The discovery that thalidomide and its analogues work by inducing the degradation of transcription factors demonstrated both that protein degradation is a viable mechanism of action for therapeutic small molecules, and that it could be used to target transcription factors, which are undruggable by both biologics and traditional inhibitors.
BOSTON – Researchers presented data at The Liver Meeting 2019 and in Nature this week demonstrating that bacteriophages targeted to specific members of the microbiome could be useful in fighting two different liver diseases.
BOSTON – In a late-breaking oral presentation on the last day of The Liver Meeting 2019, Durect Corp. reported results from its phase IIa trial of DUR-928 for the treatment of alcoholic hepatitis. All 19 patients enrolled in the study survived for 28 days after treatment with DUR-928 , and all but two responded to the drug as measured by Lille scores, which is used for predicting risk of death.
BOSTON – For diagnosing nonalcoholic steatohepatitis (NASH), liver biopsy is "the reference standard," Dean Hum, president of Genfit Corp., told BioWorld. "I'm not going to call it the gold standard." Some of the reasons for denying biopsies a gold sticker are obvious. Biopsies are invasive, which makes them risky, expensive and loathed by patients and doctors alike. "In the real world, doctors don't always do biopsies – many say they very rarely do biopsies unless other data points in several directions and they need that for clarity," Gail Cawkwell told BioWorld.
Researchers at Harvard Medical School and The Banner Alzheimer's Institute have identified a potential protective variant that appears to have delayed the onset of clinical symptoms by several decades in one individual with a presenilin mutation. Presenilin mutations cause an autosomal dominant, early onset form of AD that usually leads to clinically noticeable cognitive impairments in one's 40s.
A subtype of schizophrenia is related to abnormally high brain levels of hydrogen sulfide (H2S), which has important implications for the development of new treatments, according to a study by researchers at the RIKEN Center for Brain Science (CBS) in Japan.
A Chinese study has identified small-molecule compounds that can selectively reduce levels of the mutant Huntingtin (HTT) protein involved in the pathogenesis of Huntington's disease (HD), and possibly other neurodegenerative diseases involving mutant protein accumulation.
BOSTON, Mass. – KRAS used to be the poster child for undruggability. No more. Amgen Inc. and Mirati Therapeutics Inc. are in the clinic with KRAS inhibitors. Boehringer Ingelheim GmbH is planning to join them by the end of the year. And multiple other companies are moving programs forward.
RSS
