Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).
Sangamo Therapeutics Inc. has received IND clearance from the FDA for ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to idiopathic small fiber neuropathy (iSFN).
Astrazeneca plc has developed a potent oral fibroblast activation protein (FAP) inhibitor, AZD-2389, that avoids the cleavage of FGF21 and α2-AP. AZD-2389 was tested in cynomolgus monkeys with diet-induced MASH.
DAN-222 is a polymeric nanoparticle covalently conjugated with the topoisomerase I inhibitor camptothecin, which has previously demonstrated promising clinical activity in heavily pretreated patients with breast cancer (NCT05261269).
Hypertrophic cardiomyopathy (HCM) is the most common genetic cardiovascular disorder with around 85% of people with HCM remaining undiagnosed. There are no treatments approved for nonobstructive HCM (nHCM) to date.
Eli Lilly & Co. has elected to pursue the development of therapeutics against two validated drug targets for amyotrophic lateral sclerosis (ALS) from its collaboration with Verge Analytics Inc. (dba Verge Genomics).
Recurrence rates and acquired treatment resistance in colorectal cancer have been linked to colorectal cancer stem cells, which are a subpopulation of slowly dividing cells within the tumor environment.
Gilead Sciences Inc. has synthesized 4-aminopyrrolo[2,1-F][1,2,4]triazine C-nucleoside analogues acting as prodrugs of GS-441524 and reported to be useful for the treatment of viral infections.
Arkuda Therapeutics Inc. has patented new fused tricyclic mucolipin-1 (MCOLN1; TRPML1) blockers reported to be useful for the treatment of cancer, liver diseases, amyotrophic lateral sclerosis, cardiovascular disorders, frontotemporal dementia, Alzheimer’s, Huntington’s and Parkinson’s disease.
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