On this Rare Dis­ease Day, let's all com­mit to help­ing rec­og­nize the life and work of biotech gi­ant Hen­ri A. Ter­meer

Last May the Cam­bridge com­mu­ni­ty and en­tire bio­phar­ma in­dus­try suf­fered a tremen­dous loss with the un­ex­pect­ed pass­ing of Hen­ri A. Ter­meer. Hen­ri was a for­mer chair­man, pres­i­dent and CEO of Gen­zyme Cor­po­ra­tion for near­ly three decades pri­or to its ac­qui­si­tion by the French drug mak­er Sanofi. Re­tir­ing from Gen­zyme in 2011, af­ter his 28-year tenure, Hen­ri led the com­pa­ny’s growth from a small start-up of 20 to 12,000 em­ploy­ees glob­al­ly serv­ing pa­tients in more than 90 coun­tries all while es­tab­lish­ing Mass­a­chu­setts as the mec­ca of biotech. He was known for his ser­vice to the rare dis­ease com­mu­ni­ty and his un­sur­passed en­tre­pre­neur­ial lead­er­ship that spurred the rise of an in­dus­try ded­i­cat­ed to in­no­v­a­tive treat­ments for or­phan dis­eases. Hen­ri set a stan­dard, al­ways putting pa­tients first, and he forged the path for build­ing a sus­tain­able rare dis­ease busi­ness, with many – in­clud­ing Al­ny­lam – fol­low­ing his foot­steps.  He was a men­tor, a col­league, and a friend.

John Maraganore

To hon­or Hen­ri’s lega­cy and cel­e­brate his many con­tri­bu­tions to the thriv­ing in­dus­try that he helped build, a num­ber of in­dus­try, aca­d­e­m­ic and com­mu­ni­ty lead­ers have come to­geth­er to form the Hen­ri A. Ter­meer Trib­ute Com­mit­tee, which we have the priv­i­lege of co-chair­ing, work­ing close­ly with Be­lin­da and Adri­ana Ter­meer.

The fo­cus of every­thing Hen­ri did was to al­ways do what’s right for pa­tients. Fit­ting­ly, to­day on Rare Dis­ease Day, a time ded­i­cat­ed to rais­ing aware­ness for rare dis­eases and im­prov­ing ac­cess to treat­ment and med­ical rep­re­sen­ta­tion for in­di­vid­u­als with rare dis­eases and their fam­i­lies, we will be re-nam­ing a square in his hon­or as the “Hen­ri A. Ter­meer Square,” lo­cat­ed just op­po­site from the Gen­zyme Cen­ter in Kendall Square, Cam­bridge. To­day would have al­so been Hen­ri’s 72nd birth­day.

Robert Cough­lin

We have al­so com­mis­sioned renowned sculp­tor Pablo Ed­uar­do to cre­ate a life-size sculp­ture of Hen­ri to serve as a last­ing re­mem­brance of all he did for pa­tients, next gen­er­a­tion lead­ers and the com­mu­ni­ty – the sculp­ture will be in­stalled on the square in 2019.

Our cur­rent ef­forts and fu­ture ini­tia­tives fo­cused on the de­vel­op­ment of our biotech com­mu­ni­ty and on the men­tor­ship of fu­ture lead­ers are in­tend­ed to con­tin­ue Hen­ri’s re­mark­able lega­cy.

To learn more about these ef­forts, please vis­it ter­meer­tribute.org.

— John Maraganore and Robert Cough­lin


John Maraganore is the CEO of Al­ny­lam Phar­ma­ceu­ti­cals. Robert Cough­lin is the pres­i­dent and CEO of Mass­Bio.


Im­age: Feb. 16, 2011: Hen­ri Ter­meer, chief ex­ec­u­tive of­fi­cer of Gen­zyme, lis­tens dur­ing a news con­fer­ence an­nounc­ing the deal where France’s Sanofi-Aven­tis agreed to buy Ter­meer’s com­pa­ny, end­ing a nine-month pur­suit of Gen­zyme. Bloomberg via Get­ty Im­ages

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Am­gen puts its foot down in shiny new South San Fran­cis­co hub as it re­or­ga­nizes R&D ops

Amgen has signed up to be AbbVie’s neighbor in South San Francisco as it moves into a nine-story R&D facility in the booming biotech hub.

The arrangement gives Amgen 240,000 square feet of space on the Gateway of Pacific Campus, just a few minutes drive from its current digs at Oyster Point. The new hub will open in 2022 and house the big biotech’s Bay Area employees working on cardiometabolic, inflammation and oncology research.

Ab­b­Vie, Scripps ex­pand part­ner­ship, for­ti­fy fo­cus on can­cer drugs

Scripps and AbbVie go way back. Research conducted in the lab of Scripps scientist Richard Lerner led to the discovery of Humira. The antibody, approved by the FDA in 2002 and sold by AbbVie, went on to become the world’s bestselling treatment. In 2018, the drugmaker and the non-profit organization signed a pact focused on developing cancer treatments — and now, the scope of that partnership has broadened to encompass a range of diseases, including immunological and neurological conditions.

South Ko­rea jails 3 Sam­sung ex­ecs for de­stroy­ing ev­i­dence in Bi­o­Log­ics probe

Three Samsung executives in Korea are going to jail.

The convictions came in what prosecutors had billed as “biggest crime of evidence destruction in the history of South Korea”: a case of alleged corporate intrigue that was thrown open when investigators found what was hidden beneath the floor of a Samsung BioLogics plant. Eight employees in total were found guilty of evidence tampering and the three executives were each sentenced to up to two years in prison.

Nick Plugis, Avak Kahvejian, Cristina Rondinone, Milind Kamkolkar and Chad Nusbaum. (Cellarity)

Cel­lar­i­ty, Flag­ship's $50M bet on net­work bi­ol­o­gy, mar­ries ma­chine learn­ing and sin­gle-cell tech for drug dis­cov­ery

Cellarity started with a simple — but far from easy — idea that Avak Kahvejian and his team were floating around at Flagship Pioneering: to digitally encode a cell.

As he and his senior associate Nick Plugis dug deeper into the concept, they found that most of the models others have developed take a bottom-up approach, where they assemble the molecules inside cells and the connections between them from scratch. What if they opt for a top-down approach, aided by single-cell transcriptomics and machine learning, to gauge the behavior of the entire cellular network?

Sanofi’s big week in­cludes a promis­ing PhI­II for an or­phan dis­ease drug, with plans for a pitch to the FDA

The biopharma R&D food chain is paying off with a plan at Sanofi to pitch regulators on a new drug for an orphan disease called cold agglutinin disease.

The pharma giant ushered out a statement Tuesday morning — after it spelled out plans to radically restructure the company, abandoning cardio and diabetes research altogether — saying that their C1s inhibitor sutimlimab had cleared the pivotal study.