CAMP4 Enters Collaboration with BioMarin to Advance Novel Regulatory RNA-Targeting Medicines

Rare Daily Staff

CAMP4 Therapeutics said it entered into a research collaboration with BioMarin Pharmaceutical to advance novel therapeutics that increase protein levels by targeting key elements controlling gene expression known as regulatory RNA sequences.

CAMP4 is developing regulatory RNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels across a range of genetic diseases.

The agreement will provide CAMP4 with BioMarin’s drug development experience while helping to invigorate BioMarin’s pipeline after it recently pared back programs, faces market disappointment on adoption of its hemophilia A gene therapy, and is considers potential competition to its products as drug developers advance new therapies toward the market.

Under the terms of the agreement, BioMarin has the right to select two targets identified by CAMP4’s RAP Platform to advance into clinical development. The companies did not disclose financial terms of the agreement, but said BioMarin will provide an upfront payment and that CAMP4 will be eligible for milestones and tiered royalties.

CAMP4’s RAP Platform is designed to identify and characterize the active RNA regulatory elements controlling every expressed gene and tens of thousands of druggable enhancer and promoter regRNA sequences that control protein-coding genes rapidly and systematically.

Once a disease-associated target gene is identified, CAMP4 applies its RAP Platform to identify the controlling regRNA and rapidly generate novel antisense oligonucleotide candidates, called RNA Actuators. These are designed to bind to the identified regRNA and amplify the expression of the target gene in a specific and controllable way with the potential of restoring healthy protein levels.

“BioMarin is a leader in genetic medicines with a successful track record of advancing disease-modifying therapies for patients with rare genetic conditions,” said Josh Mandel-Brehm, CEO of CAMP4. “We believe this collaboration will expand the reach of our approach and enable us to leverage our RAP Platform to identify and target additional regRNAs associated with disease.”

Photo: Josh Mandel-Brehm, CEO of CAMP4