Eight years after Novartis AG gained U.S. FDA approval of the first CAR T therapy, Kymriah (tisagenlecleucel), for B-cell acute lymphoblastic leukemia, developers are advancing prospects that could significantly impact another disease space outside of cancer – autoimmunity. The efforts are getting a swirl of attention, with Bristol Myers Squibb Co. announcing Oct. 10 that it would offer $1.5 billion in cash to buy three-year-old privately held Orbital Therapeutics Inc., including its lead, next-generation CAR T-cell therapy OTX-201, which is designed to reprogram cells in vivo for autoimmune diseases. A day earlier, Cabaletta Bio Inc. presented data indicating its autologous CAR T therapy resecabtagene autoleucel, without preconditioning, led to complete B-cell depletion in two of three patients with a rare, autoimmune skin disorder. That followed three scientists winning the Nobel Prize in Physiology or Medicine on Oct. 7 for their discovery of regulatory T cells, which are critical for maintaining immune tolerance and preventing autoimmune attacks.

M&As slow as biopharma dealmaking value jumps 32% in September 2025

Biopharma dealmaking was up 32% month over month in September 2025, reaching $27.15 billion in total value, up from $20.52 billion in August. It was the fourth-highest month for deals of the year, which thus far has an average of $23.6 billion a month. Ten biopharma deals in September topped $1 billion in potential value. The largest was Monte Rosa Therapeutics Inc., in its second deal with Novartis AG of the past 11 months, which could swell to $5.7 billion for Monte Rosa.

Gastroesophageal cancer in crosshairs of industry, NIH

The U.S. National Institutes of Health’s National Cancer Institute recently launched a program to “examine and define the earliest biological changes in malignant transformation” of gastroesophageal cancer, an indication targeted by a slew of drug developers. Antibody-drug conjugates (ADCs) represent a popular mechanism, but that’s hardly the only approach taken. Astrazeneca plc has in the works an ADC, a checkpoint inhibitor, and a T-cell engager meant to tackle the tumor type.

Gene therapy for hemophilia stalls but doesn’t stop at ESGCT

 While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy, held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity. Lindsey George, assistant professor of pediatrics (hematology) at the University of Pennsylvania School of Medicine, discussed “one of the key current challenges in hemophilia A gene therapy,” during her presentation “Next generation hemophilia A gene therapy” included in the parallel session “Bleeding disorders.”

Also in the news

Abli, Adcendo, Advantage, Alterity, Apogee, Ariceum, Ascendis, Aviadobio, Chance, Cidara, Essa, Expedition, Huadong Medicine, IAVI, Italfarmaco, Neurogene, Odyssey, Ono, Orexa, OS Therapies, Probiogen, Pyxis, Regeneron, Henlius, Sunshine, Taysha Gene Therapies, Ugenex, Vtv, Xeno, Xspray, Zelluna, Zymeworks