BioMarin is adding kindling to the R&D fire, striking a match with CAMP4 Therapeutics for rights to select two targets identified by the biotech’s RNA platform designed to help create treatments for genetic diseases.
The partners will work to unlock ways in which regulatory RNAs could unlock new ways to address diseases characterized by suboptimal protein expression, Stuart Bunting, BioMarin’s group vice president and head of research, said in an Oct. 1 release.
CAMP4’s tech, known as the RAP platform, is designed to quickly identify the active RNA regulatory elements that control gene expression with the mission of creating RNA-targeting therapies that restore healthy protein levels.
BioMarin will pay CAMP4 an undisclosed upfront payment plus potential milestones and royalties, according to the company release.
While the deal announcement didn’t specificy what indications the two partners will be going after, CAMP4 currently touts a pipeline of metabolic and central nervous system programs. Its most advanced therapy, dubbed CMP-CPS-001, is currently being studied in a phase 1 urea cycle disorder trial. The asset has secured both orphan drug and rare pediatric disease designations from the FDA.
The Cambridge, Massachusetts-based biotech came out of stealth in May 2018, going on to ink partnerships with Alnylam Pharmaceuticals and Biogen. But the biotech later ended those partnerships as the company's focus shifted from signaling pathways to regulatory RNA, heading solo into the wilderness. Now, the biotech is part of a small pack, heading toward the mountaintop with BioMarin in tow.