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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.

Christophe Bourdon, Leo Pharma CEO

Leo Phar­ma looks 'be­yond the skin' in atopic der­mati­tis aware­ness cam­paign

As Leo Pharma aims to take on heavyweight champ Dupixent in atopic dermatitis, the company is launching “AD Days Around the World,” an awareness campaign documenting real patient stories across Europe.

The project, unveiled on Monday, spotlights four patients: Marjolaine, Laura, Julia and África from France, Italy, Germany and Spain, respectively, in short video clips on the challenges of living with AD, the most common form of eczema.

Ying Huang, Legend CEO

Lentivi­ral vec­tor ramp-up: J&J and Leg­end to in­vest $500M in New Jer­sey man­u­fac­tur­ing to sup­port Carvyk­ti

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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