Nessan Bermingham (file photo)

An­i­mal da­ta in hand, Nes­san Berming­ham banks $91.5M for At­las-backed RNA edit­ing play

Ko­r­ro Bio, the RNA edit­ing start­up that got start­ed on $4 mil­lion in seed fund­ing from At­las and some more cash from New En­ter­prise As­so­ci­ates, has brought in $91.5 mil­lion for its Se­ries A haul.

Un­der ex­ec­u­tive chair­man Nes­san Berming­ham, the biotech has racked up an­i­mal da­ta that he said show “pret­ty high tar­get­ed edit­ing straight out of the gate.” Off-tar­get ac­tiv­i­ty wasn’t an is­sue, and the OPERA plat­form ap­peared able to ze­ro in on mul­ti­ple types of RNA.

Now is the time to be­gin build­ing the team in earnest — and pave the path to the clin­ic, where Berming­ham hopes to ar­rive “well in ad­vance of” the fund­ing’s three-years-plus run­way.

“We val­i­dat­ed the tech­nol­o­gy, we val­i­dat­ed the ap­proach,” he told End­points News, “and now let’s take the time to be thought­ful as we think about the ap­pli­ca­tion from the clin­i­cal stand­point.”

That means pick­ing a lead can­di­date in a tar­get in­di­ca­tion — he’s not ready to di­vulge which — that fol­lows clear prece­dents in terms of clin­i­cal strat­e­gy, pa­tient strat­i­fi­ca­tion and end­points. But it is the sec­ond and third back­up pro­grams that will of­fer a win­dow to the new ther­a­peu­tic par­a­digm that OPERA opens up.

Josh Rosen­thal

At the core of the ap­proach is an en­doge­nous en­zyme fam­i­ly known as adeno­sine deam­i­nase act­ing on RNA, or ADAR. Co-founder Josh Rosen­thal elu­ci­dat­ed the mech­a­nism in squid and oc­to­pus through his work at the Ma­rine Bi­o­log­i­cal Lab­o­ra­to­ry, us­ing oligonu­cleotide guides to re­cruit these en­zymes to a spe­cif­ic site where they would con­vert an A to a G. That kind of change can make a world of dif­fer­ence for dis­or­ders like Rett syn­drome, Berming­ham not­ed.

Bor­row­ing heav­i­ly from es­tab­lished re­search on oligonu­cleotides, Ko­r­ro still lists the liv­er, eye and the cen­tral ner­vous sys­tem as its ini­tial tar­get tis­sues. In the CNS, in par­tic­u­lar, a va­ri­ety of play­ers have been show­ing that they can go be­yond the spinal cord and cor­tex to deep­er brain re­gions.

Mus­cle, car­dio­vas­cu­lar and oth­er ar­eas are al­so in sight down the road.

It’s a com­ple­men­tary tool for the ge­net­ic med­i­cine tool­box, to use an anal­o­gy Berming­ham is fond of, as the de­liv­ery sys­tem might be able to avoid the safe­ty chal­lenges faced by gene ther­a­py de­vel­op­ers such as Sol­id and Au­dentes — not to men­tion the cost of goods and scal­a­bil­i­ty.

“I think it fur­ther ex­em­pli­fies or fur­ther high­lights the im­por­tance of try­ing to co-opt en­doge­nous sys­tems in cells that are al­ready there ver­sus hav­ing to de­liv­er or look­ing to de­liv­er much larg­er pay­loads in­to these cells,” he said.

There’s much work left to prove it. Hav­ing tak­en space at 1 Kendall Square, Ko­r­ro plans to dou­ble the head­count to 40 or 50 (per­haps in­clud­ing a CEO) by the end of the year.

Wu Cap­i­tal led the new fi­nanc­ing along­side new in­vestors: Qim­ing Ven­ture Part­ners USA, Sur­vey­or Cap­i­tal, Cor­morant As­set Man­age­ment, MP Health­care Ven­ture Man­age­ment and Alexan­dria Ven­ture In­vest­ments.

At­las re­turned for the round, as did NEA, which al­so in­vest­ed in Shape Ther­a­peu­tics — a ri­val RNA edit­ing com­pa­ny that had its com­ing out par­ty last No­vem­ber, one month af­ter Ko­r­ro did.

“We ac­tu­al­ly came af­ter Shape, so they’d al­ready com­mit­ted to in­vest,” Berming­ham said. “Be­ing up­front about it, I think any tech­nol­o­gy and any area re­al­ly does need mul­ti­ple par­tic­i­pants in there. […] We’re on­ly bet­ter for that be­cause no one com­pa­ny frankly can do it all.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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An Endpoints Zoom meeting; and the email header employees will see if your company is a Premium subscriber

What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Fast on Glax­o­SmithK­line's heels, Au­rinia wins OK to steer a sec­ond lu­pus nephri­tis drug straight to the mar­ket

GlaxoSmithKline’s Benlysta isn’t alone in the small circle of approved lupus nephritis drugs anymore.

Little Aurinia Pharmaceuticals has gotten the green light from the FDA to start marketing its first and only program, voclosporin, under the brand name Lupkynis — something CEO Peter Greenleaf says it’s been ready to do since December.

Regulators went right down to the wire on the decision, keeping the company and the entire salesforce it’s already assembled on its toes.

Charlie Fuchs, Roche and Genentech global head of product development for oncology and hematology (Yale Cancer Center)

Yale can­cer spe­cial­ist Char­lie Fuchs tapped as new glob­al de­vel­op­ment chief for Roche/Genen­tech

Roche and their big sub Genentech have just recruited a top cancer specialist at Yale to head up global product development in oncology and hematology.

I just got word that the pharma giant, which leads one of the most active cancer research operations in the world, recruited Charlie Fuchs, director of the Yale Cancer Center and physician-in-chief of Smilow Cancer Hospital. He’ll join the global operation March 1 and will be based in South San Francisco, where Genentech is based.

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Jonathan Weissman (MIT)

Can a new CRISPR tech­nique un­lock the se­crets of how can­cer spreads?

Jonathan Weissman’s team watched the cancer cells spread across the doomed mouse. Engineered with a bioluminescent enzyme, they appeared in scans first as a small navy blue diamond lodged near the heart; a week later, as a triangle splayed across the mouse’s upper body, with streaks of green and two distinct bright red hubs of activity. By day 54, the mouse resembled a lava lamp.

The images would have been familiar to any cancer biologist, but they didn’t actually tell you much about what was going on: why the cancer was metastasizing or which cells were responsible. For that, Weissman’s team had designed a new tool. Inside the original navy blue diamond, they had engineered the microbiological equivalent of an airplane’s black box — a “molecular recorder” that, after the mouse’s death, could allow them to extract the cells and wind back intimate footage of a single cancer’s ascent.

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Kimberly Smith, ViiV R&D chief (ViiV Healthcare)

Af­ter sting­ing FDA set­back, Glax­o­SmithK­line's Vi­iV fi­nal­ly notch­es US ap­proval for long-act­ing HIV in­jec­tion

GlaxoSmithKline’s HIV unit ViiV was dealt a stinging loss back in late 2019 when the FDA slammed the brakes on its application for a once-monthly injection based on manufacturing issues. Now, with that roadblock in the rearview, ViiV has finally made good on its promise to change the HIV game.

The FDA on Thursday approved ViiV’s Cabenuva (cabotegravir and rilpivirine) as a long acting, once-monthly therapy for HIV-positive adults who are virologically suppressed and on a stable antiviral regimen.