RARE Daily

MiNA and BioMarin Enter Collaboration to Advance RNAa Platform

April 25, 2023

Rare Daily Staff

MiNA Therapeutics, a pioneer of small activating RNA (RNAa) therapeutics, said it has entered into a multi-target research collaboration and option licensing agreement with BioMarin Pharmaceutical for the discovery, and potential development and commercialization of RNAa therapeutic candidates targeting a number of rare genetic diseases.

Under the agreement, BioMarin will employ MiNA Therapeutics’ proprietary RNAa algorithm and technology platform to identify and characterize RNAa molecules targeting a number of genetic diseases for which there are currently no or minimal therapeutic options. The agreement does not cover oncology, or other therapeutic areas outside of genetic disease.

BioMarin has an option to license the assets fully for worldwide development and commercialization.

As part of the deal, MiNA will receive an upfront payment and future royalties will be subject to BioMarin exercising options from the deal. The companies did not disclose financial terms of the agreement.

MiNA’s RNAa platform has been clinically validated in more than 120 patients to date. The platform has the potential to address the root cause of any defective gene and enables the body to self-correct. The company says its approach holds the potential to treat currently “undruggable” diseases.

“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,” said Kevin Eggan, senior vice president and chief scientific officer of BioMarin. He said the collaboration will allow BioMarin to target the root cause of several genetic disorders in a new way.

Photo: Kevin Eggan, senior vice president and chief scientific officer of BioMarin

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