Neuroblastoma can be a particularly insidious cancer. In about half of all cases, tumors regress, even without therapy. In ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona ...

A proof-of-concept study finds that donated insulin-producing cells can be genetically modified to avoid provoking the recipient's immune system.

After a strong launch for its cancer gene therapy, Ferring Pharmaceuticals is setting the stage for long-term growth.

Arguably the FDA’s most anticipated decision this month is for a subcutaneous induction formulation of Biogen and Eisai’s ...

In medicine and biotechnology, the ability to evolve proteins with new or improved functions is crucial, but current methods ...

A 38-year-old man in Italy has regained his sight after undergoing a groundbreaking experimental gene therapy—the first of its kind in the world. The patient, diagnosed with Usher syndrome type 1b, a ...

Net loss per share (GAAP) was $0.62, but remained negative as the company continues to invest heavily in research and development. The pivotal Phase 2 trial for Danon disease was placed on clinical ...

Sarepta Therapeutics Inc.’s shares soared after the US biotech reported revenue that beat estimates, which analysts ...

For some patients with gastrointestinal (GI) cancers like colorectal and pancreatic cancer, chemotherapy can cause severe, ...

Jonathan D. Grinstein, PhD, Manfred Scholz, PhD, and Erik Wiklund, PhD, go behind the headlines to address a shifting policy landscape and what it means for the future of chronic disease, diagnostics, ...