Autosomal dominant polycystic kidney disease (ADPKD) is a severe genetic disorder primarily caused by mutations in the PKD1 or PKD2 genes, which encode polycystins 1 and 2, affecting over 12 million people globally.
Pimavanserin is a 5-HT2A inverse agonist that is FDA approved for treating Parkinson’s disease psychosis. Acadia Pharmaceuticals Inc. searched for a compound that relies on pimavanserin but with an improved profile, including shorter half-life and reduced QT prolongation risk, which led to the identification of ACP-204.
Wave Life Sciences Ltd. has submitted a clinical trial application (CTA) for WVE-007, an investigational GalNAc-conjugated small interfering RNA (siRNA) designed to silence inhibin βE (INHBE) gene expression, for the treatment of obesity.
Epirium Bio Inc. has obtained IND clearance from the FDA for MF-300, a first-in-class orally administered, 15-hydroxyprostaglandin dehydrogenase (15-PGDH) enzyme inhibitor.
Hervolution Therapeutics ApS (formerly Inprother Aps) has announced a $11.7 million series A financing to support its work developing novel human endogenous retrovirus (HERV)-targeted immunotherapies to address diseases of aging.
Engineered probiotics, mainly bacterial probiotics, are an emerging platform for in situ delivery of therapeutics, including anticancer therapies, to the gut. Writing in Cell Chemical Biology, researchers from Washington University School of Medicine and collaborators present a novel yeast-based therapeutic oral platform to deliver next-generation immune checkpoint inhibitor proteins directly to gastrointestinal tumors.
First, the good news about pandemics – and in 2024, there was big “good news.” Science Magazine named lenacapavir (Gilead Sciences Inc.) as the Breakthrough of the Year. In two separate trials, lenacapavir prevented HIV transmission with 100% efficacy in cisgender African women and 99.9% efficacy in men and gender-diverse persons when administered twice a year.
In a deal worth $100 million up front and up to $1.25 billion in milestone payments, Bioarctic AB licensed its pyroglutamate-amyloid-β (pyroglutamate-Aβ) antibody program to Bristol Myers Squibb Co. to advance treatments for Alzheimer’s disease.
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