An elite team of young, transpa­cif­ic sci­en­tists out of Har­vard and MIT starts plow­ing next-gen cell ther­a­py tech field

This isn’t the com­plete sto­ry about the lat­est biotech up­start to raise its head in the cell ther­a­py field. It’s not even half the sto­ry. But it is an in­trigu­ing glimpse — based large­ly on the peo­ple who are in­volved.

Xi Chen

Even be­fore the first CAR-Ts hit the mar­ket, just about every­one who was any­one in the field had set their sights on the next leg of the clin­i­cal jour­ney. Im­pres­sive as they were in the first piv­otal tri­als, cell ther­a­pies bris­tled with lim­i­ta­tions re­lat­ed to the kind of cells they re­lied on, their eas­i­ly ex­haust­ed state that blunt­ed dura­bil­i­ty and a lean se­lec­tion that would ben­e­fit from more pow­er­ful dos­ing.

Those are a few of the prob­lems, and a lit­tle start­up named Root­Path has set out to tack­le them. 

“We cant dis­close 100%,” says CEO Xi Chen, who was a post­doc fel­low at the Wyss In­sti­tute, in what would prove to be some­thing of an un­der­state­ment. Root­Path, he said, is still in stealth mode to a sig­nif­i­cant de­gree.

Le Cong

Chen and a group of sci­en­tists that in­cludes Le Cong — a high-pro­file young sci­en­tist who grad­u­at­ed from Ts­inghua Uni­ver­si­ty, played a mar­quee role in Feng Zhang’s CRISPR lab at The Broad and now works as an as­so­ciate pro­fes­sor at Stan­ford — start­ed Root­Path. The in­ner cir­cle in­cludes Yin­qing Li, new­ly in­stalled at Ts­inghua U af­ter get­ting his PhD at MIT, and found­ing sci­en­tist Ely Porter. Har­vard Med re­searcher and MIT grad Cheryl Cui is al­so on board.

Yin­qing Li

We know that they have some in­sights in­to con­quer­ing those hur­dles that re­main, like so many oth­er com­pa­nies, but there’s very lit­tle in­sight of­fered in­to ex­act­ly what they mean when they talk about work­ing in sin­gle-cell analy­sis and ma­nip­u­la­tion at a high-through­put lev­el.

Ely Porter

Their ob­jec­tives, though, have a wide fol­low­ing: Get­ting bet­ter T cell pop­u­la­tions to en­gi­neer as per­son­al­ized an­ti-can­cer ther­a­pies, with greater dura­bil­i­ty and much bet­ter speci­fici­ty. And let’s throw in eas­i­er to man­u­fac­ture, to en­sure a tight turn­around. The same could be said for a dozen or more ri­val re­search groups.

The group just gar­nered $7 mil­lion in seed cash to get go­ing, with Se­quoia Chi­na jump­ing in along­side Vol­canics Ven­ture,  BV  (Baidu  Ven­tures) and  Nest.Bio Ven­tures, which helped the group get start­ed last year. 

It’s not a lot to go on. But as the biotech in­dus­try in Chi­na, the US and Eu­rope con­tin­ues to spark new, glob­al ini­tia­tives like this, these ex­ecs have a chance to play a role in cre­at­ing the next chap­ter of the drug de­vel­op­ment in­dus­try.

Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

UP­DAT­ED: J&J's Xarel­to, Amar­in's Vas­cepa are cost-ef­fec­tive, not bud­get friend­ly — ICER

ICER, an increasingly influential cost-effectiveness watchdog in the United States, has concluded in its review of treatments for cardiovascular disease that while the cost of J&J’s Xarelto and Amarin’s Vascepa meet its benchmark for value pricing — the two treatments will not likely treat as many patients as hoped without surpassing the annual budget threshold calculated by ICER for each therapy.