Fabry disease is an X-linked lysosomal storage disorder caused by a deficiency of the enzyme α-galactosidase A (GLA), which leads to complex multisystemic involvement, with the majority of patients developing hypertrophic cardiomyopathy.
Arbor Biotechnologies Inc.’s ABO-101 has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of primary hyperoxaluria type 1 (PH1).
Entos Pharmaceuticals Inc. has been awarded a $4 million grant by California Institute for Regenerative Medicine (CIRM) to support the completion of IND-enabling activities with ENTLEP-001, a durable genetic medicine for the treatment of congenital generalized lipodystrophy.
Cour Pharmaceuticals Development Co. Inc. has obtained IND clearance from the FDA for CNP-103, a nanoparticle in development to address the underlying autoimmunity of type 1 diabetes. A first-in-human phase Ib/IIa trial will begin later this year and will enroll adults (aged 18-35 years) and pediatric patients (aged 12-17) who have stage III or newly diagnosed (within the last 6 months) type 1 diabetes as well as C-peptide >0.2 ng/mL.
Jiangsu Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. have divulged proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitors acting as LDL-lowering agents reported to be useful for the treatment of stroke, hepatic steatosis, dyslipidemia, coronary artery disease, metabolic syndrome and atherosclerosis.
Er Stress Research Institute Inc. has disclosed benzothiazoimidazolyl compounds acting as endoplasmic reticulum (ER) stress inhibitors reported to be useful for the treatment of cancer, diabetes, neurodegenerative, autoimmune and metabolic diseases as well as renal, cardiovascular and renal disorders.
Glucocorticoid-induced osteoporosis, the leading cause of secondary osteoporosis, is characterized by diminished bone density and compromised osteoblast function. As current treatment options often have significant side effects, researchers are actively seeking new drug candidates to improve patient outcomes.
Hypoxia-inducible factors (HIFs) are crucial to maintain oxygen homeostasis by regulating cellular metabolic adaptation under hypoxia conditions. Depletion of factor inhibiting HIF (FIH), an enzyme that negatively regulates the activity of the HIF-1α isoform, has been associated with reductions in hepatic steatosis and body mass in mice.
Researchers from Fundación Jiménez Díaz and affiliated organizations have published data from a study that aimed to identify new genes involved in the progression of atherosclerosis, a chronic multifactorial inflammatory disease characterized by the accumulation of lipids and leukocytes within the arterial wall.
Bioage Labs Inc. has nominated BGE-102 as a development candidate. The orally available, small-molecule NLRP3 inhibitor has high potency and high brain penetration.
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