Contera Pharma A/S, a clinical-stage biotech company pioneering innovative treatments for neurological disorders, has announced the preclinical candidate nomination of CP0014753, a highly potent and selective first-in-class antisense oligonucleotide directed to modulate expression of one of the genes involved in development of Canavan disease. The therapeutic concept behind CP-102 aims to reverse toxic accumulation of NAA in the brain of Canavan disease patients, thereby slowing or stopping disease progression.
Canavan disease is a severe genetic disorder that affects young children, leading to neurological damage and a drastically shortened lifespan. With no approved treatments available, families are left without options to slow or stop disease progression. There is an urgent need for innovative and effective therapies to address this life-threatening condition. Advances in RNA-based therapeutics offer new hope for these children and their families.
“The nomination of CP0014753 as a preclinical candidate ready for IND-enabling studies marks an important milestone for Contera Pharma. We are very impressed by the preclinical data package that not only demonstrated the safety and efficacy of the CP0014753, but also established NAA as robust non-invasive biomarker. This positions CP0014753 strongly for clinical development and represents a promising step for patients and their families affected by this fatal disease. Furthermore, it strengthens our confidence in our pioneering RNA technology platform and the proprietary methods that allow us to precisely target and leverage the most druggable RNA regions for both small molecules and oligonucleotide-based RNA therapeutics“, says Søren Rasmussen, CSO of Contera Pharma.
Thomas Sager, CEO of Contera Pharma, adds: “The progress of the CP-102 project and specific nomination of CP0014753 to enter pre-IND studies is a significant milestone for Contera Pharma. The achievements underscore our ability to design and advance innovative RNA-modulating therapeutics with precision and efficiency, reinforcing our commitment to delivering transformative treatments for patients in need. We look forward to advancing the Canavan program and engaging with investors and pharma partners to drive CP0014753 further towards early clinical development for this devastating pediatric disorder”.
About Canavan disease
Canavan disease is a rare and fatal genetic disorder that primarily affects infants and young children. It is caused by mutations in the ASPA gene, leading to a deficiency of the enzyme aspartoacylase. This results in the toxic buildup of N-acetylaspartic acid (NAA) in the brain, causing progressive damage to the nervous system. Symptoms typically appear within the first few months of life and include poor muscle tone, developmental delays, seizures, difficulty swallowing, and vision impairment. Over time, children lose motor function and the ability to communicate, with most requiring full-time care. There are no approved treatments for Canavan disease, though supportive therapies such as physical therapy, feeding assistance, and seizure management help improve quality of life. Experimental gene and RNA-based therapies are currently being explored as potential breakthroughs to slow or halt disease progression.
About CP-102
CP-102 is an investigational therapy and is currently in preclinical development. Its safety and efficacy have not been established, and it is not approved by any regulatory authority.
About Contera Pharma A/S
Contera Pharma A/S is focused on developing novel treatments for people suffering from neurological disorders. Contera Pharma’s science is based on a precision medicine approach integrating external and internal data sources to support rational selection of human disease-validated drug discovery targets to identify novel pipeline opportunities within neurological disorders. Contera Pharma has full ownership and holds global rights to the CP-102 program. Contera Pharma is based in Hørsholm, Denmark.
For more information, visit www.conterapharma.com
