Bristol Myers, VCs launch new RNA therapeutics biotech Ceptur with $75M to silence pre-mRNA

Ceptur Therapeutics wants to challenge existing drug technologies by going after pre-mRNA to evade competition for engagement and gene silencing. The Philadelphia biotech picked up $75 million in series A funds for its Wednesday launch. 

With technology in-licensed from Rutgers University, Ceptur's U1 adaptor is used to silence mRNA before further splicing and maturation of the transcript, said Peter Ghoroghchian, Ph.D., M.D., co-founder, CEO and president, in an interview.

Qiming Venture Partners USA and venBio Partners co-led the series A (PDF), which included financing from Bristol Myers Squibb, Perceptive Xontogeny Venture, Janus Henderson and others.

Since pre-mRNA exists in the nucleus, there's an advantage to silencing at this stage because the treatment wouldn't run into other mRNA-binding proteins in the cytoplasm, he said. That means potential for more molecular targets than small interfering RNA or anti-sense oligonucleotides. Hence, "less competition for engagement and silencing," the CEO added.

The goal of the biotech's oligos, or synthetic RNA, technology is to edit specific sequences of a given target gene's mRNA, Ghoroghchian said. Ceptur looks to drug targets outside the liver. Gene silencing in the liver and central nervous system has "come of age" through the likes of Alnylam and Ionis, but developing RNA therapeutics outside those organs and the eye is "still in its relative infancy," the CEO said.

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The series A will bankroll more discovery work to select two development candidates and test them in studies next year that set the biotech up for clinical trials, Ghoroghchian said. The CEO was previously senior vice president and head of therapeutic development at Repertoire Immune Medicines and prior to that chief technology officer at Poseida Therapeutics.

Ceptur's pipeline spans oncology, the central nervous system, nephrology and immunology. In the preclinical stage are assets for KRAS-driven solid tumors, diffuse large B-cell lymphoma, multiple myeloma and Huntington's disease. Beyond those, in the discovery stage, are assets targeting amyotrophic lateral sclerosis, chronic kidney disease and psoriasis, among other diseases.

The biotech, with a biology team in Philadelphia and a chemistry team in Denmark, is looking at two main delivery routes for its treatments. FDA-approved drugs like Biogen's Spinraza for neuromuscular disorder spinal muscular atrophy have set a precedent for delivering "naked oligonucleotides" via an injection into the spinal canal, Ghoroghchian said. Ceptur is also working on its own in-house delivery systems including antibody-oligo conjugates. 

Since Ceptur's oligos don't require an enzyme as part of the drug's effect on the body, there could be potential to avoid the two-step delivery needed by gene editing technologies, the CEO said. 

"Because our molecular effector is present in every cell in the body at such high concentrations, we are excited about evoking RNA editing using a one delivery [method], really the oligo only," he said.