Ceptur Launches U1 Adaptors with $75 Million Series A

Ceptur Therapeutics, based in Hillsborough, NJ, Philadelphia and Copenhagen, closed on a Series A financing round worth $75 million. The company’s Co-founder, President and Chief Executive Officer Dr. P. Peter Ghoroghchian, M.D., Ph.D.,, took time to discuss the company and the financing round with BioSpace.

Dr. Ghoroghchian co-founded Ceptur in 2021 along with Samuel Gunderson, Head Scientific Advisor and a Professor at Rutgers University, and Rafal Goraczniak, Director of Platform Development. The company was founded,  Ghoroghchian said, “to advance therapeutics based on U1 Adaptor technology. U1 Adaptors are small, synthetic RNA oligonucleotides that engage the U1 small nuclear ribonuclear protein (U1 snRNP), which is a ubiquitous intracellular RNA-protein complex that controls the expression levels and maturation of endogenous mRNA.”

Every cell in the body has this molecular machinery present at very high concentrations, Ghoroghchian said, with a conserved RNA sequence in every person and eukaryotic species on the planet. The U1 Adaptors give the U1 snRNP the ability to “engage the pre-mRNA of a specific target gene in order to regulate its expression levels or to edit its sequence, thereby correcting or compensating for its abnormal gene function.”

In other words, it is programmable and predictable and can be easily modified with well-validated oligonucleotide chemistries. The company believes it can generate U1 Adaptors, Ghoroghchian said, “with specific activity against different genes and cell types, to tune their pharmacology, and to modulate their potency and specificity in a rapid and cost-effective manner.”

The company is now focused on advancing its discovery pipeline of U1 Adaptor therapeutics. The focus is on diseases where changing the expression levels or splice isoforms of the pre-mRNA of specific genes might help treat a specific disease.

“In particular,” Ghoroghchian said, “the focus is in therapeutic areas wherein conventional in vivo gene silencing, genome editing or RNA editing technologies may have delivery or target engagement challenges that could be overcome with a U1 Adaptor therapeutic that engages the highly present and conserved U1 snRNP found within the diseased cells.”

The company website indicates it has preclinical compounds in oncology and central nervous system (CNS) conditions and discovery programs in nephrology and immunology.

The Series A was co-led by venBio Partners and Qiming Venture Partners USA. New investors who participated included Perceptive Xontogeny Venture (PXV) Fund, Bristol Myers Squibb and Janus Henderson Investors. Also investing were existing Seed investors Affinity Asset Advisors, Boxer Capital and LifeSci Venture Partners.

The funds raised from the Series A will be used for platform optimization, advance the Discovery pipeline, and expand operations in Philadelphia and Copenhagen.

With the financing, Dr. Aaron Royston, managing partner at venBio, and Colin Walsh, partner at Qjming Venture Partners, are joining Ceptur’s board of directors.

Walsh stated, “RNA-based drugs have already become an essential tool in our therapeutic arsenal; and, we strongly believe that this modality will continue to deliver meaningful new therapies for patients. Ceptur’s use of synthetic oligonucleotides that engage U1 snRNP offers the ability to co-opt this master regulator of the transcriptome to regulate mRNA in a highly targeted fashion.”

The company is also bringing on some more people to the company’s Scientific Advisory Board, including:

• Thomas Andresen, Chief Executive Officer of T-Cypher Bio

• Dennis Benjamin, former Senior Vice President of Research at Seagen

• Steven F. Dowdy, Professor of Cellular & Molecular Medicine at the UCSD School of Medicine

• Dr. Sridhar Ganesan, Associate Director for Translational Science, Chief of the Section of Molecular Oncology, and the co-Leader of the Clinical Investigations and Precision Therapeutics Program at the Rutgers Cancer Institute of New Jersey

• Adrian Krainer, St. Giles Professor at Cold Spring Harbor Laboratory and Deputy Director of Research at the CSHL Cancer Center

• Iain Mattaj, founding Director of Fondazione Human Technopole in Milan, Italy

• Henrik Oerum, C-Founder and Chief Science Officer of Civi BioPharma

• Thomas Tuschl, Professor of RNA Molecular Biology at Rockefeller University.

In a statement, Daniel Heller, general partner and chief investment officer at Affinity Asset Advisors, said, “In leading the Series Seed round, we identified early the potential of U1 Adaptor technology. Over the past year, we have worked closely with Peter and the Ceptur team and are delighted at the progress that has been made towards establishing the platform. In this financing round, we have significantly expanded upon our initial commitment and are inspired to partner with our new investor syndicate to advance U1 Adaptors for unmet patient needs.”