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.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.