MiNA Therapeutics Enters Research Collaboration with BioMarin Pharmaceutical to Advance RNAa Platform Research in Rare Genetic Diseases

Key highlights:

• MiNA Therapeutics and BioMarin Pharmaceutical have partnered to discover and develop RNAa therapeutic candidates for rare genetic diseases.
• BioMarin will use MiNA's RNAa algorithm and technology platform to identify RNAa molecules targeting genetic diseases.
• The RNAa platform has been clinically validated in more than 120 patients to date and has the potential to address any gene.

MiNA Therapeutics and BioMarin Pharmaceutical Inc. have signed a multi-target research collaboration and option licensing agreement to discover and develop RNAa (RNA activation) therapeutic candidates targeting a number of rare genetic diseases.

BioMarin will use MiNA's RNAa algorithm and technology platform to identify RNAa molecules targeting genetic diseases. The RNAa platform has been clinically validated in more than 120 patients to date and has the potential to address any gene given its endogenous mechanism of action. Financial terms of the agreement were not disclosed.

Robert Habib, CEO at MiNA said, "Our RNAa therapeutic platform has delivered unique and differentiated clinical benefits in other early clinical studies, demonstrating that MiNA’s platform may have significant benefits over other genetic medicine approaches. We are proud to announce this collaboration with BioMarin, a global leader in the development and commercialization of treatments for rare genetic diseases.’’

Kevin Eggan, SVP and Chief Scientific Officer of BioMarin added, "Activating RNA therapeutics have the potential to change the way we treat certain genetic diseases, particularly those characterized by the limited production of key proteins. This collaboration with MiNA continues that legacy of innovation, creating a partnership around a novel scientific approach that we believe will allow us to target the root cause of several genetic disorders in a new way.”