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.”

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.

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.

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.