Gene-edit­ing up­start lays out a $100M IPO with a plan to quick­ly leapfrog the lead­ers in their field

CRISPR/Cas9. TAL­EN. Zinc fin­ger nu­cle­ase tech. The ARC nu­cle­ase.

You may have heard about those first 3 gene-edit­ing plat­forms. But what’s an ARC nu­cle­ase?

Matthew Kane

AR­CUS was put to­geth­er by a group of sci­en­tists in North Car­oli­na who have been mak­ing the pitch that they have a bet­ter way to ac­com­plish the DNA hack­ing pop­u­lar­ized 5 years ago by the orig­i­nal trio of star­tups: CRISPR Ther­a­peu­tics, Ed­i­tas, In­tel­lia. Those biotechs are just now get­ting in­to the clin­ic, with Pre­ci­sion Bio­Sciences com­ing in right be­hind with its own new­ly filed IND. They’re fo­cused on a gene-edit­ed al­lo­gene­ic (off the shelf) CAR-T cell pro­gram tar­get­ing CD19 (not for the first time) which they plan on launch­ing soon, with a Phase I/IIa clin­i­cal tri­al in pa­tients with acute lym­phoblas­tic leukemia and non-hodgkin lym­phoma. 

AR­CUS be­longs to the start­up Pre­ci­sion Bio­Sciences, which on Fri­day filed for an IPO, pen­cilling in $100 mil­lion as the tar­get.

Their claim to fame rests on a one-step en­gi­neer­ing process, which they are sell­ing as a sim­pler, more ef­fec­tive way of com­plet­ing the gene edit­ing process that will trans­late well to a less ex­pen­sive mass pro­duc­tion ap­proach. 

The sim­ple ex­pla­na­tion is that Pre­ci­sion Bio be­lieves it has a bet­ter sur­gi­cal tool — the ARC nu­cle­ase — for slic­ing in­to a spe­cif­ic DNA se­quence need­ed to cor­rect a dis­ease.

Jeff Smith

This ARC nu­cle­ase, they say, is “a ful­ly syn­thet­ic en­zyme sim­i­lar to a hom­ing en­donu­cle­ase but sig­nif­i­cant­ly im­proved to be the start­ing point for the genome-edit­ing plat­form.” It’s small, they claim, with “in­com­pa­ra­ble” speci­fici­ty that can be cus­tomized to hit the right tar­get in just the right way to im­prove po­ten­cy.

Every one of the pi­o­neers has a sim­i­lar claim to the best tech. CRISPR $CR­SP and In­tel­lia $NT­LA are Cas9 spe­cial­ists, pop­u­lar­iz­ing a new tool cre­at­ed by Jen­nifer Doud­na and Em­manuelle Char­p­en­tier that’s known for be­ing cheap and easy to use. This tech has spread like wild­fire in aca­d­e­m­ic labs. Ed­i­tas $ED­IT is us­ing a new-and-im­proved ver­sion of Cas9. Cel­lec­tis $CLLS CEO An­dré Chouli­ka is diplo­mat­ic about it, but he’s pas­sion­ate about TAL­EN, which he helped cre­ate. Sang­amo, which on­ly re­cent­ly of­fered its first hu­man da­ta, was off tar­get on the da­ta but hap­py about the ef­fect it was see­ing in hu­mans.

All the pi­o­neers have seen their shares beat up over the past year. But then, that’s true for a lot of pub­lic biotechs.

Derek Jantz

The whole field, which has at­tract­ed large in­vest­ments, is pri­mar­i­ly based on non-hu­man pri­mate da­ta. But it’s at a cross­roads, with much more hu­man da­ta on the near hori­zon. The win­ners will be rich­ly re­ward­ed. The losers will face the scrap heap.

The Durham, NC-based biotech with close con­nec­tions to Duke rolled out a $110 mil­lion mega round last sum­mer from a laun­dry list of back­ers that in­clud­ed Gilead. And as we said at the time, it had every ear­mark of a clas­sic crossover round point­ed straight at the $100 mil­lion IPO you’re read­ing about now.

David Thomp­son

Ar­row­Mark Part­ners led the deal and was joined by oth­er new in­vestors: Franklin Tem­ple­ton In­vest­ments, Cowen Health­care In­vest­ments, Brace Phar­ma Cap­i­tal, Pon­tif­ax AgTech, OCV Part­ners, Adage Cap­i­tal Man­age­ment, Cor­morant As­set Man­age­ment, Vi­vo Cap­i­tal, Alexan­dria Ven­ture In­vest­ments, Ridge­back Cap­i­tal, Agent Cap­i­tal, and en­ti­ties af­fil­i­at­ed with Leerink Part­ners. Ex­ist­ing in­vestors ven­Bio, F-Prime, RA Cap­i­tal Man­age­ment, Am­gen

Ven­tures, Os­age Uni­ver­si­ty Part­ners, DU­MAC, and the Longevi­ty Fund al­so par­tic­i­pat­ed in the fi­nanc­ing.

Gilead fol­lowed up with a $445 mil­lion pact with Pre­ci­sion in the fall, fo­cused on he­pati­tis B. And then gene edit­ing ex­perts at the Uni­ver­si­ty of Penn­syl­va­nia stepped up with a sci­en­tif­ic col­lab­o­ra­tion. They split off their ag ops just ahead of the new round last year.

Abid Ansari

The top 3 ex­ecs haven’t ex­act­ly short­changed them­selves on in­come. CEO Matthew Kane took home a com­pen­sa­tion pack­age worth $1.6 mil­lion for last year. CFO Abid Ansari snagged $1.4 mil­lion and David Thomp­son, the chief de­vel­op­ment of­fi­cer, got $1.8 mil­lion — all big mon­ey in the start­up world. They al­so got rais­es for their base salary, now at $523,000 for Kane, who al­so has 5.6% of the stock, which will be worth mil­lions if the IPO comes in as they hope.

Jeff Smith — a co-founder and CTO out of Duke Uni­ver­si­ty — has a wedge of 10% of the eq­ui­ty, which puts him up with the two top in­vestors: ven­Bio at 11% and F-Prime at 9.7%. The oth­er sci­en­tif­ic co-founder is Derek Jantz, whose bio in­cludes a ci­ta­tion for ear­ly work de­vel­op­ing the zinc fin­ger tech.

Biogen CEO Michel Vounatsos (via Getty Images)

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The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

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The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Overnight for­tunes are be­ing made in biotech these days — and it's both en­cour­ag­ing and more than a lit­tle bit scary

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That leaves newly-named CEO Sean Bohen holding a batch of 1,110,896 shares with a strike price of $4.82. As of Tuesday morning, the stock is now trading at $53.40, giving him a portfolio value of $53.4 million. Not bad for someone who was hired in September.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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