RARE Daily

Zikani Therapeutics Raises $7.5 Million to Treat Rare Nonsense Mutations

April 21, 2020

Rare Daily Staff

Zikani Therapeutics, a biotech developing novel ribosome modulating agents for the treatment of rare, nonsense mutation-driven diseases, has closed a $7.5 million financing.

Advent Life Sciences, Gurnet Point Capital, and Roche Venture Fund all participated in the series A-1 round.

Zikani will use the proceeds to advance its current nonsense mutation read-through programs from lead optimization to candidate selection and form collaborations with partners to advance its platform. The company has demonstrated positive, preclinical data in multiple disease areas, including: APC mutant colon cancer, familial adenomatous polyposis (FAP), class 1 cystic fibrosis, and recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB).

“Zikani has generated promising data that validates its TURBO-ZM platform and addresses a completely unmet need in oncology therapeutics: restoring expression of a major tumor suppressor gene,” said Keith Flaherty, director of clinical research at Massachusetts General Hospital Cancer Center and a senior advisor to Zikani.

Ribosomal RNAs form the translation machinery that generates function proteins from genetic sequences. Ribosome modulation provides a therapeutic approach to addressing a number of diseases, but the development of disease-specific ribosome modulating agents (RMAs) has been a challenge. Zikani’s proprietary platform TURBO-ZM allows rapid synthesis of novel RMAs that can be optimized to target the human ribosome in a disease specific manner.

“We’re highly encouraged by the data we’ve generated to date and are working rapidly to advance compounds into preclinical development,” said Sumit Aggarwal, president and CEO of Zikani.

Photo: Sumit Aggarwal, president and CEO of Zikani


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