clinical-trials

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.

MagicRNA's mRNA lipid nanoparticle therapy reprogrammed patients' own T cells inside the body, wiping out disease-driving B cells in refractory lupus patients without conventional cell manufacturing.