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.

Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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Sean Parker, AP

Sean Park­er helps cre­ate a CRISPRed cell ther­a­py 2.0 play — and he’s got a high-pro­file set of lead­ers on the team

You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. It’s another appealing Bay Area group that’s attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.

Today it’s Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenberg’s new venture investment operation based in LA.
Drazan — a J&J Innovation vet with a long record of entrepreneurial endeavors — exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasn’t long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.