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Vertex Pharmaceuticals is halting work on an experimental drug for a rare protein deficiency after safety problems were reported in Phase 2 testing. The → Read More
Codiak BioSciences now has $82.5 million to fund clinical tests of a new class of medicines based on exosomes, tiny bubbles formed by cells that the → Read More
Roche made a splash with its acquisition of Spark Therapeutics, whose Luxturna, a gene therapy for an inherited form of vision loss, was the first such → Read More
Gossamer Bio’s lead drug, an experimental treatment for asthma and allergies, has failed in two mid-stage clinical trials but the biotech sees enough in → Read More
An opioid that Avenue Therapeutics reformulated for treating post-surgical pain in hospital settings has been rejected by the FDA due to safety concerns. → Read More
An Alkermes antipsychotic treatment designed to offer the benefits of a commonly prescribed medicine, but without the weight gain side effect, won the → Read More
Arrowhead Pharmaceutical’s gene silencing treatment for a rare liver disease will move through the regulatory process and potential commercialization with → Read More
The first generation of CRISPR-based therapies showed how the technology can edit DNA, making genetic fixes to unwanted mutations. But those treatments → Read More
Scientists at microbiome therapy developers often talk about the importance of community, how the bugs in our gut act in concert in addition to → Read More
Bristol Myers Squibb is bolstering its cardiovascular drug lineup with a $13.1 billion deal to acquire MyoKardia, a company whose experimental therapy for → Read More
Solid Biosciences has answered the FDA’s outstanding questions about its Duchenne muscular gene therapy, clearing a clinical trial to now resume after → Read More
Genfit is halting all work on its lead drug in the fatty liver disorder nonalcoholic steatohepatitis (NASH) and implementing a corporate restructuring → Read More
CStone Pharmaceuticals has touted its experimental drug sugemalimab as potentially the best in its class of cancer immunotherapies. Pfizer is placing a → Read More
Orphazyme has fresh cash from the sale of shares in the US and Europe, which will support its lead rare disease drug through regulatory review and, if → Read More
Praxis Precision Medicines kept mostly quiet about its research on central nervous system disorder (CNS) drugs until May, when it emerged from stealth and → Read More
Had things turned out differently, scientists at Galecto might be watching their idiopathic pulmonary fibrosis drug candidate develop in the hands of a → Read More
In biotech, you can go home again. Four years ago, Illumina spun out cancer diagnostics startup Grail. This week, the gene sequencing giant announced it → Read More
Many drugs work by binding to a protein and blocking it. Technology for eliminating disease-causing proteins altogether remains experimental, but this → Read More
Amyotrophic lateral sclerosis (ALS) has no cure and few available treatments but the past decade has revealed new knowledge about the pathology of the → Read More
An Alzheimer’s disease drug from partners AC Immune and Roche has failed a mid-stage clinical trial, adding another compound to the list of experimental → Read More