The U.S. Food and Drug Administration accepted Alnylam Pharmaceuticals Inc.’s New Drug Application for givosiran – an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 in development for the treatment of acute hepatic porphyria – and granted Priority Review for the NDA.
In a deal-making morning for South San Francisco-based Genentech, the Roche subsidiary struck three separate transactions with small companies to boost drug development.
Researchers with Massachusetts General Hospital have identified some crosstalk communication between TREM2 and CD33, two genes that play a role in inflammation and Alzheimer’s disease.
Working in the rare disease space has never been more rewarding than it is today. Technology is turning what was once thought to be impossible into actual, tangible realities for physicians, patients, and families. There are many exciting advancements happening now – in real time – and even more on the horizon that demand our attention and advocacy to help bring them to light.
Johnson & Johnson expects to file marketing applications for at least 10 new drugs between 2019 to 2023, to strengthen the company’s pharma unit which has been a major growth driver.
With a $75 million infusion of new financing, Massachusetts-based Arrakis Therapeutics will focus the company’s pipeline on oncology and genetically validated targets in other diseases.
Six months after forging a hepatitis B drug development deal valued at more than $3.7 billion with Arrowhead Pharmaceuticals, Janssen Pharmaceutical presented data that showed the RNA interference (RNAi) therapeutic candidate exhibited robust effects on the liver disease that takes the lives of more than 900,000 people annually.
Regeneron Pharmaceuticals is investing $800 million in Alnylam Pharmaceuticals to develop treatments for eye and central nervous system diseases using gene-silencing RNA interference technology.
ViiV Healthcare presented three-year data from the SWORD 1 and 2 clinical trials in HIV.
The return on R&D investment for leading biopharmaceutical manufacturers fell to a nine-year low while the U.S. FDA approved a record-breaking amount of novel medicines during 2018.
