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Vertex Pharmaceuticals said a patient with a 40-year history of Type 1 diabetes experienced a 91 percent reduction in daily insulin dose after an infusion of its VX-880 stem-cell-derived islet cell replacement therapy. Read More
Tofersen, Biogen’s potential blockbuster for amyotrophic lateral sclerosis (ALS) caused by mutations of the SOD1 gene, has missed its primary endpoint of improved function in a highly anticipated trial. Read More
AZD1222, which is not authorized in the U.S., is currently approved for use in the UK and EU, while Valneva is not yet approved for use anywhere. Read More
Liver cancer patients who received AstraZeneca’s blockbuster human monoclonal antibody Imfinzi (durvalumab) paired with the company’s anti-CTLA4 antibody tremelimumab in a phase 3 trial lived longer. Read More
CRISPR Therapeutics’ off-the-shelf CAR-T treatment conferred clinical benefits similar to those achieved with CAR-T treatments derived from the patients’ own cells, posting a 58 percent overall response and 38 percent complete response rate in a small series of patients with large B-cell lymphoma. Read More
Real-world evidence demonstrated that a drug prescribed to treat swelling and high blood pressure could help reverse the effects of Alzheimer’s disease, claimed researchers from the San Francisco, Calif.-based Gladstone Institutes and University of California, San Francisco (UCSF). Read More
The neurodegenerative disease drug development pipeline is full, but getting investigative drugs over the FDA finish line is both a biological and regulatory challenge. Read More