Car­olyn Bertozzi (Illustration: Assistant editor Kathy Wong for Endpoints News)

Car­olyn Bertozzi, re­peat biotech founder and launch­er of a field, shares in chem­istry No­bel win

Car­olyn Bertozzi, pre­dict­ed by some to be­come a No­bel lau­re­ate, clinched one of the world’s top awards in the wee hours of Wednes­day, win­ning the No­bel Prize in Chem­istry along­side a re­peat win­ner and a Copen­hagen re­searcher.

The Stan­ford pro­fes­sor, Morten Mel­dal of Uni­ver­si­ty of Copen­hagen and 2001 awardee K. Bar­ry Sharp­less of Scripps shared the prize equal­ly. The No­bel is some­times split in quar­ters and/or halves.

“I am ab­solute­ly stunned. I am sit­ting here and can hard­ly breathe,” Bertozzi said to the com­mit­tee in the mid­dle of the night Cal­i­for­nia time. “I’m still not en­tire­ly pos­i­tive that it’s re­al, but it’s get­ting re­al by the minute.”

The in-the-mo­ment re­ac­tion was re­flect­ed sim­i­lar­ly in her in­ter­nal com­mu­ni­ca­tion with her lab group. The sub­ject line: “I can’t be­lieve it’s re­al.” The body of the note: “Ap­par­ent­ly it is!”

Sharp­less and Mel­dal won for their con­tri­bu­tions to so-called click chem­istry. Bertozzi el­e­vat­ed the find­ings by find­ing click re­ac­tions that weren’t tox­ic to cells and made “chem­i­cal re­ac­tions that don’t in­ter­fere with the nor­mal bio­chem­istry of life. This now al­lows us to at­tach all kinds of mol­e­cules to bi­o­log­i­cal ones,” the Roy­al Swedish Acad­e­my of Sci­ences said in its an­nounce­ment.

Bertozzi, the new­ly-mint­ed No­bel lau­re­ate, spear­head­ed the field of bioorthog­o­nal chem­istry to help map cells. She joins just sev­en oth­er women grant­ed the award — out of 191 lau­re­ates — in­clud­ing CRISPR pi­o­neers Jen­nifer Doud­na and Em­manuelle Char­p­en­tier for their ground­break­ing Cas9 gene edit­ing work that has el­e­vat­ed both to new heights and made Doud­na a house­hold name in some cir­cles.

Sharp­less joins a lim­it­ed club of re­peat win­ners: John Bardeen (’56, ’72), Marie Curie (’03, ’11), Li­nus Paul­ing (’54, ’62) and Fred­er­ick Sanger (’58, ’80). The No­bel com­mit­tee deemed Sharp­less the orig­i­na­tor of the “click chem­istry” con­cept, which they de­scribed as “sim­ple and re­li­able chem­istry” that al­low re­ac­tions to hap­pen fast and avoid un­de­sired byprod­ucts.

He and Mel­dal in­de­pen­dent­ly put to­geth­er the “crown jew­el” of click: the cop­per cat­alyzed azide-alkyne cy­cload­di­tion. The chem­i­cal re­ac­tion plays out in the de­vel­op­ment of drugs, map­ping DNA and cre­at­ing new ma­te­ri­als, the No­bel com­mit­tee wrote.

The No­bel Com­mit­tee for Chem­istry an­nounces the win­ners: Car­olyn Bertozzi, Morten Mel­dal and K. Bar­ry Sharp­less (Jonathan Nack­strand/AFP via Get­ty Im­ages)

Click on the im­age to see the full-sized ver­sion

In comes Bertozzi, who es­ca­lat­ed the work of Mel­dal and Sharp­less by map­ping gly­cans, bio­mol­e­cules on the sur­face of cells. Her de­vel­op­ments, in­clud­ing a met­al-free click re­ac­tion, work in­side hu­mans. The re­search is at the heart of new treat­ments be­ing test­ed in clin­i­cal tri­als to­day.

In sum­ma­ry, the trio’s find­ings have “tak­en chem­istry in­to the era of func­tion­al­ism.” On the press and com­mit­tee call right af­ter the an­nounce­ment, Bertozzi said bioorthog­o­nal and click chem­istry are “still in its ear­ly phas­es” and that the im­pacts will be seen in biotech and phar­ma for years to come.

Bertozzi’s work has led to the foun­da­tion of nine biotechs (a fig­ure she tal­lied up pri­or to an in­ter­view with End­points News in May), in­clud­ing Palleon Phar­ma­ceu­ti­cals, In­ter­Venn, Ly­cia Ther­a­peu­tics and oth­ers. The lat­ter came to­geth­er out of a 24-hour hum­drum of ven­ture cap­i­tal in­ter­est af­ter she tweet­ed out a preprint her group had post­ed on Chem­Rx­iv that de­scribed LY­TACs, she pre­vi­ous­ly re­called to End­points.

Asked by re­porters what ap­pli­ca­tions of her she thinks are mak­ing a lot of im­pact, she said in med­i­cine, the main one is the de­liv­ery of ther­a­peu­tics “to make sure that drugs go to the right place and stay away from the wrong place.”

Bertozzi di­rects Stan­ford’s Sarafan ChEM-H, short for Chem­istry, En­gi­neer­ing, and Med­i­cine for Hu­man Health. She in­flu­ences the next gen­er­a­tion as a teacher but al­so as a par­ent who helps with her chil­dren’s sci­ence fair projects. Mu­si­cal in­ter­ests pop­u­late her free time as she en­joys play­ing pop, rock n’ roll and jazz songs on the pi­ano, and oc­ca­sion­al­ly “keep[s] up with karaoke nights at some of the clubs in San Fran­cis­co.” She’s al­so a self-pro­claimed “fit­ness buff.”

The Howard Hugh­es Med­ical In­sti­tute in­ves­ti­ga­tor has al­so co-found­ed Grace Sci­ence, a biotech that is at­tempt­ing to trans­late re­search out of the Grace Sci­ence Foun­da­tion to cre­ate treat­ments for NG­LY1 de­fi­cien­cy and oth­er in­di­ca­tions. Bertozzi has served on the Eli Lil­ly board of di­rec­tors and re­search ad­vi­so­ry board at GSK.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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