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.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 113,500+ biopharma pros reading Endpoints daily — and it's free.

Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 113,500+ biopharma pros reading Endpoints daily — and it's free.

Jeffrey Bluestone, Sonoma CEO (Photo credit: Steve Babuljak)

Jeff Blue­stone just raised $265M to de­vel­op cu­ra­tive cell ther­a­pies. We asked him how

Jeff Bluestone had some big goals in mind when he decided to make a switch from a decades-long career in academia and non-profit research to a biotech startup CEO. And now — 18 months after the $40 million launch party — he has a whole lot more money on hand to pay for the considerable amount of work ahead at Sonoma Biotherapeutics.

This morning Bluestone is taking the wraps off a $265 million B round after boosting the core syndicate of A-list investors he started with. Even by today’s standards, that sum dwarfs the kind of $100 million-plus megarounds that have become standard fare in biotech over the last 2 years.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 113,500+ biopharma pros reading Endpoints daily — and it's free.