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.”

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Philipp Spycher

Promis­ing bet­ter link­er tech to ADC field, Araris has 'very, very am­bi­tious' plans for the clin­ic

A couple months after raising CHF 2.5 million ($2.76 million) in initial seed funding, one-year-old Araris Biotech is topping off the round with another CHF 12.7 million ($14 million).

The Paul Scherrer Institute and ETH Zurich spinout now has CHF 15.2 million to work with, and CEO Philipp Spycher has big plans. He hopes to bring one of the company’s antibody-drug conjugates (ADC) to the clinic by late 2022 or early 2023. “It’s very, very ambitious, but we are very optimistic that we actually can make it,” he said.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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David Hung (file photo)

Mas­ter deal­mak­er David Hung re­tools a SPAC sedan in­to a fi­nanc­ing mus­cle ve­hi­cle that leaves his can­cer start­up with $850M and a place on Wall Street

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

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Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)

Pfiz­er is on the verge of claim­ing a multi­bil­lion-dol­lar first-mover ad­van­tage with their Covid-19 vac­cine — an­a­lyst

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerink’s Geoff Porges has been running the numbers on Pfizer’s vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

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CEO Grace Colón (InCarda)

Look­ing to re­pur­pose an old drug to treat ir­reg­u­lar heart­beats, In­Car­da rais­es $30M in first Se­ries C close

A little less than two years after completing its $42 million Series B round, InCarda has returned to the venture well.

The San Francisco-based biotech announced the first portion of its Series C on Wednesday, pulling in $30 million in new funding. Most of the money will give enough runway for InCarda’s InRhythm program, an inhaled therapeutic aiming to treat sudden episodes of irregular heartbeats, through its Phase II trials and prepare it for Phase III.

UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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Steve Chen, Cellis Therapeutics president and CMO (Cellics)

UC San Diego spin­out award­ed up to $15M for nanosponge de­signed to soak up sep­sis-caus­ing tox­ins

CARB-X, a global partnership looking to spur the development of new antibacterial drugs, is awarding Cellics Therapeutics $3.94 million to do what president and CMO Steve Chen calls “looking at traditional drug development upside down.”

Instead of going after a target directly — in this case bacterial toxins and inflammatory cytokines that cause sepsis — Cellics researchers “flip it around” to examine the host cells being attacked. The UC San Diego spinout then creates what it calls “nanosponges” — nanoparticles cloaked in the fragments of macrophage cell membranes. Chen says the “sponges” are designed to trap the sepsis-causing endotoxins and cytokines on their cell membranes, neutralizing them.