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.
Sensorium Therapeutics Inc. has identified alkylated derivatives of mesembrine or mesembrenone acting as serotonin transporter (SERT) and/or phosphodiesterase PDE4 inhibitors reported to be useful for the treatment of anxiety, depression and stress disorders.
Rome Therapeutics Inc. has synthesized 4’-halomethyl-cytidine phosphoramidate compounds acting as LINE-1 retrotransposable element ORF1 protein (L1RE1) and/or HERV-K reverse transcriptase inhibitors reported to be useful for the treatment of viral infections, cancer, inflammation, neurodegeneration and immunological disorders.
Shenzhen Borui Pharmaceutical Technology Co. Ltd. has disclosed Mas-related G-protein coupled receptor member X4 (MRGPRX4; SNSR5; SNSR6) antagonists reported to be useful for the treatment of autoimmune disease, cancer and more.
Researchers from Landeskrankenhaus Salzburg (SALK) - Universitätsklinikum der PMU have identified a novel loss-of-function variant in the NDUFA7 gene in a patient with Leber’s hereditary optic neuropathy (LHON).
Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co. Ltd. has received IND clearance in China by the National Medical Products Administration (NMPA) to conduct a phase I trial of its antibody-drug conjugate (ADC) FZ-AD005 for advanced solid tumors.
The FDA has granted orphan drug designation to Be Biopharma Inc.’s BE-101, a novel engineered B-cell medicine being developed for the treatment of hemophilia B.
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