Zikani Therapeutics, a Watertown, Mass.-based developer of novel ribosome modulating agents (RMAs) for the treatment of rare, nonsense mutation-driven diseases, closed a $7.5m Series A-1 financing.
Backers included Advent Life Sciences, Gurnet Point Capital and Roche Venture Fund.
The company will use the proceeds to advance its current nonsense mutation readthrough programs from lead optimization to candidate selection.
Led by Sumit Aggarwal, President and CEO, Zikani Therapeutics is an emerging leader in the science of ribosome modulation, leveraging its innovative TURBO-ZMTM chemistry technology platform to develop novel Ribosome Modulating Agents (RMAs) as therapeutics for people with limited treatment options. Its TURBO-ZMTM platform allows rapid synthesis of novel compounds that can be optimized to modulate the ribosome in a disease specific manner. The company is actively moving into pre-clinical development to target select rare diseases including inherited diseases and cancers caused by nonsense mutations. Zikami has demonstrated positive, pre-clinical 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).