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Non-profit to advance low-dose Duchenne gene therapy Read in browser
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1. Biopharma is wagering billions of dollars that CAR-T therapy can be easier. Will it work this time?
2. French non-profit bets on Duchenne gene therapy that comes in lower dose than Elevidys
3. Cabaletta Bio says small number of autoimmune patients responded to its CAR-T without toxic preconditioning
4.
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Satellos to run a trial for Duchenne pill in kids; Idorsia raises $81M
5. Bristol Myers joins in vivo CAR-T race with $1.5B Orbital acquisition
6.
peer review
Shape's former CSO picks up tech role at Moderna; Lilly reels in Peter Marks
7. Samsara BioCapital eyes $200M opportunity fund
8. Ypsomed spends $248M to gain its first US manufacturing site 
9. Altos reveals early hints about its anti-aging work with tests run in organs kept alive outside the body
10. Evommune files for IPO to fund two Phase 2 autoimmune drugs
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Jaimy Lee
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We’re holding our annual AI Day next Tuesday. The event will feature discussions with leaders at Isomorphic Labs and Google DeepMind as well as Pulitzer-winning doctor and author Siddhartha Mukherjee. Check out the agenda here, and get your virtual pass here. For those attending the live event in New York, be sure to say hello!

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Jaimy Lee
Deputy Editor, Endpoints News
1
by Lei Lei Wu

Large bio­phar­ma com­pa­nies have spent over $4 bil­lion in 2025 to buy star­tups work­ing on in vi­vo CAR-T ther­a­pies as the first da­ta in hu­mans emerge.

While it's still ear­ly days for in vi­vo cell ther­a­py, the deals show that bio­phar­ma is go­ing in on the idea that these next-gen­er­a­tion ther­a­pies can fight can­cer and au­toim­mune dis­ease like tra­di­tion­al CAR-T ther­a­pies, but they’ll be less ex­pen­sive and time-con­sum­ing.

Bris­tol My­ers Squibb be­came the lat­est ac­quir­er on Fri­day morn­ing, com­mit­ting $1.5 bil­lion to pur­chase Or­bital Ther­a­peu­tics. As­traZeneca, Ab­b­Vie and Gilead have all bought biotechs mak­ing in vi­vo treat­ments this year.

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2
by Lei Lei Wu

Genethon's lat­est da­ta sug­gest its Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py helped sta­bi­lize func­tion­al out­comes af­ter two years. How­ev­er, the tri­al en­rolled on­ly five pa­tients, who were then com­pared with those from a par­al­lel nat­ur­al his­to­ry study.

The non-prof­it gene ther­a­py de­vel­op­er, which is part of the French Mus­cu­lar Dy­s­tro­phy As­so­ci­a­tion known as AFM-Téléthon, re­cent­ly start­ed a Phase 3 tri­al in hopes of repli­cat­ing the find­ings in more pa­tients and com­par­ing them to place­bo.

Genethon an­nounced the up­dat­ed Phase 1/2 re­sults on Thurs­day at the an­nu­al Eu­ro­pean So­ci­ety of Gene and Cell Ther­a­py meet­ing in Seville, Spain.

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3
by Max Gelman

A mi­cro­cap biotech from Philadel­phia re­leased ear­ly, but pos­i­tive find­ings from its CAR-T tri­al for a rare au­toim­mune dis­or­der, sug­gest­ing it could be ad­min­is­tered with­out one of the most ar­du­ous as­pects of cell ther­a­py.

Ca­balet­ta Bio on Thurs­day said its CAR-T ther­a­py called rese­cab­ta­gene au­toleu­cel, or rese-cel and pre­vi­ous­ly known as CA­BA-201, in­duced pre­lim­i­nary re­spons­es de­spite pa­tients not re­ceiv­ing any tox­ic pre­con­di­tion­ing reg­i­mens. Re­searchers test­ed the ther­a­py in pem­phi­gus vul­garis, an au­toim­mune dis­ease af­fect­ing the skin and mu­cus mem­branes.

Pre­con­di­tion­ing has been a sta­ple of CAR-T since its in­cep­tion and is an im­por­tant part of the can­cer-fight­ing process.

Pa­tients who take ap­proved CAR-Ts for blood can­cers must first re­ceive a reg­i­men of ex­treme­ly in­ten­sive chemother­a­py that ef­fec­tive­ly wipes out the body’s en­tire im­mune sys­tem. Then, the pa­tient’s T cells are ge­net­i­cal­ly reengi­neered and put back in­to the body, train­ing the im­mune sys­tem to fight can­cer cells from scratch.

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