In­scrip­ta push­es fund­ing to $260 mil­lion as they launch genome edit­ing plat­form

In­scrip­ta pre­sen­ta­tions can be­gin with the ad­vent of agri­cul­ture. Or even fur­ther back: The emer­gence of man.

Nan­di­ni Kr­ish­na­murthy

“We’ve come a long way, start­ing with nat­ur­al se­lec­tion,” In­scrip­ta mi­cro­bial di­rec­tor Nan­di­ni Kr­ish­na­murthy told a ses­sion this year at Syn­Bio­Be­ta, the new an­nu­al con­fer­ence for the syn­thet­ic bi­ol­o­gy field.

Be­hind her was a slide that’s re­curred in com­pa­ny pre­sen­ta­tions, show­ing from left to right across the bot­tom the clas­sic evo­lu­tion-of-man chart (the ‘hu­mor­ous’ kind that ends on an over­weight so­da-drinker), a pic­ture show­ing the de­vel­op­ment of corn from thin strand to bul­bous Iowan, and then a squig­gly pro­tein close-up of “di­rect­ed evo­lu­tion.” Be­low that runs an ar­row and a tick­er of how long each takes, from 10^9 years to 1.

“The ques­tion is how do you go from pre­cise­ly en­gi­neer­ing pro­teins to pre­cise­ly en­gi­neer­ing path­ways and genomes to har­ness the pow­er of bi­ol­o­gy?” she said.

Kevin Ness

It’s the kind of grand rhetoric and am­bi­tion you’ve come to ex­pect from a syn­thet­ic bio field that fea­tures Gingko Bioworks and a com­pa­ny billing it­self as the “Ne­spres­so ma­chine for DNA syn­the­sis.” In­scrip­ta keynot­ed the 2019 con­fer­ence to launch their Onyx plat­form for genome en­gi­neer­ing, and to­day they’ve an­nounced $125 mil­lion in Se­ries D fund­ing to com­mer­cial­ize that plat­form. It brings their to­tal fund­ing to $260 mil­lion. The pitch is sim­ple: Do for CRISPR what lap­tops and phones have done for su­per­com­put­ers.

“We’ve de­moc­ra­tized ac­cess to the foundry,” CEO Kevin Ness told End­points News, re­fer­ring to the mi­crotiter plates of­ten used for DNA-edit­ed cell lines. “We’ve cre­at­ed the equiv­a­lent of a desk­top com­put­er.”

The foundries are groups like Gingko and Zymer­gen that of­ten use their large fa­cil­i­ties to ed­it or­gan­isms for a ser­vice fee. In­scrip­ta is try­ing to put much of that sys­tem in the hands of re­searchers, a key cog they think will help un­leash emer­gent “bioe­con­o­my:” a world where genomes are edit­ed as much as they are read. The price, though, is not in­signif­i­cant: Around $350,000 to­tal.

In­scrip­ta first made a name when they un­veiled MAD7, an al­ter­na­tive CRISPR en­zyme to CAS9 and then gave it away for free to all re­searchers. They pitched it as an al­tru­is­tic ways of break­ing the li­cens­ing fees around CAS9 and help­ing move the en­tire field for­ward, but it was good busi­ness: In elim­i­nat­ing one bar to genome edit­ing, they widened the po­ten­tial mar­ket for their re­al prod­uct: Onyx. It was a sim­i­lar strat­e­gy to the one em­ployed Il­lu­mi­na – the genome read­ing com­pa­ny In­scrip­ta specif­i­cal­ly mar­kets them­selves around.

“We do for genome edit­ing what Il­lu­mi­na did for genome read­ing,” Ness said.

Ness has ex­pe­ri­ence on the genome-read­ing side, work­ing ear­ly in his ca­reer on PCR and lat­er co-found­ing 10x Ge­nomics, and In­scrip­ta em­ploys mul­ti­ple Il­lu­mi­na alum­ni, in­clud­ing their co-founder and first CEO: John Stuelp­nagel.

So how does it work?

A bi­ol­o­gist with the plat­form can se­lect the genes they want to knock out from In­scrip­ta’s com­put­er in­ter­face. In­scrip­ta then cre­ates the agents to knock out those genes at their man­u­fac­tur­ing fa­cil­i­ty and sends them to the bi­ol­o­gist’s lab. The bi­ol­o­gist ap­plies those agents to the cell lines, cre­at­ing thou­sands of dif­fer­ent lines that are “bar-cod­ed.” The bi­ol­o­gist can run what­ev­er ex­per­i­ment — ex­pos­ing them to a drug, say, or an agent — and the In­scrip­ta tech can see which cell lines sur­vived.

So far the plat­form is on­ly avail­able for E. Coli and S. cere­visi­ae and the com­pa­ny’s ex­po­sure out­side syn­thet­ic bi­ol­o­gy and bac­te­ria spaces has been lim­it­ed, but In­scrip­ta has found a big pro­po­nent in MIT’s Jim Collins. Collins has used the plat­form to cre­ate bac­te­ria cell lines with 5,000 dif­fer­ent gene ed­its. He then used those to test which genes con­ferred re­sis­tance.

It al­lowed us “to ex­plore the ge­net­ic de­pen­den­cies of an­tibi­ot­ic func­tion in un­prece­dent­ed de­tail.” Collins told GEN in Oc­to­ber.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.