Drug­ging RNA has be­come a hot bio­phar­ma top­ic in re­cent years, par­tic­u­lar­ly af­ter the mas­sive suc­cess­es of the Covid-19 vac­cines. But one biotech is aim­ing to take things a step fur­ther, uti­liz­ing the sin­gle-strand­ed mol­e­cules be­fore they even be­come RNA.

Cep­tur Ther­a­peu­tics launched Wednes­day morn­ing with a $75 mil­lion Se­ries A, the com­pa­ny an­nounced. The goal is to push for­ward a slate of oligonu­cleotide-based ther­a­pies that can con­trol gene ex­pres­sion, and even si­lence some tar­get­ed genes, at the “pre-mR­NA lev­el,” co-founder and CEO Pe­ter Ghoroghchi­an told End­points News.

The biotech has re­cruit­ed a blue-chip syn­di­cate as well, with ven­Bio and Qim­ing Ven­tures co-lead­ing the round. In ad­di­tion, Cep­tur re­ceived new sup­port from Per­cep­tive Xon­toge­ny Ven­ture Fund, Bris­tol My­ers Squibb and Janus Hen­der­son In­vestors.

Cep­tur’s main idea cen­ters around a pro­tein com­plex known as the U1 snRNP (pro­nounced SNURP), which Ghoroghchi­an said serves as a bi­o­log­i­cal gate­keep­er when DNA is tran­scribed in­to RNA and RNA in­to pro­teins. When cells go through this process, they’re con­stant­ly splic­ing ge­net­ic se­quences in and out, with some left out of the fi­nal mR­NA.

“The splic­ing ma­chin­ery that helps mR­NA to ma­ture is re­cruit­ed by the U1 snRNP, and the U1 snRNP al­so helps to turn off and con­trol the lev­el of tran­scripts, so how much of a giv­en mR­NA is made,” Ghoroghchi­an said. “It does this for every sin­gle gene in the body, so it has this very cen­tral role in bi­ol­o­gy.”

To take ad­van­tage of these gate­ways and cre­ate drugs, re­searchers led by Rut­gers pro­fes­sor Sam Gun­der­son, an­oth­er Cep­tur co-founder, de­vel­oped bi­va­lent oligonu­cleotides called U1 Adap­tors. The adap­tors tar­get spe­cif­ic ge­net­ic se­quences and re­cruit the U1 snRNP to de­stroy the de­sired pre-mR­NA, es­sen­tial­ly con­trol­ling which genes make it in­to the fi­nal pro­teins.

It’s an ap­proach sim­i­lar to an­ti­sense oligonu­cleotides and siR­NA ther­a­pies, Ghoroghchi­an said, on­ly Cep­tur aims to drug the RNA at a much more gran­u­lar lev­el. And be­cause the U1 snRNP is so ubiq­ui­tous through­out the body — Ghoroghchi­an says there are more than one mil­lion copies in every cell — Cep­tur be­lieves dif­fer­ent cell types and genes won’t im­pede the com­pa­ny’s progress.

Col­in Walsh

Col­in Walsh, a part­ner at Qim­ing Ven­tures, not­ed the RNA drug­ging field has boomed in re­cent years, ever since the idea took off in the ear­ly days of Io­n­is and Al­ny­lam. But he be­lieves Cep­tur rep­re­sents a big step for­ward in how such ap­proach­es will shape the space in the fu­ture, par­tic­u­lar­ly once it moves past the gene si­lenc­ing ap­pli­ca­tion.

“There is a large ap­petite for ways to con­trol pro­tein func­tion at the RNA lev­el,” Walsh said. “The U1 Adap­tor pro­tein com­plex is re­al­ly a mas­ter reg­u­la­tor of the tran­scrip­tome, it’s splic­ing and oth­er things. Though knock­down is the first place where you can take this, we see this as a re­al­ly broad po­ten­tial plat­form for mod­u­lat­ing RNA us­ing oli­go-based ther­a­peu­tics.”

For its in-house pipeline, Cep­tur isn’t re­veal­ing much just yet. Ghoroghchi­an said the team has a few can­di­dates that could hit IND-en­abling stud­ies some­time in 2023, in­clud­ing on­col­o­gy pro­grams tar­get­ing KRAS and MYC in can­cer, as well as one for Hunt­ing­ton’s dis­ease. Past that, though, every­thing re­mains in the dis­cov­ery phase.

And though the biotech is try­ing to con­trol ge­net­ic se­quenc­ing, Ghoroghchi­an be­lieves Cep­tur’s drugs will prove far safer than gene ther­a­pies that some­times con­tin­ue edit­ing DNA be­yond the ther­a­peu­tic use. Be­cause Cep­tur isn’t en­gag­ing in DNA or base edit­ing, its drugs will on­ly work as long as the adap­tor is in the pa­tient’s sys­tem.

Cep­tur’s oligonu­cleotides are ex­treme­ly small, Ghoroghchi­an said, and “every nu­cleotide is chem­i­cal­ly mod­i­fied. And we can use a lot of ex­ist­ing and val­i­dat­ed chemistries to di­al in how long it lasts, ei­ther to how long they can knock the gene down, or how quick­ly it can be de­grad­ed, in or­der to di­al in the safe­ty as­pect.”

On top of the new in­vestors, Cep­tur al­so re­ceived Se­ries A sup­port from ex­ist­ing seed in­vestors Affin­i­ty As­set Ad­vi­sors, Box­er Cap­i­tal and LifeSci Ven­ture Part­ners.

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.