Andy Scharenberg, Michael Jensen

MPM, DCVC Bio back $53M round for a bold con­cept: Let pa­tients make their own CAR-T in vi­vo

The oner­ous process of mak­ing a per­son­al­ized CAR-T — one that in­volves ex­tract­ing cells from pa­tients, en­gi­neer­ing and then in­ject­ing them back — is one of the defin­ing fea­tures of the first gen­er­a­tion of en­gi­neered cell ther­a­pies. It’s part­ly why the treat­ments are so ex­pen­sive; it’s al­so an in­spi­ra­tion for new al­lo­gene­ic ap­proach­es that promise to be faster and cheap­er.

But a biotech start­up in Seat­tle wants to go fur­ther.

Umo­ja Bio­phar­ma is start­ing out with $53 mil­lion in Se­ries A cash to test whether a lentivi­ral vec­tor de­liv­ered di­rect­ly in vi­vo can gen­er­ate a pop­u­la­tion of T cells that ex­pand and fight can­cer just as well as the ex­ter­nal­ly man­u­fac­tured ones do.

“So we don’t ac­tu­al­ly have to man­u­fac­ture the cells,” co-founder and CEO Andy Scharen­berg told End­points News. “We’re gonna be ef­fec­tive­ly mak­ing a com­plex bi­o­log­ic.”

Scharen­berg, who’s ex­plored mul­ti­ple ways to do gene trans­fer through the years at places like Cel­lec­tis and Gen­er­a­tion Bio, high­light­ed the “in­cred­i­ble safe­ty record” of lentivi­ral vec­tors and their abil­i­ty to not just trans­duce but al­so in­te­grate the ge­nom­ic pay­load in­to daugh­ter cells. Be­cause the re­sult­ing CAR-T would ex­pand in a way that repli­cates a nat­ur­al im­mune re­sponse, he al­so fore­sees few of the side ef­fects, such as cy­tokine re­lease syn­drome, that pa­tients ex­pe­ri­ence with Kym­ri­ah and Yescar­ta.

A sec­ond tech­nol­o­gy uti­lizes small mol­e­cules to con­trol and tune these self-gen­er­at­ed CAR-T cells fol­low­ing a sin­gle ad­min­is­tra­tion. And Umo­ja has lined up an ad­di­tion­al tech plat­form to push it in­to sol­id tu­mors by tag­ging can­cer cells for recog­ni­tion by T cells.

Bring­ing all three ap­proach­es and the re­spec­tive ex­perts un­der the same roof is a key idea be­hind Umo­ja — a Swahili word for “uni­ty.”

“In many ways the sci­en­tif­ic con­cept un­der­ly­ing the com­pa­ny is a lit­tle bit sum­ma­rized in that word,” Scharen­berg said. “In or­der to have a re­al­ly im­pact­ful ap­proach to ad­vanc­ing im­munother­a­pies, you need to in­te­grate mul­ti­ple tech­nol­o­gy plat­forms.”

Philip Low

With Michael Jensen of Seat­tle Chil­dren’s and Philip Low of Pur­due Uni­ver­si­ty on board as co-founders, Umo­ja plans to pur­sue par­al­lel de­vel­op­ment paths for the in vi­vo gene de­liv­ery and tu­mor tag tech­nolo­gies, which the CEO de­scribes as two sides of the same coin. Jensen will lead a col­lab­o­ra­tion to eval­u­ate the tu­mor tag ap­proach among chil­dren with os­teosar­co­ma in the com­ing year. Mean­while, Umo­ja will get ready to put its ini­tial CAR-T hit­ting a fa­mil­iar tar­get — CD19 — in the clin­ic.

If “things go re­al­ly well,” Scharen­berg sees the com­pa­ny stack­ing the two ap­proach­es for a more po­tent ef­fect in the 2023, 2024 time frame.

MPM Cap­i­tal and DCVC Bio seed­ed the com­pa­ny, with Qim­ing Ven­ture Part­ners USA join­ing to co-lead the Se­ries A.

As cel­lu­lar im­munother­a­pies grad­u­al­ly move from their cur­rent ap­pli­ca­tions to­ward the front­line — some­thing Scharen­berg en­vi­sions — he sees the scale and ac­ces­si­bil­i­ty be­ing his com­pa­ny’s key mantra.

“When we think about what Umo­ja as­pires to be as an or­ga­ni­za­tion, it’s not on­ly to ad­vance and make im­munother­a­pies that are more ef­fec­tive against all tu­mors,” he said, “but al­so to make them ac­ces­si­ble and to al­le­vi­ate some of the eco­nom­ic tox­i­c­i­ties that are as­so­ci­at­ed with the cost of some of the cur­rent ther­a­pies as well.”

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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Vipin Suri, Catamaran Bio CSO

Cata­ma­ran Bio sails in­to the CAR-NK wa­ters with a $42M launch round

Catamaran Bio’s founding members decided to jump into the CAR-NK game last December over drinks at a trendy bar in Boston.

They were sitting around a table, discussing an MD Anderson study which provided some of the first clinical proof that natural killer (NK) cells can be reengineered to attack tumors, much like CAR-T therapies. It was a “long and lively” discussion, COO Mark Boshar recalls. And by the time it was over, they had a starting point to launch a company.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Peter Thiel (Riccardo Savi/Sipa via AP Images)

Tech bil­lion­aire Pe­ter Thiel backs a lead­ing psy­che­del­ic drug de­vel­op­er

Right on the heels of investing in antibody drug developer AbCellera, Facebook billionaire Peter Thiel has jumped into a syndicate putting up $125 million for a company with a portfolio of psychedelic drugs in the clinic for mental health.

The C round — which includes a $32 million conversion of notes to equity — will fuel the development programs at ATAI Life Sciences, a Berlin-based biotech that has assembled a portfolio of companies with psychedelic and non-psychedilc approaches to depression, anxiety and addiction.

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