Novartis, BMS pause autoimmune CAR-T trials after safety events. Novartis suspended several autoimmune and neurology trials of rap-cel after 3 patient deaths linked to immune effector cell-associated hemophagocytic syndrome, and BMS separately paused zola-cel studies after observing inflammatory safety events. Novartis has suspended several autoimmune and neurology trials of its investigational CD19 therapy rapcabtagene autoleucel (rap-cel) after 3 patients died following serious cases of immune effector cell-associated hemophagocytic syndrome (IEC-HS).[1,2] Bristol Myers Squibb has separately paused at least some studies of its own CAR-T candidate, zolacabtagene autoleucel (zola-cel), after observing transient and reversible inflammatory events in its autoimmune development program.[1,2] A Novartis spokesperson confirmed the suspension in a statement, saying the 3 IEC-HS cases led to complications that ultimately proved fatal.[1] The pause affects the phase 2 AUTOGRAPH studies evaluating rap-cel in systemic lupus erythematosus, systemic sclerosis, and idiopathic inflammatory myopathies (NCT06665256), along with phase 1/2 trials in rheumatoid arthritis and Sjogren disease (NCT07048197), generalized myasthenia gravis, and certain types of multiple sclerosis.[1] Studies of rap-cel in cancer are not affected by the pause.[1] Novartis is reviewing safety data as BMS acts out of caution. Novartis said it is analyzing safety data to identify the factors that may have contributed to the fatal complications and is working with independent data monitoring committees on enhanced safety measures, including tighter patient monitoring, earlier identification of adverse events, and improved management strategies.[1] BMS, describing its decision as a step taken out of an abundance of caution, confirmed it is voluntarily pausing at least some zola-cel studies to review clinical data across the therapy's development program.[1,2] The company has ongoing zola-cel trials across several autoimmune indications, including myasthenia gravis, multiple sclerosis, and rheumatoid arthritis, though it remains unclear whether the pause extends to all of them.[1] No deaths have been reported in connection with zola-cel; a single prior case of IEC-HS involving the therapy was described in results published in February from a phase 1 study.[2] "The drug's safety profile remains consistent with the known profile of CAR-T therapies," a BMS spokesperson said.[2] Analysts point to rapid manufacturing as a possible contributing factor. IEC-HS is a rare but serious adverse event previously reported with CD19 and BCMA-directed CAR-T therapies across blood cancer indications.[1] In a note to investors, analysts at William Blair pointed to rapid manufacturing, a feature shared by both rap-cel and zola-cel, as a possible contributor to the safety signals.[1] "Rapid manufacturing could be driving increased cell expansion and the reported toxicities," the analysts wrote, while also noting that indication-specific factors or a patient predisposition to a hyperinflammatory state could be playing a role.[1] The analysts cautioned that it remains difficult to extrapolate this risk to other CAR-T programs in development for autoimmune disease, noting that companies including Autolus, Cabaletta Bio, CRISPR Therapeutics, Kyverna Therapeutics, and Allogene Therapeutics are also developing CD19-directed CAR-T therapies for autoimmune indications.[1] Differentiated safety profiles among these competing programs could offer an advantage if development of the Novartis and BMS therapies remains constrained, according to the analysts.[1] Frequently asked questions. * What prompted Novartis to pause rap-cel trials in autoimmune disease? Three patients died after developing serious immune effector cell-associated hemophagocytic syndrome (IEC-HS) following treatment with rap-cel; the pause does not affect ongoing cancer studies of the therapy. * Why did BMS pause zola-cel studies? BMS voluntarily paused at least some zola-cel trials after observing transient and reversible inflammatory events, describing the move as a precaution while it reviews clinical data across the program; no deaths have been reported. * What might be driving these safety signals? Analysts have pointed to rapid manufacturing, a feature shared by both CAR-T candidates, as a possible contributing factor to increased cell expansion and toxicity, though indication-specific factors or patient predisposition may also play a role.

Investors undervaluing the life-changing power of AI. By Derek Rose Updated September 4 2026 - 1:03pm, first published 1:00pm Humanoid robots are among products in which a prominent futurist is investing billions of dollars. Photo: James Ross/AAP PHOTOS Australians underestimate just how transformative artificial intelligence will be, says a high-profile futurist who's bringing her investing prowess to local shores. ARK Invest chief executive and co-founder Cathie Wood said while investors understood the opportunities offered by data centres, there's more to AI than that. "What's underappreciated, in terms of AI, is what we call embodied AI or physical AI," Ms Wood told AAP in an interview while visiting Australia. The Florida-based executive became one of the most celebrated investment gurus on the planet during the COVID-19 pandemic, after her flagship fund delivered a stunning 153 per cent return in 2020 thanks to big, bold bets on disruptive technologies. Cathie Wood's fund backs companies in gene editing, autonomous transport and humanoid home robots. (PR IMAGE PHOTO) These days the fund's largest position remains in Elon Musk's electric vehicle and AI robotics company, Tesla, which she describes as the biggest AI project on Earth. The fund holds more than $1.6 billion in Tesla shares, making up roughly a tenth of its portfolio. "Autonomous transportation, we think, is going to deliver revenues within the next five to 10 years of $US10 trillion ($14 trillion) per year," Ms Wood said, adding that this reflects the entire ecosystem including China. "Beyond that, humanoid robots, which is also Tesla. We think that's a $US25 trillion ($35 trillion) revenue opportunity per year, both in the home and in manufacturing." Mr Musk's humanoid "Tesla bot" might be on sale to the public in 2027. Ark Invest's biggest holding remains Elon Musk's electric vehicle and AI robotics company Tesla. (AAP PHOTOS) However, Ms Wood believes the biggest transformative opportunity for AI lies in health care. "We think that AI is going to collapse the time and the cost to discover new drugs and therapies," she said. "With just blood tests, it will help us diagnose diseases before they're even symptomatic." AI-powered gene editing will cure diseases, Ms Wood said, pointing to Swiss-American company CRISPR Therapeutics that is already using gene editing to treat rare blood diseases sickle cell and beta thalassemia. "Many people say, 'Oh, it's so small. These are just rare diseases'. It's not just rare diseases," Ms Wood said. Health care is where ARK Invest co-founder Cathie Wood sees the biggest scope for AI to transform. (Joel Carrett/AAP PHOTOS) CRISPR is also working on a gene-editing therapy to cure bad cholesterol, and Ms Wood believes the one-time infusion has blockbuster investor potential. Monash University's Victorian Heart Hospital was among institutions worldwide to participate in an early 15-person clinical trial that concluded in November. ARK has invested in other companies using AI to quickly develop potential treatments for endometriosis and alopecia, a disorder that causes hair loss. It currently takes about 13 years and $US2.4 billion ($3.4 billion) to develop a new drug, and Ms Wood believes that AI will collapse that to five years and $US600 million to $US700 million (roughly $907 million). "We think what's going to happen in this new age is we're going to transform sick care, which is where most money is spent today in health care, into real health care and preventative care," she said. "I think it's one of the most exciting developments in healthcare history." Cathie Wood is pitching her funds in disruptive companies to Australian wholesale investors. (Lukas Coch/AAP PHOTOS) Ms Wood was speaking to AAP ahead of opening her venture capital fund to Australian wholesale investors through a partnership with Associate Global Partners. The ARK Venture Fund invests in disruptive public and private companies such as OpenAI, Anthropic and the prediction market Kalshi. Ms Wood will return in Australia within weeks to meet with potential investors, and praised the nation for its open mind to innovation. "I shouldn't be playing Europe against Australia, but you know, Europe is so tied up in regulation," she said. Australian Associated Press

CRISPR Therapeutics outlined a busy second-half pipeline and reported strong quarterly growth in its marketed gene therapy. Shares rose 1.4% in after-hours trading on Monday. Updates are expected on a gene-editing treatment for severe high triglycerides and hard-to-control cholesterol, with results to be presented at a major heart conference on 28 August. More data is also anticipated for a blood-clotting therapy and an immune-cell treatment being tested in autoimmune diseases and blood cancers. CASGEVY, the gene therapy for sickle cell disease and a related blood disorder, generated $76 million in second-quarter revenue, a 78% increase from the prior quarter. The FDA recently approved it for children as young as two. Net loss narrowed to $91.2 million from $208.5 million a year earlier.