Part pro­ce­dure, part drug: Robert Ang joins Sid­dhartha Mukher­jee in pi­o­neer­ing a new type of cell en­gi­neer­ing

In a re­cent chron­i­cle on the promise and price of cell ther­a­pies, Sid­dhartha Mukher­jee — au­thor, on­col­o­gist and Co­lum­bia pro­fes­sor among his oth­er ti­tles — mused about how en­gi­neered T cells had blurred the tra­di­tion­al bound­aries sep­a­rat­ing a pro­ce­dure and a drug. “Pro­ce­dures come alive in the tin­ker­ing, fuss­ing hands of their op­er­a­tors,” he ob­served, while a “drug, in con­trast, is a de­per­son­al­ized en­ti­ty.” For the new gen­er­a­tion of metic­u­lous­ly man­u­fac­tured CAR-T to reach the mass­es, in­no­va­tors must mar­ry the it­er­a­tive na­ture of a pro­ce­dure with the pro­duc­tion ef­fi­cien­cy of a drug — and add a ruth­less pur­suit of the lean­est, cheap­est process pos­si­ble.

Robert Ang

He’s putting that con­cept to work in a new ven­ture. Vor Bio­phar­ma, a biotech fledg­ling bred at PureTech and now set­tling in­to a new nest at Kendall Square, is en­gi­neer­ing hematopoi­et­ic stem cells to rid them of the anti­gen CD33 so that acute myeloid leukemia pa­tients may reap the ben­e­fits of CD33- tar­get­ed ther­a­pies with­out the tox­i­c­i­ties.

What Mukher­jee came up with is an “ex­treme­ly el­e­gant” ap­proach, says Robert Ang, who’s start­ing his first week as CEO of Vor. The prod­uct, VOR33, would in ef­fect shield healthy blood cells and the bone mar­row from a T cell at­tack.

Sid­dhartha Mukher­jee

An ex vi­vo pro­ce­dure in­volv­ing a sin­gle ge­net­ic ed­it of healthy donor cells rep­re­sent “ide­al en­try points for CRISPR-based ther­a­peu­tics, and that’s pre­cise­ly what VOR33 is,” he says. “And so we ex­pect the pro­duc­tion process to be rel­a­tive­ly straight­for­ward with on­ly sev­er­al-day process but of course you can nev­er take that for grant­ed.”

“I’ve just been itch­ing to get go­ing,” adds the biotech vet­er­an, who had keen­ly learned lessons about com­pa­ny build­ing as em­ploy­ee #5 and chief busi­ness of­fi­cer of neoanti­gen play­er Neon Ther­a­peu­tics.

Kush Par­mar

Soon af­ter he start­ed pon­der­ing about lead­ing a com­pa­ny of his own he got in touch with Kush Par­ma, man­ag­ing part­ner at 5AM Ven­tures and Vor chair­man who’s al­so work­ing with Mukher­jee on an­oth­er CAR-T project. 5AM was joined by RA Cap­i­tal Man­age­ment, Os­age Uni­ver­si­ty Part­ners and PureTech, as well as J&J’s ven­ture arm and No­var­tis In­sti­tutes for Bio­Med­ical Re­search, for a $42 mil­lion Se­ries A an­nounced ear­li­er this year.

It’s still ear­ly days — so ear­ly that he’s not ready to share how many are on the team — but Ang al­so has a clear time­line for en­ter­ing the clin­ic in 18 months. A more near-term goal would be to sit down with the FDA about an IND by the end of the year.

“We want to make sure that the prod­uct is safe, and that the prod­uct en­grafts just like oth­er typ­i­cal trans­plants,” he says of the first hu­man tri­al they are work­ing to­ward. “But at the same time we’re al­so very in­ter­est­ed to see what hap­pens when the pa­tients — if they do progress, how the trans­plants be­have when tar­get­ed ther­a­py is ap­plied.”

The com­par­i­son to stan­dard-of-care would be cru­cial as Vor proves that it’s just as good, if not bet­ter than the bone mar­row trans­plants that AML pa­tients would oth­er­wise have re­ceived.

As for the sub­se­quent tar­get­ed ther­a­py that VOR33 would dove­tail in­to, the sea­soned BD ex­ec main­tains there’s a good amount of op­tion­al­i­ty as to which modal­i­ty they go with. So far Pfiz­er claims the on­ly mar­ket­ed drug hit­ting CD33 in My­lotarg, and a slate of oth­ers are in de­vel­op­ment. Nonethe­less, he notes that J&J and No­var­tis both have strong pipelines in tar­get­ed ther­a­py and CAR-T.

It’s not just CD33, ei­ther. Vor plans to ap­ply the same gene-edit­ing ap­proach to a num­ber of tar­gets, cre­at­ing com­pan­ion pro­ce­dures for mul­ti­ple tar­get­ed ther­a­pies.

Ang, who trained as a doc­tor in Aus­tralia and had stints at both Boston Con­sult­ing Group and Fra­zier Health­care Ven­tures be­fore tak­ing on front­line biotech roles, sees build­ing up the cul­ture at Vor as one of his key tasks.

“Neon’s cul­ture is ab­solute­ly in­cred­i­ble, I’d love to em­u­late that, and al­so build on the great cul­ture that Vor it­self has cre­at­ed over the past few months,” he says. “Vor peo­ple are high­ly col­lab­o­ra­tive, love sci­ence, but are al­so dri­ven by a high­er pur­pose. So build­ing on that and for­mal­iz­ing that will be re­al­ly im­por­tant in the months ahead.”

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Look­ing for 'ex­ter­nal in­no­va­tion,' Boehringer In­gel­heim re­serves $500M+ for new Shang­hai hub

Now that Boehringer Ingelheim’s bet on contract manufacturing in China has paid off, the German drugmaker is anteing up more to get into the research game.

Boehringer has set aside $507.9 million (€451 million) for a new External Innovation Hub to be built in Shanghai over five years. The site will become one of its “strategic pillars” as the team strives to get 71 approvals — either for new products or indications — by 2030, said Felix Gutsche, president and CEO of Boehringer Ingelheim China.

Vas Narasimhan, Novartis CEO (Patrick Straub/​EPA-EFE/​Shutterstock)

No­var­tis pays $678M for kick­back scheme as Vas Narasimhan tries to dis­tance phar­ma gi­ant from shady be­hav­ior

Novartis has reached another large settlement to resolve misconduct allegations, agreeing to pay more than $678 million to settle claims that it had spent hundreds of millions of dollars on lavish dinners, so-called speaking fees and expensive alcohol “that were nothing more than bribes” to get doctors to prescribe Novartis medications.

The top-shelf alcohol and lavish meals included a $3,250 per person night at Nobu in Dallas, a $672-per person dinner at Washington DC’s Smith & Wollensky and a $314 per person meal at Sushi Roku in Pasadena, according to the Justice Department complaint. There were at least 7 trips to Hooters and fishing trips in Alaska and off the Florida coast. Each of these events were supposed to be “speaker programs” where doctors educated other doctors on a drug, but the DOJ alleged many were “bogus” wine-and-dine events where the drug was barely mentioned, if at all.  (“Nobody presented slides on the fishing trips,” the complaint says.)

No­vavax snags Ben Machielse for CMC and pro­motes a trio of staffers; Mar­ty Du­vall lands an­oth­er CEO post at On­copep­tides

Novavax has been making waves recently by securing a $384 million commitment from CEPI to cover R&D and manufacturing for its Covid-19 vaccine while also spending $167 million on a 150,000 square-foot facility. The Maryland biotech continues to shore up its leadership team as well, bringing in Ben Machielse as their EVP of CMC just a couple weeks after nabbing AstraZeneca vet Filip Dubrovsky as their new CMO. Machielse was president and CEO of Vtesse from 2014-17, and before that, he also spent more than 11 years at MedImmune and was EVP of operations for the back half of his tenure.

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

Paul Tesar (Convelo Therapeutics)

Io­n­is, lead­ing MS re­searcher throw an­ti­sense at a new type of brain cells

No matter how many molecules he threw at them, Paul Tesar couldn’t get the brain cells to survive. Or he got them to survive, but then — to everyone’s bafflement — they still couldn’t do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.