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.”

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

UK gov­ern­ment, pri­vate in­vestors dole out $340M+ to drug, di­ag­nos­tic man­u­fac­tur­ers

The government of the United Kingdom is giving out grants to several manufacturers that have a presence in England, Wales and Northern Ireland.

The government announced that four companies, including Ipsen, contract manufacturer Pharmaron, DNA manufacturer Touchlight and diagnostic test producer Randox, will receive a total of £277 million ($341.1 million). According to a release from the UK government, this represents the first portion of grants from the Life Sciences Innovative Manufacturing Fund.