligo consisting of 20 nucleic acids has 19 chiral centers, meaning the final drug could contain a half-million unique molecules with a range of pharmaceutical properties.

Although scientists working on such drugs have long pondered the consequences of that mixture, their challenge has been controlling the chirality of each phosphorothioate to test for efficacy and safety, Bolno says. WaVe has found a way to do that and is now designing isomers that maximize the best features of a drug.

WaVe already has a partnership with a big pharma firm and a team of 20 people across its labs in the U.S. and Japan, Bolno says. And it has plans to “significantly grow this year,” he adds.

Separately, Cambridge-based LTI also has secured an influx of cash. Founded last year by a team of ex-Genzyme executives including former CEO Henri A. Termeer, LTI aims to exploit genetic connections between rare lysosomal storage disorders and more common neurodegenerative disorders. The biotech firm’s first program is based on the observation that people with Gaucher’s disease are predisposed to the more common Parkinson’s disease.

The company has three goals for the cash, according to CEO Kees Been. Its top priority is to optimize and gather preclinical data on a glucocerebrosidase (GCase) lysosomal enzyme activator for Parkinson’s disease. The money will also support a biomarker study, which the company hopes will identify patients who respond to its GCase activator. Last, LTI wants to start new programs for other neurodegenerative diseases.

LTI currently has 10 employees and is looking to add to its team.