CPS1

CAMBRIDGE, Mass., Sept. 17, 2024 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics, 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 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 available for patients with UCDs, and this community is in desperate need of innovative approaches to help mitigate potentially devastating health outcomes, including neurologic 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 shift the paradigm in how patients with UCDs are treated. Our preclinical studies have demonstrated that by modulating the activity of the target regRNA, CMP-CPS-001 increases the levels of a key enzyme in the urea cycle, which allows for more ammonia to be converted into urea, thereby lowering ammonia levels to normal, healthy ranges.” About Orphan Drug DesignationOrphan Drug Designation is granted by the FDA and is designed 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 qualifies companies for certain incentives, which may include tax credits for qualified clinical trials, exemption from FDA user fees, and potential eligibility for seven years of market exclusivity in the United States upon FDA approval. Drugs for rare diseases are held to the same rigorous scientific review processes as any other drug for approval or licensing. About CMP-CPS-001CMP-CPS-001 is an antisense oligonucleotide (ASO) therapeutic candidate for the treatment of UCDs targeting carbamoyl phosphate synthetase 1 (CPS1), an enzyme that catalyzes the first step of the urea cycle, which 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 under evaluation in a Phase 1 clinical trial in healthy volunteers (NCT06247670). CMP-CPS-001 has been granted Rare Pediatric Disease Designation by the FDA. About CAMP4 TherapeuticsCAMP4 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 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

CAMBRIDGE, Mass., Aug. 27, 2024 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics, a clinical-stage biotechnology company developing a pipeline of RNA-based therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels across a range of genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to the Company’s lead investigational drug candidate CMP-CPS-001 for the treatment of urea cycle disorders (UCDs). The safety, tolerability, and pharmacokinetics of CMP-CPS-001 are currently being evaluated in a randomized, placebo-controlled Phase 1 trial in Australia. “This designation from the FDA underscores the seriousness of this rare disease and the critical need for new therapeutic options for patients living with UCDs who currently rely on supportive treatments that fail to address the underlying driver of the disease,” said Josh Mandel-Brehm, Chief Executive Officer of CAMP4. “By upregulating the expression of the gene encoding a key enzyme in the urea pathway to restore healthy protein levels, CMP-CPS-001 has the potential to be the first disease-modifying treatment for UCDs. With our Phase 1 clinical study underway, we look forward to evaluating the potential of this candidate for patients living with serious disorders of urea metabolism.” UCDs are a group of rare, severe, inherited metabolic diseases impacting protein metabolism that affect patients of all ages. People with UCDs accumulate excessive ammonia in their blood, which may cause irreversible brain damage, disability, and seizures, and can be fatal. There are currently no approved disease-modifying therapeutics for the most prevalent forms of UCDs. About Rare Pediatric Disease DesignationThe FDA's Rare Pediatric Disease Designation program incentivizes drug development for rare pediatric conditions. A rare pediatric disease is defined as a serious or life-threatening disease in which the manifestations primarily impact individuals from birth to 18 years of age. A sponsor who receives FDA approval for a therapeutic for a rare pediatric disease may qualify for a voucher that can be used to obtain priority review for a subsequent marketing application for a different product. About CMP-CPS-001CMP-CPS-001 is an antisense oligonucleotide (ASO) therapeutic candidate for the treatment of urea cycle disorders targeting carbamoyl phosphate synthetase 1 (CPS1), the first enzyme in the metabolic 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 under evaluation in a Phase 1 clinical study (NCT06247670). About CAMP4 TherapeuticsCAMP4 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 design of optimal chemistry to generate potent therapeutic candidates to address more than 1,200 genetic diseases across multiple tissues. 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

March 21, 2024 -- CAMP4 Therapeutics Corp., a clinical-stage biotechnology company harnessing the power of regulatory RNA to restore healthy gene expression, today announced that the first participant has been dosed in the company’s Phase 1 clinical study of CMP-CPS-001, a potential first-in-class therapeutic for the treatment of urea cycle disorders (UCDs). “This trial initiation is an exciting milestone, representing CAMP4’s first investigational drug candidate to be studied in a clinical trial and one of the first mRNA-amplifying therapeutics in the clinic,” said Josh Mandel-Brehm, CEO of CAMP4. “Most importantly, the clinical development of CMP-CPS-001 is a step toward potentially bringing a new, disease-modifying treatment to individuals living with UCDs. Going from initial screen to clinical initiation within three years provides further validation of the power and hyper-efficiency of our RAP Platform to create precisely targeted therapies for genetic diseases by amplifying mRNA to increase healthy gene expression.” UCDs are a group of rare, severe, inherited metabolic diseases impacting protein metabolism. People with urea cycle disorders accumulate excessive ammonia in their blood, which may cause irreversible brain damage, disability, and seizures, and may be fatal. These disorders occur across all age groups, from infants to adults, and mild symptoms may go unnoticed until a stressor — such as illness, protein consumption, or environmental stress — overwhelms compensatory functions, resulting in an acute metabolic crisis. No approved, disease-modifying therapeutics exist for the most prevalent forms of UCD, leaving patients, and their clinicians, with few tools other than nitrogen scavengers, strict diet, lifestyle constraints, and hyper-vigilant monitoring with supportive care during crises. Mutations in genes encoding urea cycle enzymes result in insufficient levels of these important proteins. CMP-CPS-001 targets carbamoyl phosphate synthetase 1 (CPS1), a key enzyme that catalyzes the first step of the urea cycle. CMP-CPS-001 is designed to amplify CPS1 mRNA to potentially improve or restore urea cycle activity. The Phase 1 study is a randomized, double-blind, and placebo-controlled study designed to evaluate the safety, tolerability, and pharmacokinetics of CMP-CPS-001 in healthy volunteers. The study is currently active in Australia and anticipates enrolling a total of 96 participants across single- and multiple-ascending dose cohorts. For more information about the Phase 1 clinical study of CMP-CPS-001, please visit clinicaltrials.gov (NCT06247670). CMP-CPS-001 is an antisense oligonucleotide (ASO) designed to amplify CPS1 mRNA by harnessing fundamental cellular gene expression control mechanisms. CAMP4’s proprietary RAP Platform enabled the discovery of CMP-CPS-001, a potential new therapeutic to treat urea cycle disorders. 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 design of optimal chemistry to generate potent therapeutic candidates to address hundreds of genetic diseases across multiple tissues. Learn more about us at www.CAMP4tx.com and follow us @CAMP4tx. The content above comes from the network. if any infringement, please contact us to modify.