clinical-trials

The US government's health research agency has funded the first large-scale clinical trials testing whether rapamycin, semaglutide, and other repurposed drugs can slow biological aging in healthy adults, with results expected within three years.

The RUBY trial of renizgamglogene autogedtemcel (reni-cel), a one-time CRISPR-Cas12a gene-editing therapy, showed that 27 of 28 patients with severe sickle cell disease had no painful crises after treatment, with hemoglobin levels normalizing to 13.8 g/dL by six months.

An Fc-engineered CD40 agonist injected into a single tumor triggered systemic immune responses that eliminated cancer across the body in two patients, with expanded trials now under way at Memorial Sloan Kettering and Duke.

A redesigned CD40 agonist antibody injected into a single tumor triggered body-wide immune responses that eliminated all metastases in two patients, with nearly 200 now enrolled in follow-up trials.

Tozorakimab becomes the first IL-33-targeting biologic to show statistically significant reductions in COPD exacerbations across two confirmatory Phase 3 trials, after competitors Sanofi and Roche stumbled in the same drug class.

A brain-computer interface developed by Mass General Brigham and Brown University decoded intended finger movements into QWERTY keystrokes at 110 characters per minute with a 1.6 percent word error rate, offering a faster alternative to eye-gaze systems for people with ALS and spinal cord injuries.

Gene-edited pig kidneys have entered FDA-cleared clinical trials, a $176.8 million federal program is funding five teams to bioprint livers, and new cryopreservation methods could extend organ viability from hours to days.

A Cambridge-led study identifies CAG-170 bacteria as a global marker of gut health, Siolta Therapeutics reports a 64 percent reduction in infant atopic dermatitis risk, and UF Health isolates a bacterial metabolite that doubles lung cancer immunotherapy response in mice.

Life Biosciences began the first human trial of a cellular rejuvenation therapy using partial epigenetic reprogramming in Q1 2026, while senolytic pilot data shows safety in Alzheimer's-risk patients.

Takeda's oveporexton, an oral drug that restores the brain chemical missing in narcolepsy type 1, has received FDA priority review after two Phase 3 trials showed it brought patients to near-normal wakefulness and cut cataplexy episodes by more than 80 percent.

The FDA has cleared Intellia Therapeutics to resume its MAGNITUDE Phase 3 trial of nexiguran ziclumeran for ATTR cardiomyopathy, five months after a patient death and subsequent clinical hold prompted a safety overhaul.

Gene-edited pancreatic islet cells transplanted into a patient with type 1 diabetes continue producing insulin 14 months later with no immunosuppressive drugs, advancing the case for a functional cure.