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CAMP4 Therapeutics to Tackle Rare Diseases

Forms research collaboration with Alnylam

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By: Tim Wright

Editor-in-Chief, Contract Pharma

CAMP4 Therapeutics has formed a research collaboration with Alnylam Pharmaceuticals focused on identifying new druggable targets to address an undisclosed rare disease of the liver. The collaboration brings together CAMP4’s expertise in pinpointing signaling targets that control the expression of genes with Alnylam’s expertise in RNAi therapeutics development to advance novel rare disease drug candidates.
 
“We are excited to partner with Alnylam given its leadership in the RNAi and liver disease space and our companies’ shared commitment to advancing new medicines to address critical unmet patient needs,” said Josh Mandel-Brehm, chief executive officer, CAMP4. “Our Gene Circuitry Platform is yielding the first-ever, comprehensive and dynamic understanding of the Cellular Operating System used by human liver cells to regulate the activation of genes and is applicable to any disease-associated genes in the liver. We look forward to applying this knowledge to elucidate the optimal targets Alnylam can explore for potential RNAi intervention for a disease where there are currently no available treatment options.”
 
CAMP4 will lead all activities to predict and validate disease-modifying targets in signaling pathways. Alnylam will lead in vivo proof-of-concept activities and may elect a target against which to develop RNAi therapies at the completion of the research term. CAMP4 will receive an undisclosed upfront payment from Alnylam as well as milestone payments upon successful achievement of in vitro proof of concept and target election. CAMP4 is also eligible to receive development, approval and sales milestones plus royalties on products developed against the elected target.
 
“CAMP4’s Gene Circuitry Platform provides an innovative approach to target identification and is in line with Alnylam’s strategy to fuel its R&D engine ensuring sustainable growth,” said Kevin Fitzgerald, senior vice president, research, Alnylam. “We welcome this collaboration and are excited for its potential to yield targets implicated in disease settings for which RNAi can be of therapeutic value.”

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