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. Read More
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. Read More
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. Read More
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.
Haihe Biopharma Co. Ltd. has disclosed benzo(hetero)cycloalkyl compounds reported to be useful for the treatment of acne, spinal and bulbar muscular atrophy, and more. Read More
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. Read More
Gan & Lee Pharmaceuticals Co. Ltd. has synthesized antibody-drug conjugates comprising a monoclonal antibody covalently linked to exatecan derivatives through a linker reported to be useful for the treatment of cancer. Read More
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. Read More
Ningbo Newbay Medical Technology Co. Ltd. has identified DNA polymerase θ (POLθ) inhibitors reported to be useful for the treatment of cancer. Read More
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. Read More
CSPC Pharmaceutical Group Ltd. has gained clinical trial approval from China’s National Medical Products Administration (NMPA) for SYH-2062 injection, a double-stranded small interfering RNA (siRNA) drug to treat hypertension. Read More
Insilico Medicine Ltd. has described protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of cancer. Read More
Xentria Inc. has entered into an exclusive license agreement with New York University (NYU) securing Xentria exclusive rights related to the development of a potential disease-modifying therapeutic for cardiovascular disease. Read More
