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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Radek Spisek, Sotio CEO (Cellestia)

A qui­et Czech biotech bags $315M to dri­ve its blos­som­ing can­cer pipeline through the clin­ic

In the rather insular world of biotech, most innovation inevitably comes from a cluster of R&D hubs — Cambridge, San Francisco, etc. But sometimes success stories sprout from rocky soil, which is most certainly the case with Prague-based Sotio Biotech and its suddenly jam-packed pipeline of cancer drugs.

After years in quiet development, Sotio now has $315 million in new funds to play with from parent company PPF Group, an investment group founded in the Czech Republic, as the biotech looks to advance its growing pipeline through early- and mid-stage trials.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi snaps up new vac­cine can­di­date and de­vis­es mR­NA game plan around it — but not for what you think

Paul Hudson has spotlighted vaccines, immunology and dermatology as some of the top R&D focuses at Sanofi. His latest deal brings all of them together.

The French pharma giant isn’t sharing any financial details about the buyout of Origimm, a low-profile, private Austrian biotech whose technology promises to identify antigens causing skin disease and build vaccines against them. Their lead candidate targets acne vulgaris.

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