Drug Development
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
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As drug candidates discovered via AI move into later-stage clinical trials, the technology seems to be doing as promised: speeding drug development.
Following the hard-won success of early anti-amyloid drugs, a new generation of Alzheimer’s modalities—from tau-targeting gene silencers to blood-brain barrier delivery platforms—is entering the pipeline to anchor future combination therapies.
Three years after the accelerated approval of its anti-amyloid Alzheimer’s therapy, Biogen—neck and neck in the market with Eli Lilly and its Kisunla offering—is focused on a near-term FDA decision for a subcutaneous induction dose of Leqembi, a presymptomatic readout in 2028 and a clutch of next-generation candidates.
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The COVID-19 pandemic has seen a full-blown effort from biopharma and researchers to develop new drugs and test old ones against the disease, with highly mixed results.
enGene announced positive results from its LEGEND Phase I/II clinical trial for treating high-grade NMIBC in patients with carcinoma in situ that are unresponsive to BCG treatment.
Statistically significant weight loss and liver fat reduction were seen across all dosage groups—with no changes to diet or exercise.
Endevica Bio isn’t trying to cure cancer. Instead, it is focused on improving cancer patients’ quality of life and preventing deaths.
Diabetes-focused life sciences company Zucara Therapeutics released preclinical findings on a daily therapy to slow and prevent plummeting blood glucose levels in Type 1 diabetes patients.
Weston Miller, M.D., senior medical director of clinical development at Astellas Gene Therapies, called the positive interim and tolerability data an important waypoint.
Antiviral pills against COVID-19 show promise for treating the disease, but they are not broadly accessible yet.
ALZ-801 is administered orally and works by blocking the formation of neurotoxic soluble amyloid oligomers that later lead to cognitive decline in Alzheimer’s patients.
The U.S. Food and Drug Administration announced they are investigating a possible death risk for TG Therapeutics’ Ukoniq (umbralisib).
The same advantage against HIV-1 possessed by a person with the inactive CCR5 variant potentially doubles as resistance to any virus that capitalizes on or manipulates that receptor.