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.”

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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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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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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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.

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. 

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.