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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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FDA ad­comm nar­row­ly votes in fa­vor of Mer­ck­'s an­tivi­ral for out­pa­tient Covid-19

With little explanation for why Merck’s potential Covid-19 antiviral was less effective in reducing Covid hospitalizations and deaths in a full analysis of a Phase III trial versus an interim look, the FDA’s antimicrobial drugs advisory committee on Tuesday voted 13-10 in favor of the pill’s benefits outweighing the risks for adults within 5 days of developing Covid symptoms.

Molnupiravir will likely be authorized by FDA in the coming days for adults with mild or moderate Covid-19. While Pfizer’s antiviral may prove to be more effective, Merck’s pill will be another weapon in the armamentarium of Covid-19 treatments for countries around the world, adding to the mAb treatments already in use in the outpatient space from Regeneron, Eli Lilly and Vir/GlaxoSmithKline.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Mar­ket­ingRx roundup: Ab­b­Vie’s Hu­mi­ra TV turns fo­cus to HS skin con­di­tion; Sanofi amps par­ent­ing pol­i­cy

After years as the top spending pharma TV advertiser, AbbVie’s Humira brand finally downshifted earlier this year, ceding much of its marketing budget to up-and-coming sibling meds Skyrizi and Rinvoq. However, now Humira is back on TV with ads for another condition — Hidradenitis suppurativa (HS).

The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.