J&J Strikes In Vivo CAR T Deal with Sail
Johnson & Johnson has entered a strategic collaboration with Sail Biomedicines to develop in vivo CAR T-cell therapies for immune-mediated diseases.
Unlike conventional ex vivo CAR T-cell therapies, Sail’s platform is designed to reprogram a patient’s immune cells directly inside the body. The approach aims to reset the immune system while offering a simpler and more scalable route to durable disease control.
J&J will make initial payments totaling $785 million, including a $465 million equity investment, with a further $140 million tied to development milestones. The company has also secured an exclusive option to acquire Sail for $2.58 billion, bringing the potential value of the agreements to more than $3.5 billion.
The partners will initially advance Sail’s lead immune-mediated disease program, with scope to apply the platform to additional therapeutic targets. The transactions remain subject to regulatory approvals and other closing conditions. Source
FDA Committee Votes Against Deramiocel for Duchenne Cardiomyopathy
An FDA advisory committee has concluded that available evidence does not support the effectiveness of deramiocel for treating cardiomyopathy in patients with Duchenne muscular dystrophy.
The Cellular, Tissue and Gene Therapies Advisory Committee voted three to nine against the therapy, with no abstentions. The vote is nonbinding, and the FDA is expected to make a decision by the August 22, 2026, Prescription Drug User Fee Act target date.
Deramiocel is an allogeneic cardiosphere-derived cell therapy designed to preserve cardiac and skeletal muscle function through immunomodulatory and antifibrotic activity. The cells secrete exosomes that are thought to shift macrophages from a pro-inflammatory state toward a healing phenotype.
Capricor Therapeutics noted that the committee considered a narrower cardiomyopathy indication than the company had proposed and did not vote on the therapy’s overall benefit-risk profile. The company said discussion of upper-limb results from the Phase III HOPE-3 trial was more supportive. Source
Inherited Variants Shape CAR T Safety and Activity
A patient’s inherited genetic makeup may influence both the efficacy and toxicity of autologous CAR T-cell therapy, according to a study involving more than 200 people with aggressive lymphoma.
Researchers sequenced the participants’ entire genomes using samples from two major CAR T clinical trials. Variants that silenced the gene STXBP2 were associated with treatment-related toxicity in one trial, while cell-culture experiments showed that engineered T cells carrying the variants triggered inflammation.
Across the trials, variants in ADAMTSL3 were associated with protection from toxicity. Variants in PTPN22 were also strongly linked to greater CAR T-cell expansion, an important determinant of therapeutic efficacy.
The findings suggest that incorporating germline genetic analysis into clinical trials could help predict patient responses and guide the design of personalized therapies. The approach may also inform donor selection for future allogeneic CAR T-cell products manufactured from a single donor for multiple patients. Source
Immature NK Cells Undermine Cord Blood CAR NK Therapy
Removing a small population of immature natural killer cells from donated cord blood could improve the potency and consistency of off-the-shelf CAR NK cell therapies.
Researchers at The University of Texas MD Anderson Cancer Center found that cord blood units containing more mature NK cells were associated with stronger treatment responses and better patient outcomes. Higher levels of immature NK cells, meanwhile, correlated with reduced efficacy.
The immature cells acquired cancer-associated proteins through trogocytosis and displayed them on their surfaces. Engineered CAR NK cells then mistook these cells for tumor targets and attacked them, reducing the fitness and persistence of the therapeutic cell population.
Depleting the immature subset before manufacturing strengthened CAR NK cell function and improved tumor control and survival in preclinical models of lymphoma and ovarian cancer. The findings could support improved donor selection and manufacturing strategies for next-generation cord blood-derived CAR NK therapies. Source
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