CAMP4 Therapeutics secures orphan drug status for CMP-CPS-001 for the treatment of urea cycle disorders

CAMBRIDGE, Mass., Sept. 17, 2024 (GLOBE NEWSWIRE) — CAMP4 Therapeutics, a clinical-stage biotechnology company developing a pipeline of regulatory RNA-targeted therapeutics designed to upregulate gene expression to restore healthy protein levels in a range of genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CMP-CPS-001 for the treatment of urea cycle disorders (UCDs).

“Today, there are no disease-modifying therapies for patients with UCDs, and this community is in urgent need of innovative approaches to mitigate potentially devastating health consequences such as neurological disability, seizures and death,” said Josh Mandel-Brehm, Chief Executive Officer of CAMP4. “Based on our preclinical studies, we believe CMP-CPS-001 has the potential to change the paradigm in the treatment of patients with UCDs. Our preclinical studies have demonstrated that by modulating the activity of the target regRNA, CMP-CPS-001 increases levels of a key enzyme in the urea cycle, allowing more ammonia to be converted to urea and reducing ammonia levels to normal, healthy ranges.”

About the status of an orphan drug
Orphan drug designation is granted by the FDA and is intended to support the development of new therapeutics for rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation entitles companies to certain incentives, including tax credits for qualified clinical trials, exemption from FDA user fees, and potential eligibility for seven years of market exclusivity in the United States following FDA approval. Orphan disease drugs are subject to the same rigorous scientific review processes as any other drug for approval or licensing.

About CMP-CPS-001
CMP-CPS-001 is an antisense oligonucleotide (ASO)-based therapeutic candidate for the treatment of UCDs that targets carbamoyl phosphate synthetase 1 (CPS1), an enzyme that catalyzes the first step of the urea cycle that converts ammonia to urea. CMP-CPS-001 is designed to upregulate CPS1 gene expression by binding to a CPS1-specific regulatory RNA sequence to ultimately increase CPS1 protein levels. CMP-CPS-001 is currently being evaluated in a Phase 1 clinical trial in healthy volunteers (NCT06247670). CMP-CPS-001 has been designated as a rare pediatric disease by the FDA.

About CAMP4 Therapeutics
CAMP4 is developing disease-modifying treatments for a broad range of genetic diseases where amplification of healthy proteins can provide therapeutic benefits. Our approach amplifies mRNA by exploiting a fundamental mechanism of gene control. 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 the generation of therapeutic candidates targeting the regRNAs of haploinsufficient and recessive partial loss-of-function diseases, of which there are more than 1,200 and where a moderate increase in protein expression may be clinically meaningful. Learn more about us at www.CAMP4tx.com and follow us on LinkedIn And X.

The story continues

Contacts

Media:
Jason Braco, Ph.D.
LifeSci Communication
[email protected]
(646) 876-4932