Thomas Perlmann (far right), Secretary of the Nobel Assembly, announces the 2020 Nobel laureates in Physiology or Medicine to (left on screen) Harvey Alter, Michael Houghton, center, and Charles Rice (Claudio Bresciani/TT via AP Images)

Three sci­en­tists win No­bel Prize in Med­i­cine for work in dis­cov­er­ing he­pati­tis C virus

Mon­day marks the start of No­bel Prize week, and the Swedish com­mit­tee kicked things off by hand­ing out the first award to three vi­rol­o­gists.

Har­vey Al­ter, Michael Houghton and Charles Rice have joint­ly won the No­bel Prize in Phys­i­ol­o­gy or Med­i­cine for their work on the dis­cov­ery of the he­pati­tis C virus. Their re­search pro­vid­ed the foun­da­tion for test­ing and an­tivi­rals that can help those in­fect­ed re­ceive treat­ment, as well as a po­ten­tial vac­cine for the liv­er dis­ease.

“For the first time in his­to­ry, the dis­ease can now be cured, rais­ing hopes of erad­i­cat­ing He­pati­tis C virus from the world pop­u­la­tion,” the No­bel com­mit­tee said in a state­ment.

Al­ter, the el­der states­man of the group, be­gan his work in the he­pati­tis field back in the 1960s and 1970s, around the time when the he­pati­tis B virus was first dis­cov­ered. Work­ing at the NIH, he had been study­ing the oc­cur­rence of the dis­ease in pa­tients who had re­ceived blood trans­fu­sions when he no­ticed that not all of the cas­es could be ex­plained by the new­ly iden­ti­fied virus.

Near­ly a decade lat­er, Houghton and his co-work­ers at the phar­ma­ceu­ti­cal com­pa­ny Ch­i­ron iso­lat­ed the ge­net­ic se­quence of the virus through ex­per­i­ments with chim­panzees. By study­ing the blood of the in­fect­ed an­i­mals, Houghton was able to pre­dict which of the DNA frag­ments with­in the genome came from the chim­panzee and which came from the virus.

The Ch­i­ron team was then able to clone the vi­ral DNA. While search­ing for an­ti­body re­spons­es, Houghton dis­cov­ered an RNA virus de­rived from the Fla­vivirus fam­i­ly that was ul­ti­mate­ly de­ter­mined to be he­pati­tis C.

Houghton’s work proved piv­otal, but one ma­jor ques­tion re­mained — could the virus alone cause he­pati­tis C? Rice, then a re­searcher at Wash­ing­ton Uni­ver­si­ty in St. Louis, be­gan in­ves­ti­gat­ing if the cloned virus could repli­cate and cause dis­ease. He de­ter­mined that a pre­vi­ous­ly un­char­ac­ter­ized re­gion in the virus genome could be in­flu­en­tial in repli­ca­tion, and en­gi­neered an RNA vari­ant to de­ter­mine this re­gion’s ef­fect.

Af­ter in­ject­ing the vari­ant in­to chim­panzees, Rice de­tect­ed the virus in their blood, con­firm­ing that he­pati­tis C could cause dis­ease and repli­cate on its own.

Al­ter con­tin­ues to work at the NIH as the chief of in­fec­tious dis­ease at its Clin­i­cal Cen­ter. Houghton, the on­ly non-Amer­i­can in the group be­ing from the UK, is cur­rent­ly a pro­fes­sor at the Uni­ver­si­ty of Al­ber­ta in Cana­da. Rice now works at Rock­e­feller Uni­ver­si­ty in New York.

The No­bel Com­mit­tee drew a par­al­lel be­tween the work in he­pati­tis C and re­search be­ing cur­rent­ly un­der­tak­en to try to iden­ti­fy and cure the coro­n­avirus at the cen­ter of the Covid-19 pan­dem­ic, per an As­so­ci­at­ed Press re­port. Com­mit­tee mem­ber Pa­trik Ern­fors called the dis­cov­ery of a virus a “crit­i­cal mo­ment.”

“The first thing you need to do is to iden­ti­fy the caus­ing virus,” he told re­porters. “And once that has been done, that is in it­self the start­ing point for de­vel­op­ment of drugs to treat the dis­ease and al­so to de­vel­op vac­cines against the dis­or­der.”

Ac­cord­ing to num­bers from the WHO, there are over 70 mil­lion cas­es of he­pati­tis C around the world with ap­prox­i­mate­ly 400,000 deaths per year.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Charlie Fuchs, Roche and Genentech global head of product development for oncology and hematology (Yale Cancer Center)

Yale can­cer spe­cial­ist Char­lie Fuchs tapped as new glob­al de­vel­op­ment chief for Roche/Genen­tech

Roche and their big sub Genentech have just recruited a top cancer specialist at Yale to head up global product development in oncology and hematology.

I just got word that the pharma giant, which leads one of the most active cancer research operations in the world, recruited Charlie Fuchs, director of the Yale Cancer Center and physician-in-chief of Smilow Cancer Hospital. He’ll join the global operation March 1 and will be based in South San Francisco, where Genentech is based.

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Jonathan Weissman (MIT)

Can a new CRISPR tech­nique un­lock the se­crets of how can­cer spreads?

Jonathan Weissman’s team watched the cancer cells spread across the doomed mouse. Engineered with a bioluminescent enzyme, they appeared in scans first as a small navy blue diamond lodged near the heart; a week later, as a triangle splayed across the mouse’s upper body, with streaks of green and two distinct bright red hubs of activity. By day 54, the mouse resembled a lava lamp.

The images would have been familiar to any cancer biologist, but they didn’t actually tell you much about what was going on: why the cancer was metastasizing or which cells were responsible. For that, Weissman’s team had designed a new tool. Inside the original navy blue diamond, they had engineered the microbiological equivalent of an airplane’s black box — a “molecular recorder” that, after the mouse’s death, could allow them to extract the cells and wind back intimate footage of a single cancer’s ascent.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Deval Patrick (Mary Altaffer/AP Images)

De­val Patrick joins Cerev­el board, fur­ther in­ter­twin­ing com­pa­ny with Bain Cap­i­tal

Tony Coles’ team at Cerevel Therapeutics is adding two high-profile board members, including an ex-governor that has lots of connections to the Boston area where the biotech is based.

Former Massachusetts Gov. Deval Patrick is hopping on Cerevel’s board of directors, the company announced Thursday, joining less than three months after Cerevel went public on the backs of Perceptive’s ARYA II SPAC. And in a twist, Pfizer’s new business development chief Deborah Baron is joining the board as well, about three years after the Big Pharma shuttered the neuroscience pipeline that Cerevel is seeking to revive.

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Fast on Glax­o­SmithK­line's heels, Au­rinia wins OK to steer a sec­ond lu­pus nephri­tis drug straight to the mar­ket

GlaxoSmithKline’s Benlysta isn’t alone in the small circle of approved lupus nephritis drugs anymore.

Little Aurinia Pharmaceuticals has gotten the green light from the FDA to start marketing its first and only program, voclosporin, under the brand name Lupkynis — something CEO Peter Greenleaf says it’s been ready to do since December.

Regulators went right down to the wire on the decision, keeping the company and the entire salesforce it’s already assembled on its toes.