CRISPR pi­o­neer Feng Zhang's up­start biotech seeks $100M-plus IPO for pre­clin­i­cal base edit­ing work

The CRISPR pi­o­neers’ new, gen­tler-touch com­pa­ny is aim­ing for a $100 mil­lion ini­tial pub­lic of­fer­ing.

Beam Ther­a­peu­tics, head­ed by for­mer Ed­i­tas founders and gene-edit­ing trail­blaz­ers David Liu, Feng Zhang, and J Kei­th Joung, de­buted last year, push­ing a soft­er, more flex­i­ble ver­sion of gene edit­ing, re­flec­tive of a larg­er trend in the biotech in­dus­try. The promise was that in­stead of hack­ing at base pairs like first-gen CRISPR does, Beam could in­stead mod­i­fy them in a far less dis­rup­tive way.

Un­sur­pris­ing for a com­pa­ny found­ed by sci­en­tists who helped re­shape ge­net­ic dis­ease med­i­cine, Beam has been well-fund­ed since its de­but. It emerged out of stealth mode with $87 mil­lion, pri­mar­i­ly from ARCH and F-Prime Cap­i­tal Part­ners, and then a $135 mil­lion Se­ries B from A round in­vestors plus Red­mile Group, Cor­morant As­set Man­age­ment, Al­ti­tude Life Sci­ence Ven­tures. It’s built in part off tech from Ed­i­tas and Liu’s lab at MIT.

ARCH and F-Prime are the biggest stock­hold­ers at 23.0% and 19.4%, re­spec­tive­ly, while sub­sidiaries of Hill­house Cap­i­tal and Temasek claim around 7% each. No­tably, founders Liu and Zhang kept 9.1% and 7.2% for them­selves. CEO John Evans owns 3.6% of the com­pa­ny.

CRISPR was rev­o­lu­tion­ary six years ago. It made widescale gene edit­ing pos­si­ble for the first time, of­fer­ing un­prece­dent­ed pre­ci­sion that al­lowed sci­en­tists to knock out one gene with­out hit­ting the rest.

The in­creas­ing­ly clear prob­lem, though, is that it func­tions as a very price nail re­mover. It can pull out the rusty or oth­er­wise prob­lem­at­ic nail you need gone, open­ing a slew of long-un­der­stood ge­net­ic dis­eases to po­ten­tial cures, but leaves ques­tions about the fall­out to the house those nails held up. And once out, you can’t put a nail back in.

Part of Beam’s an­swer to that ques­tion is the in­tu­itive one: It re­places the old nails with new ones; rather than just cut­ting out base pairs, it can splice in new ones, re­plac­ing in­di­vid­ual mu­tant nu­cleotides that give rise to dis­ease. It ac­com­plish­es this large­ly with ver­sions of base-edit­ing en­zyme called deam­i­nase.

Con­trast­ing with the con­ven­tion­al metaphor of CRISPR as “mol­e­c­u­lar scis­sors,” Beam likes to com­pare it to a pen­cil that can erase and rewrite genes. The cru­cial point is that the process doesn’t cre­ate the dou­ble-strand breaks in DNA that the first ver­sions of CRISPR tech­nol­o­gy do.

Beam is just one of a hand­ful of new biotechs now aim­ing to ac­com­plish CRISPR’s ini­tial goals with­out caus­ing these dou­ble-strand dis­rup­tions. An­oth­er Feng Zhang com­pa­ny, Ar­bor, is pi­o­neer­ing RNA — as op­posed to DNA — edit­ing, Flag­ship Pi­o­neer­ing’s new Omega Ther­a­peu­tics is work­ing on an in­no­v­a­tive epi­ge­net­ic mod­el, and Tru­Code is us­ing a syn­thet­ic com­pound called PNA to nat­u­ral­ly in­duce gene edit­ing.

Though still pre­clin­i­cal, Beam lists 10 dis­eases it’s aim­ing for, in­clud­ing long­time gene-edit­ing tar­gets be­ta tha­lassemia and sick­le cell dis­ease.

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.

Su­per-se­cre­tive an­ti-ag­ing biotech Cal­i­co tees up the first vis­i­ble clin­i­cal tri­al of an ex­per­i­men­tal drug. And it’s for can­cer?

Over the past 7 years, Calico has been so much more than your average, run-of-the-mill secretive biotech players. It’s a riddle, wrapped in a mystery, inside an enigma, to repurpose an old Winston Churchill line dating from the time he confronted the Iron Curtain surrounding Stalin’s thoughts.

Launched by industry legend Art Levinson of Genentech fame, with the infinitely deep pockets of Google for support, one of the few big headlines the anti-aging biotech has sparked focused on a major alliance with AbbVie — a giant outfit that conversely likes to show off its drug prospects whenever it can. Together, they’ve been focused on diseases that limit life span — quite an arc of ailments.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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IN8bio CEO William Ho (IN8bio)

Bring­ing their ge­net­i­cal­ly mod­i­fied gam­ma delta T cells to Nas­daq, IN8bio files for $86M IPO

The biotech IPO parade continues marching forward as 2020 turns toward the fourth quarter.

IN8bio, a New York-based company focused on genetically modified gamma delta T cell therapies, filed to go public Friday seeking an $86 million raise. The company has two clinical-stage candidates being studied in glioblastoma and leukemia, respectively.

By any stretch of the imagination, 2020 has already been a huge year for biotech, and nowhere does it appear more obvious than the vast amounts of companies hitting the public market.

Covid-19 roundup: Pars­ing Bourla, a top an­a­lyst sees im­proved chances for Pfiz­er vac­cine; Fau­ci: No sur­prise that Trump was hit by Covid-19

With a medley of adverse events hobbling the late-stage development of vaccines and drugs, Pfizer CEO Albert Bourla’s latest — extended — timeline for the mRNA approach they’re working on with BioNTech is giving some top analysts added confidence that the pharma giant can come up with the regulatory goods next month.

Parsing Bourla’s language in his comments last week, SVB Leerink’s Geoffrey Porges notes that Bourla’s decision to say they “may” be able to nail down the positive efficacy of their vaccine in a matter of days — a big change from his earlier certainty — may also indicate a delay on that to early November.

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News brief­ing: Ab­b­Vie and Roche's Ven­clex­ta scores an­oth­er FDA OK; Im­muno­Gen nabs Chi­na deal with $40M cash

AbbVie and Roche’s Venclexta has gotten a new FDA thumbs up.

The pair announced Monday that regulators have approved the drug in combination with azacitidine or low-dose cytarabine for newly-diagnosed acute myeloid leukemia in adults who are 75 or older or those who can’t undergo intensive chemotherapy. This follows the drug’s accelerated approval in 2018 and positive data from two Phase III confirmatory trials.

James Sabry (Roche)

Roche's James Sabry inks his sec­ond AI deal in back-to-back pacts — this time part­ner­ing Genen­tech with Stan­ford spin­out Gen­e­sis Ther­a­peu­tics

Less than a week after Roche joined forces with Dyno Therapeutics to develop gene therapies using artificial intelligence, its giant subsidiary Genentech is hopping on the AI bandwagon with a different player.

Genentech has inked a deal with Stanford spinout Genesis Therapeutics to harness its AI power for drug development and discovery. Genesis is getting an upfront payment and milestones, but the companies are keeping the details under wraps for now. The Burlingame, CA-based biotech also stands to earn future royalties on any approved Genentech drugs that come from the deal.

Joe Biden (Carolyn Kaster, AP Images)

What about the Ger­man ne­go­ti­a­tion mod­el? Biden steers drug pric­ing de­bate to a show­down

From an ill-fated proposal to ban rebates for pharmacy benefit managers to an executive order demanding a “most-favored-nation price” for Medicare, if nothing else President Donald Trump has introduced Americans to a flurry of ideas to rein in pharma, an industry he once accused of “getting away with murder.” And now we’re getting the first glimpse of what a Joe Biden presidency might mean for prescription drug pricing.

En­do pays $658M in a fur­ther bet on col­la­gen-based med­i­cines, buy­ing out long­time bio­phar­ma part­ner

A little less than two years after Endo Pharmaceuticals’ deal to purchase Somerset Therapeutics fell through, the Irish drugmaker is returning to the well with a much bigger acquisition.

Endo has agreed to buy BioSpecifics Technologies for a whopping $658 million, the two companies announced Monday, in the culmination of a research agreement signed all the way back in 2004. Endo will purchase all of BioSpecifics’ outstanding stock for about $540 million, valuing the company at $88.50 per share — a 45% premium on the $61.02 share price at which the company closed on Friday.