BioMarin, CAMP4 to develop RNA-targeting therapies for genetic diseases

Oct. 1, 2024

BioMarin Pharmaceutical and CAMP4 Therapeutics have entered into a research collaboration to develop RNA-targeting medicines aimed at increasing protein levels in patients with rare genetic diseases. 

Under the agreement, BioMarin will select two targets identified through CAMP4’s RAP Platform for further development. CAMP4 will receive an upfront payment and is eligible for additional milestone payments and tiered royalties based on the success of the collaboration.

CAMP4’s RAP Platform focuses on identifying RNA regulatory elements that influence gene expression and developing RNA Actuators, which are designed to boost the production of proteins by targeting specific RNA sequences. The collaboration aims to identify and develop new therapies that address diseases caused by insufficient protein levels.

This collaboration strengthens BioMarin’s position in genetic medicines while allowing CAMP4 to expand its platform’s reach into additional therapeutic targets.