Rosana Kapeller listening to a member on the breakfast panel at #BIO19 discuss AI in R&D in Philadelphia (Jeff Rumans for Endpoints News)

Nim­bus founder Rosana Kapeller has a new com­pa­ny, with $50M and an eye on the ‘re­peatome’

Rosana Kapeller left Nim­bus two years ago de­ter­mined, af­ter 2 decades and 3 com­pa­nies, that her next spot would be as CEO. To­day, af­ter a 7-month sab­bat­i­cal and a stint at a top VC firm, the joc­u­lar com­pu­ta­tion­al bi­ol­o­gy pi­o­neer is back. And with full con­trol.

“I re­al­ly want­ed to… make a unique or­ga­ni­za­tion, a unique cul­ture,” Kapeller told End­points News. I want­ed that chal­lenge.”

And a chal­lenge it will be, both sci­en­tif­i­cal­ly and be­cause, well, there’s a pan­dem­ic keep­ing much of her 10-per­son team work­ing from her home. “This is a hard time to start a com­pa­ny,” she ac­knowl­edged.

Still, Kapeller is con­fi­dent, plac­ing her faith both in the am­bi­tious sci­ence of her new com­pa­ny and in her own ex­pe­ri­ence as a busi­ness­woman, dat­ing back to her up­bring­ing in a busi­ness-fo­cused fam­i­ly in Brazil. The new com­pa­ny is ROME Ther­a­peu­tics and it’s launch­ing out of stealth mode with $50 mil­lion in Se­ries A fund­ing from GV — where Kapeller served as en­tre­pre­neur-in-res­i­dence for the last year — ARCH Ven­tures, and Part­ners In­no­va­tion Fund. The biotech is one of a se­ries that have arisen in the past few years to tar­get parts of what was once deemed “junk DNA”: the 97-99% of ge­net­ic code that doesn’t code for pro­teins. Al­though sci­en­tists have known for decades now that at least parts of this vast nu­cle­ic flot­sam serve key func­tions, un­tan­gling those func­tions has been a ma­jor hur­dle. Drug­ging them has been an even larg­er one.

ROME will tar­get one seg­ment of this erst­while junk called the “re­peatome.” The name has yet to catch on — a PubMed search pro­duced 22 re­sults and the Wikipedia page is a sin­gle para­graph at­trib­uted to one PLOS pa­per out of France — but the field has slow­ly gained steam since a 2011 Sci­ence pa­per, on which ROME co-founder David Ting was lead au­thor.

The re­peatome refers to some 50% of hu­man DNA that is made up of se­quences that re­peat over and over again — like a mu­si­cal or lit­er­ary mo­tif — and that don’t make any pro­teins. Some of these, as sci­en­tists have long known, are retro­virus­es that in­fect­ed us and em­bed­ded their codes in our DNA over mil­lions of years of evo­lu­tions. Oth­er se­quences, though, are “virus-like,” said Ting. Most of the time these se­quences are blocked from do­ing any­thing, trapped by methyl agents or hi­s­tones that wrap like chains around DNA. But in some in­stances — such as some can­cers — dis­tressed cells take the chains off and trans­late the se­quences in­to RNA. Those RNA se­quences don’t make any pro­teins. But they look like RNA virus­es and ac­ti­vate the in­nate im­mune sys­tem as a virus would, call­ing it to at­tack a tu­mor.

“These re­peats are like the first re­spon­ders, telling the body these cells are be­com­ing un­con­trol­lable — con­trol it,” Kapeller said.

Two prob­lems can emerge. First, can­cer cells can de­vel­op ways of re­verse tran­scrib­ing these RNA se­quences back in­to their genome, both si­lenc­ing the im­mune sig­nals and adding to the can­cer’s ge­net­ic vari­abil­i­ty. In some au­toim­mune con­di­tions, the op­po­site prob­lem is at play, Kapeller said. Healthy cells send off these RNA re­peats, trig­ger­ing a dam­ag­ing im­mune re­sponse.

Kapeller talks about ROME’s role as restor­ing “yin and yang”: Keep­ing the im­mune sig­nals go­ing in can­cer and turn­ing them off in au­toim­mune dis­eases. ROME has not yet re­vealed how they plan on do­ing that, but when Ting first hy­poth­e­sized that can­cers were tran­scrib­ing these sig­nals, he start­ed a clin­i­cal tri­al with a com­mon HIV drug on 4th line colon can­cer pa­tients. The HIV drug, which is meant to stop the HIV virus from re­verse tran­scrib­ing it­self, ap­peared to in­hib­it the can­cer’s abil­i­ty to re­verse tran­scribe the RNA sig­nals. The pa­tients on the study main­tained their con­di­tion, a rar­i­ty for that form and stage of can­cer.

“Some­how can­cer has re­pur­posed this process to repli­cate and grow in­to tu­mor,” Ting told End­points, de­scrib­ing his dis­cov­ery. “It was kind of an ac­ci­dent, an ac­ci­dent of some­thing we were told was junk and find­ing the junk was ac­tu­al­ly do­ing some­thing.”

David Ting

Click on the im­age to see the full-sized ver­sion

That some­thing evad­ed sci­en­tists in part be­cause ear­ly DNA se­quencers lacked the abil­i­ty to pick up and an­a­lyze these vast codex­es of DNA and study which re­peats were be­ing tran­scribed in­to RNA. To do so, ROME will re­ly on two new but es­tab­lished tech­niques, se­quenc­ing of long stretch­es of DNA and se­quenc­ing of the RNA be­ing tran­scribed in­side a cell, along­side a ma­chine learn­ing ap­proach set up by the­o­ret­i­cal physi­cist Ben­jamin Green­baum. In part, that’s what made it a fit­ting project for Kapeller, who did com­pu­ta­tion­al work at Mil­len­ni­um and Ailleron and then was en­list­ed by At­las Ven­tures to launch Nim­bus, one of the first ma­jor com­pu­ta­tion­al biotechs.

Al­though it will be Kapeller’s first stint as a CEO, it will hard­ly be her first time in lead­er­ship. Nim­bus lacked a CEO for its first 4.5 years, she notes, leav­ing con­trol of the biotech be­tween her­self and the board. She says that she’s learned a few things from her ex­pe­ri­ence with past com­pa­nies: That con­trol and com­mand doesn’t work, trans­paren­cy is nec­es­sary, and di­ver­si­ty and putting women in man­age­ment mat­ters. She brings an MD-PhD’s un­der­stand­ing of pa­tients, she said, and she took some­thing be­sides busi­ness acu­men back from her home­town of Rio de Janeiro: a cer­tain warmth.

“I’m from Brazil and Brazil­ians love to use hu­mor a lot,” Kapel­lar said.

For in­stance, she led off the com­pa­ny’s first meet­ing with their PR firm with a slide of her­self and her co-founders in Ro­man glad­i­a­tor cos­tumes. She’s re­luc­tant to men­tion those jokes in in­ter­views, though, weary af­ter enough years as an ex­ec­u­tive of how it might be trans­lat­ed.

With the sci­ence, she’s bold­er. They’re one of the first jump­ing in­to a still-emerg­ing field, she said, and their goal is sus­tained re­mis­sion: can­cer and au­toim­mune treat­ments that won’t stop work­ing af­ter a few months, or years.

This is “com­plete­ly un­chart­ed ter­ri­to­ry,” Kapeller said.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Joel Dudley, new partner at Innovation Endeavors (Tempus Labs)

For­mer Google CEO’s VC is mak­ing a big­ger push in­to the biotech world, hir­ing promi­nent Ther­a­nos skep­tic

Venture capital firm Innovation Endeavors has mainly had its focus on investments across the tech space, but it has been slowly turning its attention to the biotech world. Now, a new partner is coming into the fold showing that its interest in biotech is likely to grow further.

The Silicon Valley-based company, which is headed up by former Google CEO Eric Schmidt, has brought on Joel Dudley as a partner. According to Dudley’s LinkedIn page, he is joining Innovation Endeavors after serving as the chief science officer of biotech startup Tempus Labs from 2020.

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Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.

Blaise Coleman, Endo International CEO

En­do files for Chap­ter 11 as it looks to fin­ish off its opi­oid lit­i­ga­tion

Irish drugmaker Endo International is entering into bankruptcy as it faces the weight of serious litigation related to its involvement in the opioid epidemic in the US.

The company has filed Chapter 11 proceedings in the US Bankruptcy Court for the Southern District of New York, with the company expected to file recognition proceedings in Canada, the UK and Australia. The company’s bankruptcy filing showed the company had assets and liabilities in the range of $1 billion to $10 billion.