Gene Therapies

New preclinical data reveals a novel drug, rinzimetostat, may overcome treatment resistance in prostate cancer by targeting a unique molecular switch.

Nested Therapeutics' NST-628 reports a 38% response rate in advanced melanoma, offering new hope with a novel, brain-penetrating cancer-fighting agent.

Interim study results suggest a common food product may significantly enhance the effectiveness of modern cancer treatments, heralding a new era of care.

New data reveals Mapi Pharma's long-acting GA Depot stabilizes disability in both relapsing and progressive MS, poised for a 2027 European launch.

Patients, doctors, and researchers unite for a grueling ride across Iceland, raising funds and hope for a future free from multiple myeloma.

A novel drug for a debilitating stomach disorder is set for a major reveal, offering hope to millions with limited and often risky treatment options.

Promising early safety data for a novel drug, KM-023, brings a potential first-ever targeted treatment for the devastating Olmsted syndrome a step closer.

A new FDA-approved powder form of a common blood pressure drug aims to solve a daily struggle for the elderly, children, and tube-fed patients.

With a fresh $28M investment, Verismo Therapeutics is set to unveil groundbreaking KIR-CAR data, aiming to revolutionize CAR T therapy for solid tumors.

A landmark grant funds research into scaffold-free tissue, a revolutionary approach that could one day eliminate the need for many knee replacements.

A new once-daily pill for hypoparathyroidism begins human testing, promising to replace burdensome injections and transform patient care. Is this the future?

Replimune halts a promising cancer therapy and cuts jobs after a stunning FDA rejection, citing a 'failed' system and inconsistent regulatory process.

Positive Phase 1b data for two novel drugs suggests a long-acting, once-monthly oral therapy for recurrent genital herpes may be on the horizon.

OncoC4 begins a landmark trial for ONC-841, a drug targeting immune cells in the brain, offering a new front in the fight against Alzheimer's disease.

With its lead drug in Phase 3 and a strong Biogen partnership, Stoke Therapeutics prepares to update investors on its disease-modifying Dravet syndrome therapy.

A single-dose therapy wiped out aggressive glioblastoma tumors in a landmark pre-clinical study, paving the way for human trials in 2026.

Ovid Therapeutics is spotlighting a novel drug class targeting KCC2, a key brain protein. Could this be the key to treating psychosis and epilepsy?

AIRNA has dosed the first patient with AIR-001, an RNA-editing drug that could treat both the lung and liver damage of a devastating genetic disease.

Avaí Bio and Austrianova are set to present new findings on a novel cell-based therapy designed to produce the 'longevity protein' Klotho.

A new trial for advanced liver cancer tests visugromab, a drug aiming to overcome immunotherapy resistance and reverse debilitating cancer-related wasting.