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.
FEATURED STORIES
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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Visionary tech magnate Elon Musk is taking his neurotech company to the next step toward in-human trials of his implantable brain-machine interface.
Public health officials churn out a lot of statistics related to COVID-19, but it can often be difficult to determine just how accurate they are because there are just so many unreported test results.
The Glycogen Storage Disease Program at Connecticut Children’s and UConn Health reported success in a Phase I/II trial of a gene therapy for GSD-Ia.
The data is coming in right away for Alnylam in one of its primary indications, transthyretin-mediated (ATTR) amyloidosis.
The FDA has lifted the partial hold on Kura Oncology’s KOMET-001 Phase IB trial on a potential treatment for acute myeloid leukemia.
As part of a company-wide workforce reduction, it will cut its employee headcount by at least 25% by the end of the year, and 50% of its senior management team are leaving the company.
For about a century, the way to treat Type 1 diabetes has been via insulin injections. Although research has been conducted on pancreatic islet transplants, it’s a tough nut to crack in terms of organ rejection. There’s been a potential breakthrough in that area involving nanotherapy.
World tennis superstar Novak Djokovic reportedly has an 80% stake in QuantBioRes that’s currently working on finding a treatment, not a vaccine, for COVID-19.
Dr. Anthony Fauci expects the FDA will authorize the Pfizer-BioNTech vaccine for children under the age of five in the next month.
ImmPACT Bio provided an optimistic update on a Phase I clinical study evaluating its CD19-CD20 bi-specific Chimeric Antigen Receptor T-cell (CAR-T) therapy.