Back when the patent war was brewing over CRISPR/Cas9, the Broad Institute's Feng Zhang made it clear that he believed the big innovations in gene editing still lay ahead. And in the last few days he set out to prove he was right, pointing to an alternative to Cas9 that could wind up serving as a much better tool to use to perform surgery on a gene.
A two-drug combo that matches Afinitor with an experimental compound proved effective in killing pancreatic cancer cells and blunting tumor growth in cell lines and mouse models for the lethal disease. Investigators at the University of Florida combined Afinitor with a synthetic compound coded PF-04554787. They targeted a protein known as focal adhesion kinase, or FAK, which triggers an enzyme called AKT, which in turn assist in the survival of islet cells in the pancreas. When islet cells start turning into tumors, the FAK protein is overproduced, helping pancreatic neuroendocrine tumors to resist chemo. Read more >>
Investigators at the Salk Institute have blazed a new trail branching out from the closely studied mGluR5 pathway in the brain, and they say it could ultimately lead to new therapies to treat neurodevelopmental disorders like autism and schizophrenia. Read more >>
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Matthew Shair, a professor of chemistry and chemical biology at Harvard, and his team have isolated and synthesized a molecule from sea sponges that prevents cancerous growth.
Now that Turing CEO Martin Shkreli has stirred a global frenzy over his move to jack up the price of toxoplasmosis drug Daraprim by more than 5,000%, investigators say they found that the high blood pressure drug guanabenz is effective against the latent cyst state of the parasite that causes toxoplasmosis. And as it's already approved, it could be quickly repurposed.
Scientists at Duke University Medical Center, the University of North Carolina, and the biotech MacroGenics have teamed up to design a molecule with the capacity to bind HIV-infected cells and unite them with the immune system's killer T cells.
Make no mistake. If a patient has a choice between a "breakthrough" drug and a rival therapy described in simple clinical terms, the breakthrough will win every time. That's the conclusion of a new study that tested out the impact that words like "breakthrough" and "promising" have when they're linked to a drug.
Researchers at Brigham & Women's Hospital have been exploring the links between the immune system and Alzheimer's, looking for new insights that could help guide drug development.
Researchers at Stanford University School of Medicine have found that combining two drugs that target the epigenome of cancer cells may be effective in killing off pancreatic tumor cells.
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After repeatedly batting back doubts about its lead gene therapy, Spark Therapeutics says its pivotal study for SPK-RPE65, an FDA-designated "breakthrough" for sight-blighting inherited retinal dystrophies, came through with positive data. The biotech says that the treatment hit the primary and two of three secondary endpoints in the late-stage study, setting up a biologics license application at the FDA next year.
Veeva Systems bought its nearest competitor in life science content management systems, Zinc Ahead, in a $130 million purchase that will combine the top two companies in the specialized field.