In a recent publication in Cell, researchers from the National University of Singapore and collaborators have proposed using commensal bacteria in the nasal cavity as a delivery vector for precision therapy targeting the OE and brain.
Junevity Inc. has raised $10 million in seed funding to support its work creating silencing RNA (siRNA) therapeutics to address metabolic and age-related diseases, including type 2 diabetes, obesity and frailty. The seed funding will be used to enhance the company’s RESET platform and develop its first therapeutic candidates in these indications.
Multiple sulfatase deficiency is an autosomal recessive lysosomal storage disorder caused by homozygous or compound heterozygous loss-of-function mutations in the SUMF1 gene, which encodes formylglycine generating enzyme (FGE), which catalyzes the post-translational modification of all known 17 sulfatases.
Adrenomedullin, a hormone first identified in an adrenal medullary tumor, disrupts the effect of insulin on the endothelium of blood vessels, leading to insulin resistance linked to obesity and type 2 diabetes. The clue to this discovery lies in a molecular pathway that could be blocked to restore insulin function.
Researchers from Huazhong University of Science and Technology have described the potential use of RBM47 as a therapeutic target for thyroid-associated ophthalmopathy, an autoimmune disorder characterized by proptosis, lid swelling, diplopia and compressive optic neuropathy.
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.
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