Biotechnology Clinical Trials Gene Therapy rare diseases

Scientists make a major breakthrough to treat fibrotic diseases that cause organ failure

Researchers from Duke-NUS Medical School (Duke-NUS) and the National Heart Centre Singapore (NHCS) have discovered that a critical protein, known as interleukin 11 (IL11) is responsible for fibrosis and causes organ damage. While it is surprising that the importance of IL11 has been overlooked and misunderstood for so long, it has now been very clearly […]

Biotechnology cancer

Drugs, Diseases and Proteins: New Archive Helps Precision Medicine, Drug Development

UNM Cancer Center Scientist co-leads international drug targets effort to create open-source archive for drug discovery, development, and safety Most aspirin users don’t know what aspirin does to make you feel better. Most doctors and scientists didn’t know, either, until recently. In fact, scientists know how only a handful of drugs really work and they […]

Cell Therapy

Researchers Encouraged by Continued Signs of Tolerability and Disease Modification in Gene Therapy Trial for Sanfilippo Syndrome

Researchers from Abeona Therapeutics Inc, a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, provided, an update on clinical results through 30 days post-injection for the completed low-dose cohort (n=3) in the ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH) at the Orphan Drugs & Rare Disease Conference (London, UK). The first-in-man […]

Alzheimers and Dementia

Vesicles That Trap Amyloid Appear to Also Contribute to Alzheimer’s Disease

Vesicles, fluid-filled sacs that brain cells make to trap amyloid, a hallmark of Alzheimer’s, appear to also contribute to the disease, scientists report. Reducing the production of these vesicles, called exosomes, could help reduce the amount of amyloid and lipid that accumulates, slow disease progression and help protect cognition, scientists at the Medical College of […]

Uncategorized

How Do You Kill a Malaria Parasite? Clog It with Cholesterol

Drexel University scientists have discovered an unusual mechanism for how two new antimalarial drugs operate: They give the parasite’s skin a boost in cholesterol, making it unable to traverse the narrow labyrinths of the human bloodstream. The drugs also seem to trick the parasite into reproducing prematurely. Malaria is a mosquito-borne disease caused by Plasmodium […]