University of Basel investigators have reported an association between mutations in the COQ8B gene and nonsyndromic retinitis pigmentosa (RP). RP is a genetically heterogeneous retinal degeneration disorder, which has been found to be associated with mutations in more than 70 genes. However, there are still cases for which no genetic cause has been found.
Nutcracker Therapeutics Inc. has disclosed a new drug candidate for the treatment of B-cell lymphoma – NTX-472 – along with results from in vivo studies performed to evaluate its efficacy in mice and macaques.
Alchemab Therapeutics Ltd. has been awarded a grant of $595,000 by The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support its Parkinson’s disease program.
Ovarian cancer stem cells are characterized by high activity of aldehyde dehydrogenase (ALDH) enzyme as well as high expression of CD133 marker, and the combination of both correlates with poor prognosis in patients.
Excessive inflammatory response and endothelial barrier dysfunction are the two major pathophysiological changes in acute respiratory distress syndrome (ARDS). Sphingosine-1-phosphate S1P3 receptor (S1PR3) is a G protein-coupled receptor involved in the regulation of inflammation and vascular barrier function in some diseases, but its function in ARDS is not fully understood.
An experimental drug that restored the normal function of ion channels in Alzheimer's disease (AD) prevented the loss of neurons and reduced the accumulation of amyloid-β (Aβ) plaques and hyperphosphorylated tau formed in this condition. A new class of small molecules, collectively called ReS19-T and developed by scientists at the Belgian biotechnology company Remynd NV, reorganized proteins that modulated calcium channels. Now in the clinical phase, this approach could benefit patients suffering from neurodegenerative disorders.
Fibrodysplasia ossificans progressiva is a rare and life-threatening genetic disease caused by gain-of-function mutations in the ALK2 gene, which encodes activin receptor-like kinase 2. Blueprint Medicines Corp. has elucidated the discovery of their ALK2 inhibitor BLU-782, which is now in phase II studies at Ipsen for the treatment of FOP.
Obesity specialist Sixpeaks Bio AG emerged from stealth with $110 million in funding and an option to be acquired by Astrazeneca plc within the next two years, subject to filing an IND for the lead product. Of the $110 million, $30 million is a series A round, with the balance to come from Astrazeneca, which will provide nondilutive finance of up to $80 million.
Hangzhou Glubio Therapeutics Co. Ltd. has described proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase protein-binding moiety covalently linked to a Wee1-like protein kinase (Wee1)-binding moiety reported to be useful for the treatment of cancer.
Chiesi Farmaceutici SpA has divulged TGF-β receptor type-1 (TGFBR1; ALK5; SKR4; TβR-I) inhibitors reported to be useful for the treatment of idiopathic pulmonary fibrosis.
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