After a 10-year project and a £60 million (US$80 million) investment, the UK Biobank has completed the whole body scans of 100,000 volunteers and is making the 1 billion images available for researchers worldwide. Read More
Idiopathic pulmonary fibrosis (IPF) is a progressive disease characterized by lung scarring, fibrosis and finally respiratory failure. Currently, few treatment options are available for IPF and they only slow down disease progression and do not reverse fibrosis. There is a need for new therapeutic targets that aid in the management of the disease. Read More
The U.S. FDA has cleared Genetic Leap’s IND application for GL-IL2-138, a small-molecule RNA drug that modulates natural IL-2, allowing for downregulating or upregulating of the immune system to fight diseases. Read More
Voyager Therapeutics Inc. has expanded its Alzheimer’s disease (AD) pipeline with the addition of a wholly owned program that modulates the expression of apolipoprotein E (APOE). Using a proprietary intravenous-delivered, blood-brain barrier (BBB)-penetrant Tracer capsid, the product delivers a bifunctional payload. Read More
Amphix Bio LLC has been granted U.S. FDA orphan drug designation for its lead candidate AMFX-200 for the treatment of acute spinal cord injury (SCI). AMFX-200 is an FGFR (fibroblast growth factor receptor) and ITGB1 (integrin β1) agonist peptide amphiphile scaffold. In preclinical models of acute SCI, a single injection of AMFX-200 into the spinal cord enabled motor neurons from the brain to regrow past the injury site. Read More
The Cystic Fibrosis Foundation has agreed to provide Prime Medicine Inc. an additional investment of up to $24 million to continue the development of a gene editing therapy for people with cystic fibrosis. Prime Medicine uses a gene editing technology called prime editing, which enables a wide range of modifications to the DNA with a high degree of precision. Read More
Work at Childrens Medical Center Corp. has led to the identification of new gasdermin-D (GSDMD) activators reported to be useful for the treatment of cancer. Read More
Myocardial hypertrophy is a condition characterized by thickening of the ventricular wall and commonly associated with progression to heart failure. It develops when the heart is subjected to biomechanical stress or neurohormonal or hemodynamic stimuli. Read More
A recent Merck Sharp & Dohme LLC patent details new substituted isoquinoline derivatives acting as tyrosine-protein phosphatase non-receptor type 1 (PTPN1; PTP-1B) and/or PTPN2 (TCPTP) inhibitors potentially useful for the treatment of cancer. Read More
Abdominal aortic aneurysm is a life-threatening cardiovascular disease characterized by chronic inflammation and irreversible dilatation of the abdominal aorta and is asymptomatic at the early stages of the disease, with diagnosis usually relying on imaging techniques and surgery being the main approach for treating it. Read More
Stimulating the production of β cells in the pancreas of individuals with type 1 diabetes may be a way to renormalize their glucose metabolism. Researchers from Spain and the U.S. may have moved one step closer to this goal by stimulating formation of new β cells in the pancreas of a mouse model of diabetes using the cyclic peptide THR-123, which binds and activates the receptor for bone morphogenetic protein (BMP). Read More
Abarceo AB has developed new voltage-dependent anion-selective channel protein 1 (VDAC1) inhibitors reported to be useful for the treatment of diabetes type 1 and 2. Read More
AC Immune SA has synthesized new heterocyclic modulators acting as NLRP3 inflammasome inhibitors. As such, they are reported to be potentially useful for the treatment of asthma, atherosclerosis, obesity, gout, cryopyrin-associated periodic syndromes, nonalcoholic fatty liver disease (NAFLD), Parkinson’s and Alzheimer’s disease, among others. Read More
Discoidin domain receptor tyrosine kinase 1 (DDR1) contributes to tumor progression by promoting the alignment and densification of collagen fibers within the extracellular matrix (ECM), thereby facilitating the development of an immune-excluded tumor microenvironment (TME). Read More
