CAMP4 Enters Strategic Research Collaboration to Advance Novel Regulatory RNA-Targeting Medicines for Rare Genetic Conditions
01 Octubre 2024 - 6:00AM
CAMP4 Therapeutics Corporation, a clinical-stage biotechnology
company developing a pipeline of regulatory RNA-targeting
therapeutics designed to upregulate gene expression with the goal
of restoring healthy protein levels across a range of genetic
diseases, today announced a new research collaboration with
BioMarin Pharmaceutical Inc. aimed at advancing novel therapeutics
that increase protein levels by targeting regulatory RNA (regRNA)
sequences, which are key elements controlling gene expression.
Josh Mandel-Brehm, Chief Executive Officer of CAMP4, said,
“BioMarin is a leader in genetic medicines with a successful track
record of advancing disease-modifying therapies for patients with
rare genetic conditions. 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. We look forward to partnering with the BioMarin team with
the aim of bringing forward novel therapeutic targets.”
CAMP4’s RAP Platform™ is designed to rapidly and systematically
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. 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™, designed to bind to the identified regRNA and
amplify the expression of the target gene in a specific and
controllable way, with the goal of restoring healthy protein
levels.
“We are excited to work with CAMP4 and their RAP Platform to
further understand how regulatory RNAs could unlock novel ways to
address diseases characterized by suboptimal protein expression,”
said Stuart Bunting, Group Vice President, Head of Research at
BioMarin.
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.
About CAMP4 Therapeutics
CAMP4 is developing disease-modifying treatments for a broad
range of genetic diseases where amplifying healthy protein may
offer therapeutic benefits. Our approach amplifies mRNA by
harnessing a fundamental mechanism of how genes are controlled. To
amplify mRNA, our therapeutic ASO drug candidates target regulatory
RNAs (regRNAs), which act locally on transcription factors and are
the master regulators of gene expression. CAMP4’s proprietary
RAP Platform™ enables the mapping of regRNAs and generation of
therapeutic candidates designed to target the regRNAs of
haploinsufficient and recessive partial loss-of-function disorders,
of which there are more than 1,200, in which a modest increase in
protein expression may have the potential to be clinically
meaningful. Learn more about us
at www.CAMP4tx.com and follow us on
LinkedIn and X.
Contacts
Media:Jason Braco, Ph.D. LifeSci
Communicationsjbraco@lifescicomms.com(646) 876-4932