Ceptur Therapeutics wants to challenge current drug technologies by pursuing pre-mRNA to evade competition for participation and gene silencing. The Philadelphia biotech company raised $75 million in Series A funds for its Wednesday launch.
With technology licensed from Rutgers University, Ceptur’s U1 transducer is used to silence mRNA prior to further splicing and maturation of the transcript, Peter Gorgochian, PhD, MD, co-founder, CEO, and president, said in an interview.
Qiming Venture Partners USA and venBio Partners co-led Series A (PDF), which included funding from Bristol-Myers Squibb, Perceptive Xontogeny Venture, Janus Henderson and others.
Since pre-mRNA is present in the nucleus, there is an advantage to silencing at this point because the treatment will not bump into other mRNA-binding proteins in the cytoplasm, he said. This means that more molecular targets can be achieved by small interfering RNA or antisense oligonucleotides. The CEO added, therefore, “less competition for participation and silencing.”
The goal of the oligos, or synthetic RNA technology, is to modify specific mRNA sequences of the target gene, Gorogeshian said. Ceptur is looking at drug targets outside the liver. The CEO said gene silencing in the liver and central nervous system “has reached maturity” through the likes of Elnelam and Eunice, but that development of RNA therapies outside of those organs and the eye is “still in its relative infancy”.
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Gorogshian said Series A will fund further exploratory work to select two candidates for development and testing in next year’s studies that put the biotech to clinical trials. The CEO was previously Senior Vice President and Head of Clinical Development at Repertoire Immune Medicines and before that Chief Technology Officer at Poseida Therapeutics.
Ceptur’s pipeline extends to oncology, central nervous system, nephrology, and immunology. In the preclinical stage, there are origins for KRAS-driven solid tumors, diffuse B-cell lymphoma, multiple myeloma, and Huntington’s disease. Furthermore, in the discovery phase, there are assets targeting amyotrophic lateral sclerosis, chronic kidney disease, and psoriasis, among others.
The biotech, with a biology team in Philadelphia and a chemistry team in Denmark, is looking at two main ways to deliver its treatments. Gorogeshian said FDA-approved drugs such as Biogen’s Spinraza for the neuromuscular disorder and spinal muscular atrophy set a precedent for the delivery of “naked oligonucleotides” by injection into the spinal canal. Ceptur also acts on its own internal delivery systems including antibody-oligo conjugates.
The CEO said that because Ceptur’s oligos do not require an enzyme as part of the drug’s effect on the body, there may be potential to avoid the two-step delivery that gene-editing technologies need.
“Because the molecular effector is present in every cell in the body at such high concentrations, we are excited to evoke RNA editing using a single delivery. [method]Really, only a few.”