RA Cap­i­tal dou­bles down on Sid­dhartha Mukher­jee's vi­sion for a new cell en­gi­neer­ing ap­proach, lead­ing Vor's $110M Se­ries B

Vor Bio­phar­ma is muscling up.

Robert Ang

CEO Robert Ang, who was re­luc­tant to di­vulge the head­count when dis­cussing his move from Neon Ther­a­peu­tics to Vor last Au­gust, read­i­ly of­fered that the team has grown from 6 to 50 in less than a year. The biotech is mov­ing to a larg­er of­fice on Cam­bridge Park­way Dri­ve in weeks, giv­ing it more space to com­plete the IND-en­abling work and man­u­fac­tur­ing scale-up — con­duct­ed by a CD­MO part­ner — in prepa­ra­tion for clin­i­cal tri­als planned for the first half of 2021.

It’s al­so hauled in $110 mil­lion from its Se­ries B, led by RA Cap­i­tal Man­age­ment and fea­tur­ing new in­vestors like Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, Pagli­u­ca Fam­i­ly Of­fice and Alexan­dria Ven­ture In­vest­ments. 5AM Ven­tures, which co-led the $42 mil­lion Se­ries A with RA Cap­i­tal, al­so joined along­side old-timers PureTech Health, John­son & John­son In­no­va­tion — JJDC and Os­age Uni­ver­si­ty Part­ners.

“Vor has an el­e­gant ap­proach to en­gi­neer­ing hematopoi­et­ic stem cells that we be­lieve is amongst the most promis­ing in­no­va­tions in on­col­o­gy,” Joshua Resnick, man­ag­ing di­rec­tor at RA Cap­i­tal, said in a state­ment.

Sid­dhartha Mukher­jee

Pi­o­neered by Co­lum­bia on­col­o­gist Sid­dhartha Mukher­jee (who has chron­i­cled the promise and price of the first gen­er­a­tion of CAR-T ther­a­pies), the ap­proach flips tra­di­tion­al cell ther­a­py on its head. Rather than en­gi­neer­ing im­mune cells to at­tack can­cer­ous ones, or tar­get­ing tu­mor cells, Vor is tin­ker­ing with the healthy cells.

By edit­ing out tar­gets that are present in both healthy and ma­lig­nant cells, the rea­son­ing goes, sci­en­tists can un­leash the full pow­er of tar­get­ed ther­a­pies with­out hav­ing to wor­ry about side ef­fects.

They are start­ing out with CD33 for acute myeloid leukemia, but the new cash will fund some new re­search in­to oth­er undis­closed tar­gets.

Al­so known as Siglec-3, the pro­tein is a com­mon tar­get for ex­ist­ing treat­ments like Pfiz­er’s an­ti­body-drug con­ju­gate My­lotarg. Large ge­nom­ic data­bas­es sug­gest there are in­di­vid­u­als who have lived their whole lives with­out ever ex­press­ing the CD33, Ang said, and re­searchers have shown that knock­ing it out in mice and hu­man cells in vit­ro doesn’t seem to im­pact func­tion­al­i­ty.

The re­al test, of course, will have to be in the clin­ic.

“We hope to see that it’s en­tire­ly bi­o­log­i­cal­ly re­dun­dant and safe,” Ang told End­points News.

On top of that — prov­ing not on­ly that they can ed­it the hematopoi­et­ic stem cells with­out in­duc­ing ma­lig­nan­cy, but al­so re­tain their re­pro­ducibil­i­ty — Vor is al­so aim­ing to test how their pro­gram, VOR33, may com­ple­ment My­lotarg or a CD33-tar­get­ed CAR-T ther­a­py, which would be a “nov­el nov­el treat­ment com­bi­na­tion.”

Vor has had a chance to dis­cuss these and oth­er con­cerns with FDA reg­u­la­tors back in Jan­u­ary, Ang added.

The tri­al would al­so be a test for the man­u­fac­tur­ing process Vor has de­vel­oped, pack­ing every­thing from ex­trac­tion to re­in­fu­sion in 48 hours.

“We’re do­ing it not just be­cause we can do it, but al­so be­cause I think it’s best for pa­tients that we do it,” Ang said. Not on­ly is there an ur­gency in the clin­ic, but less ma­nip­u­la­tion al­so means bet­ter chance of en­graft­ment and health­i­er cells. “What that al­so does is it gives us hope­ful­ly flex­i­bil­i­ty fur­ther down the track to think about how it could, more im­por­tant­ly, roll us out on a late de­vel­op­ment or com­mer­cial ba­sis.”

It could mean a re­gion­al mod­el or on-site man­u­fac­tur­ing; ei­ther way, it’s a de­cen­tral­ized fu­ture that Vor is now prepar­ing for.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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Ver­sant de­buts Ridge­line's start­up #4, armed with $30M and al­ter­na­tive TCR cell ther­a­pies for sol­id tu­mors

For all the iterations and advances in TCR therapies for cancer, any experimental treatments involving T cell receptors share one trait: By definition, they only recognize antigens presented as peptides on the major histocompatibility complex (MHC) on cells.

Versant reckons it’s time to expand the arsenal. With $30 million in initial funding, its Ridgeline Discovery Engine in Switzerland has been working on a non-peptidic approach that it says has tumor-agnostic potential, especially in solid tumors. They’ve named it Matterhorn, after a Swiss mountain as they did with the three other companies that have emerged from the Basel-based incubator.

CF Foun­da­tion, Long­wood team on new in­cu­ba­tor for com­pa­nies with cut­ting-edge CF treat­ments

Nine months after launching a $500 million hunt for a cure for cystic fibrosis, the Cystic Fibrosis Foundation said it will use a portion of those funds to do something it has never done before: help launch new companies.

The CF Foundation, whose venture philanthropy efforts helped fund Vertex’s line of powerful CF drugs, is teaming with Longwood Fund to create a CF incubator. The incubator will identify new companies with platforms or technologies that can be applied in the rare genetic condition. The partners can then finance early development in exchange for a commitment from the companies to focus on applications in cystic fibrosis.

UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Kiersten Stead, John Hamer (DCVC Bio)

Deep tech, round 2: DCVC Bio bags $350M fund to chase the tip of the life sci­ence spear

It took one trip from San Francisco to Vancouver for Kiersten Stead and her DCVC Bio crew to feel confident about throwing their weight — and cash — behind AbCellera.

CEO Carl Hansen’s academic background and the potential of the platform, which combined machine vision and robotics with microfluidics, were promising. But the site visit sealed the Series A deal, where DCVC was the lead and only investor.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Covid-19 roundup: BAR­DA qui­et­ly pulls plug on IL-6 drugs; BioN­Tech and Fo­s­un be­gin mR­NA tri­al

IL-6 inhibitors showed some early promise in potentially treating Covid-19 patients, but recent trial flops have dashed hopes. Now it appears BARDA has officially pulled the plug.

The HHS office’s website has quietly updated to reflect that it is “no longer supporting product development” for Covid-19 in both Actemra (tocilizumab) and Kevzara (sarilumab), dealing a blow to the Roche and Regeneron/Sanofi drugs. This all but assures that IL-6 drugs repurposed to treat Covid-19 are essentially dead in the water.

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Igor Splawski (CureVac)

Cure­Vac nabs a top No­var­tis sci­en­tist for CSO slot as mR­NA vac­cines seize the spot­light

One of the key players in the race to develop a new mRNA vaccine to fight Covid-19 has reshuffled the top spots in the executive suite. And they’re bringing in a Novartis vet out of Harvard to spearhead their work on mRNA.

CureVac, which just filed for an IPO that’s still taking shape, has formally handed Franz-Werner Haas the CEO title, after giving it to him on an interim basis. And the still rather stealthy German biotech largely owned by billionaire Dietmar Hopp has recruited Igor Splawski as its chief scientific officer.

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Tony Coles, Cerevel Therapeutics CEO

Adding $445M, Tony Coles and his big Pfiz­er neu­ro spin­out hitch a ride to Wall Street on Per­cep­tive’s SPAC

Two years ago, after Pfizer abruptly shut down its entire neuroscience division, Bain Capital bet $350 million that those assets were still worth something and packaged them into a new biotech: Cerevel Therapeutics. A year later, they got seasoned executive Tony Coles, who had recently jumped back into the C-suite of another neuroscience startup, to run the company.

Now Coles is steering Cerevel public, in what he says is the largest ever transaction of its kind. Cerevel has agreed to merge with Perceptive Advisors’ specialty acquisition company ARYA II. Between the roughly $125 million Perceptive raised through ARYA and an additional investment of $320 million Bain Capital, Perceptive and — yes, really — Pfizer, among others, Cerevel will now move forward with an added $445 million in its coffers.

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