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.

How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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Andrea Pfeifer, AC Immune CEO (AC Immune)

Look­ing to repli­cate Covid-19 suc­cess in neu­ro, BioN­Tech back­ers bet on AC Im­mune and its new­ly-ac­quired Parkin­son's vac­cine

The German billionaires behind BioNTech have found a new vaccine project to back.

Through their family office Athos Service, twin brothers Thomas and Andreas Strüngmann are leading a $25 million private placement into Switzerland’s AC Immune — which concurrently announced that it’s shelling out $58.7 million worth of stock to acquire Affiris’ portfolio of therapies targeting alpha-synuclein, including a vaccine candidate, for Parkinson’s disease.

Rajiv Shukla, Constellation Alpha Holdings

Can­del gets busy IPO week mov­ing with down­sized raise as Ra­jiv Shuk­la's third SPAC goes pub­lic

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

In a week that’s expected to see several biotechs price their IPOs, Candel Therapeutics got things kicked off Tuesday with a muted opener.

The company helmed by former GlaxoSmithKline vet Paul Peter Tak made its way to Nasdaq thanks to a $72 million raise, which was downsized by about 15% than originally anticipated, according to Renaissance Capital. Candel priced at $8 per share after initially seeking to launch in the $13 to $15 range.

Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.

Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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