Jeffrey Goldberg

Tout­ing CD161 as a test case for sin­gle-cell tech, Long­wood-backed Im­mu­ni­tas bags $58M for next-gen I/O work

Back when Long­wood un­veiled its lat­est im­munother­a­py start­up, Im­mu­ni­tas, the VC’s co-founder Lea Hachi­gian talked at length about how its sin­gle-cell ge­nomics analy­sis plat­form could en­able re­searchers to “ask ques­tions of the im­mune sys­tem we weren’t able to ask be­fore,” un­der­stand hu­man bi­ol­o­gy on a much deep­er lev­el and iden­ti­fy tar­gets that no­body else was work­ing on. They just couldn’t dis­close the pre­cise tar­get for the lead pro­gram.

Kai Wucherpfen­nig

This Feb­ru­ary, Im­mu­ni­tas fi­nal­ly did — pub­lish­ing a pa­per in Cell that ze­roes in on CD161 as a re­cep­tor on T and NK cells that sup­press­es their abil­i­ty to kill glioma cells.

Months lat­er, the start­up is back with $58 mil­lion in fresh cash to steer a CD161-block­ing an­ti­body to­ward the clin­ic while fu­el­ing a slate of oth­er pro­grams be­hind it. The goal, Im­mu­ni­tas CEO Jef­frey Gold­berg told End­points News, is to keep ex­pand­ing the pipeline at a rate of about one pro­gram per year.

Kai Wucherpfen­nig, chair of Dana-Far­ber Can­cer In­sti­tute’s de­part­ment of can­cer im­munol­o­gy and vi­rol­o­gy, co-found­ed Im­mu­ni­tas with Aviv Regev (be­fore Roche’s Genen­tech re­cruit­ed her to run R&D), Dane Wit­trup of MIT and Mass Gen­er­al’s Mario Su­và.

For Wucherpfen­nig, the dis­cov­ery of CD161 was a tes­ta­ment to the key ad­van­tages of the sin­gle-cell plat­form he helped in­vent, which start­ed out an­a­lyz­ing hun­dreds of cells but can now work with hun­dreds of thou­sands at a time, at high res­o­lu­tion. Through iso­lat­ing T cells from pa­tients’ tu­mor sam­ples (sep­a­rate from, say, stro­mal cells, tu­mor cells, and so on), clus­ter­ing them by gene ex­pres­sion and then ex­am­in­ing dif­fer­ent pop­u­la­tions, his team es­sen­tial­ly came up with an at­las of im­mune cells in glioma — all based on hu­man sam­ples.

“We’ve been asked mul­ti­ple times, why do oth­er peo­ple not pick it up? And the rea­son ac­tu­al­ly is that the bi­ol­o­gy is very dif­fer­ent be­tween mice and hu­mans,” he said. “Most peo­ple start with mouse mod­els, and then in mice there are ac­tu­al­ly mul­ti­ple genes that en­code CD161 like mol­e­cules. Some of them are in­hibito­ry, some just are ac­ti­vat­ing re­cep­tors, and the bi­ol­o­gy is very con­fus­ing. And in hu­mans it’s ac­tu­al­ly more straight­for­ward. There’s a sin­gle gene that en­codes an in­hibito­ry re­cep­tor, OK. And so most peo­ple have looked at this — I know some peo­ple have looked at this and said, nev­er mind. And they nev­er re­al­ly looked at the hu­man bi­ol­o­gy.”

As Im­mu­ni­tas — now grown to 20-plus staffers — car­ried on the work, once again re­ly­ing on the sin­gle-cell se­quenc­ing and an­a­lyt­ics tech, they found out that the re­cep­tor is not just as­so­ci­at­ed with glioma. CD161 ap­pears to be in play in T and NK cell in­ter­ac­tions with mul­ti­ple can­cer types span­ning sol­id and liq­uid tu­mors, giv­ing the biotech mul­ti­ple op­tions to ex­plore in the first hu­man tri­als, with an IND slat­ed for the first half of next year.

Specif­i­cal­ly, Im­mu­ni­tas’ IMT-009 is de­signed to bind to CD161 on T and NK cells and pre­vent it from in­ter­act­ing with CLEC2D on tu­mor cells, there­by restor­ing the im­mune cells’ can­cer-killing pow­er.

Wucherpfen­nig and Gold­berg fig­ure there are many more im­muno-on­col­o­gy tar­gets out there sim­i­lar­ly lurk­ing in cor­ners tra­di­tion­al dis­cov­ery meth­ods can’t un­cov­er.

The promise has drawn a siz­able syn­di­cate. Agent Cap­i­tal led the round, which fea­tured Med­ical Ex­cel­lence Cap­i­tal, 120 Cap­i­tal, So­las­ta Ven­tures, Mi­rae As­set, Ono Ven­ture In­vest­ment, The Mark Foun­da­tion for Can­cer Re­search, NS In­vest­ment, Bright­Edge (Amer­i­can Can­cer So­ci­ety) and the Leukemia & Lym­phoma So­ci­ety Ther­a­py Ac­cel­er­a­tion Pro­gram. Ex­ist­ing back­ers al­so re­turned: They are Alexan­dria Ven­ture In­vest­ments, Evotec, Leaps by Bay­er, M Ven­tures, No­var­tis Ven­ture Fund and, of course, found­ing in­vestor Long­wood.

Be­yond that, the sci­en­tist al­so sees the up­com­ing work as part of a big­ger ex­per­i­ment to test the un­der­ly­ing plat­form as a com­pre­hen­sive tool to close the loop on trans­la­tion.

“We’re al­so think­ing about how we can use sin­gle cell tech­nolo­gies to ac­tu­al­ly un­der­stand what the drug does in pa­tients — you know, once we start a clin­i­cal tri­al,” he said. “There ob­vi­ous­ly are some lo­gis­ti­cal chal­lenges with sam­ple pro­cure­ment, et cetera, but it could ac­tu­al­ly be an ex­cit­ing op­por­tu­ni­ty. So we — we use sin­gle cell tech­nolo­gies to dis­cov­er it, we use sin­gle cell tech­nolo­gies to de­fine in­di­ca­tions, and then al­so study mech­a­nism of ac­tion in peo­ple.”

Why it Works: Man­u­fac­tur­ing a Vac­cine in a Mul­ti-Prod­uct Fa­cil­i­ty.

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Merck CEO Rob Davis

Mer­ck emerges as lead bid­der in po­ten­tial Ac­celeron buy­out with deal pos­si­ble this week — re­port

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

Alexander Lefterov/Endpoints News

The coro­n­avirus vac­cine that the world for­got could still help save it

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

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Habib Dable, Acceleron CEO

Days of heat­ed ru­mors cul­mi­nate in a re­port that Ac­celeron is in ad­vanced buy­out talks

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

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Brian Hubbard, Anji Pharmacetuticals CEO

Look­ing to rewrite the rules of drug li­cens­ing, start­up An­ji is on the hunt for 'dy­nam­ic eq­ui­ty' joint ven­tures

Licensing is one of the most common ways big drugmakers leverage biotech innovation to drive gains across their pipelines — and the structure of those deals is pretty well established. But one biotech with home bases in China and the US thinks it may have a better way.

On Tuesday, Cambridge-based biotech Anji Pharma closed a $70 million Series B with two late-stage molecules in the fold and a mission to rewrite the rules of drug licensing through what it calls “dynamic equity” deals and a joint venture-heavy game plan. The round was funded in whole by Chinese hedge fund CR Capital.

Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

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Albert Bourla, Pfizer CEO (John Thys, Pool via AP Images)

Covid-19 roundup: Pfiz­er/BioN­Tech sub­mit vac­cine da­ta to FDA for younger chil­dren; Doc­tors kept pre­scrib­ing hy­drox­y­chloro­quine

Pfizer and BioNTech said Tuesday they submitted to FDA positive data from a Phase II/III trial of their Covid-19 vaccine in children aged 5 to less than 12 years old.

A formal EUA submission for the vaccine in these children is expected to follow “in the coming weeks,” the companies said in a statement.

The trial of 2,268 healthy participants aged 5 to less than 12 years old showed the vaccine was safe and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses, which is one-third the dose that’s administered to adults.

From left to right: Mark Springel, Kristina Wang, Lin Ao, Soufiane Aboulhouda

George Church, his stu­dents, and top VCs go na­tion­wide with a biotech train­ing camp

One night last fall, Floris Engelhardt sat down in her Boston apartment and logged onto a Zoom call, armed with a comic and a vague idea about starting a biotech.

Engelhardt was joining a student-run “match night.” A postdoc at MIT’s Bathe BioNanoLab, where researchers use DNA and RNA like Lego blocks for nanometer-sized structures, Engelhardt wanted to find real-world applications for her work. She sketched out — literally — a plan to use DNA origami, a decade-old technique for precisely folding DNA, to make therapies.

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Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi calls it quits on mR­NA Covid-19 shots, scrap­ping vac­cine from $3.2B Trans­late Bio buy­out

Sanofi is throwing in the towel on mRNA-based Covid-19 vaccines.

The French drugmaker will halt development on its unmodified mRNA Covid-19 shot despite what it said were positive Phase I/II results, a spokesperson told Endpoints News on Tuesday morning. Sanofi said the reason it’s stopping the Covid-19 mRNA program, developed in partnership with its new $3.2 billion acquisition Translate Bio, is because the market is too crowded.