Omniscope’s Post

Renatus Inc. today presented data on RN-005, a cholesterol metabolism modulator, in two preclinical chronic kidney disease models at the 2023 Scale-up Challenge Lab Conference. Renatus Inc., a pharma company focusing on cholesterol metabolism modulators that can address dysregulated cholesterol homeostasis in varying diseases, today announced preclinical data on RN-005 in diabetic nephropathy (DN) and focal segmental glomerulosclerosis (FSGS) models at the 2023 Scale-up Challenge Lab Conference in Incheon, South Korea. Cholesterol accumulation and dysregulated cholesterol homeostasis is commonly observed in the kidney of patients with chronic kidney disease (CKD), which affects more than 10% of the general population worldwide. Excessive cholesterol accumulation causes lipotoxicity, which can ultimately lead to kidney dysfunction and failure. Unfortunately, there is no drug available in the market that can directly target intracellular cholesterol and promote its efflux. Renatus' lead candidate, RN-005, is a novel synthetic cyclodextrin that can modulate cholesterol metabolism and promote its efflux. "In both chronic kidney disease models, RN-005 showed consistent renoprotective effects by decreasing blood creatinine and BUN levels and albuminuria. We also found that RN-005 effectively lowered renal cholesterol and triglycerides contents, which are significantly eleveated in the disease model," said Heegon Kim, CEO of Renatus. "These findings suggest that targeting renal cholesterol, not systemic cholesterol, can provide a new therapeutic option for the treatment of chronic kidney disease." New data also highlighted that RN-005 exerted renoprotective effects at a much lower dose than hydroxypropyl-β-cyclodextrin (HPβCD), which is being tested in clinical trials for the treatment of varying cholesterol-driven diseases. Due to the low dose requirement, RN-005 is being developed for subcutaneous formulation, as opposed to HPβCD that requires high dose administration and therefore should be infused over hours, for both human and companion animals. #cyclodextrin #RN005 #chronickidneydisease #animalhealth #veterinarymedicine #diabetickidneydisease #focalsegmentalglomerulosclerosis

Although the greatest contributors to #rheumatoid #arthritis (RA) heritability are the major histocompatibility complex (#MHC) proteins -encoded by the human leukocyte antigen (#HLA) region on chromosome 6-, 34 low-frequency and rare genetic variants have been associated with RA, including variants in #immune-related #genes such as #TYK2 that might represent therapeutic targets. 🔗https://lnkd.in/dSdVwhES

🔬 Enhanced Enzyme Delivery for Pompe Disease Pompe disease is a severe genetic disorder caused by the buildup of glycogen in the body's cells, leading to progressive muscle weakness and respiratory problems. Even if treated with the current standard treatment, enzyme replacement therapy (ERT), those with infant-onset Pompe rarely survive beyond adolescence.   At Sutura Therapeutics, we're pioneering improved treatments for Pompe disease using our innovative stapled peptides. By conjugating the therapeutic enzyme to these peptides we have been able to significantly enhance its effectiveness and delivery to the muscle cells.   A fluorescent reporter protein vividly demonstrates this advancement, showcasing how our peptides can achieve greater cellular uptake.   Stay connected with us to learn more about how our solutions are transforming the landscape of Pompe disease treatment.   #PompeDisease #RareDiseases #HealthcareInnovation

Abyntek Biopharma, mentioned in Nature Portfolio!   Access the latest research summary from our blog, published in the high-impact journal Nature, where #bioreagents from the Abyntek Research Reagents brand, such as our #ELISA kits, have played a significant role in the detection and measurement of #catecholamines.   Under the title “Methionine adenosyltransferase 1a antisense oligonucleotides activate the liver-brown adipose tissue axis preventing obesity and associated hepatosteatosis”, various research centers involved detail the most significant finding as the inhibition of the Mat1a gene, which reverses and prevents obesity, insulin resistance, and associated hepatosteatosis by reducing serum lipids. It is demonstrated that Mat1a deficiency has beneficial effects on metabolism and brown adipose tissue (BAT) function, as well as on lipid synthesis in the liver.   👉 Continue Reading in our blog: https://lnkd.in/dZ6T_md5   🔬 Discover Abyntek Research Reagents, used brand in the reagent panel for this research: https://lnkd.in/db2NPwb3   #Biotech https://lnkd.in/dZ6T_md5

Metabolic shift changes glycosylation of proteins driving development of ILDs in a positive feedback loop. In our latest review we desribe this phenomenon and propose novel therapeutic ideas, such as inhibition of CHIT1, which by modulating glycosylation exerts therapeutic effects. CHIT1 inhibitor is currently in phase II in patients with lung sarcoidosis. Primary endpoint captured by [18F]FDG PET/CT measures granuloma metabolic activity. https://lnkd.in/dvfMvXkf #OATD-01 #chitinase #glycobiology #glycolysis #sarcoidosis #immunometabolism

ARMGO Pharma and the National Institutes of Health have collaborated on a Phase 1b study of Rycal ARM210, a treatment for Ryanodine Receptor Type 1 (RyR1) Related Myopathies (RyR1-RM), an orphan muscle disease. RyR1 is an intracellular calcium-release channel vital to muscle contraction. Mutant RyR1 channels cause intracellular calcium leaks that impair muscle contraction, leading to muscle weakness and loss of function. The trial demonstrated that ARM210 is safe and well-tolerated, and it improved muscle strength and alleviated fatigue in patients with RyR1-RM. ARMGO Pharma is hopeful that ARM210 can also be used to treat Catecholaminergic Polymorphic Ventricular Tachycardia, a genetic life-threatening arrhythmia caused by mutations in RyR2. Read more about this development in the treatment of orphan muscle diseases at https://lnkd.in/gxqfTjiw. #RyanodineReceptor #CalciumChannels #RareArrhythmias #MuscleHealth #MedicalResearch

November is National Diabetes Month. Type 1 diabetes is a chronic condition in which the pancreas makes little to no insulin, the hormone responsible for allowing sugar to enter cells to produce energy. At Endsulin, we are developing a revolutionary genetic therapy for T1DM that with a single administration would restore the body’s own ability to produce and regulate insulin. Learn more about our science at https://lnkd.in/gZvwGqQk

➡️Non-alcoholic fatty liver disease (NAFLD) affects 1 in 4 people worldwide, unfortunately there's no medication that directly treats this condition. When left untreated, NAFLD can lead to liver cirrhosis and damage. ➡️Researchers at the University of Aberdeen discovered that individuals with a variant of the GPR75 gene were less likely to develop NAFLD. ➡️Eliminating the GPR75 gene also prevented fatty build up in the liver of mice fed the western diet. ➡️Efforts are currently ongoing to test the efficacy and safety of targeting the GPR75 gene for NAFLD prevention.

Insulin-like Growth Factor Binding Protein 4 (IGFBP-4) IGFBP-4 is a member of the insulin-like growth factor binding protein (IGFBP) family, which plays a crucial role in regulating the activity of insulin-like growth factors (IGFs). IGFs are potent signaling molecules involved in various physiological processes, including growth, development, differentiation, and metabolism. Therapeutic studies can help determine the effectiveness of targeting IGFBP-4 as a potential treatment for various diseases, including cancer, diabetes, and growth disorders. #bioinformatics #bioinformatician #computationalbiology

Positive results from the phase 1b/2a SELECT-HD trial assessing WVE-003 (Wave Life Sciences), an investigational first-in-class, allele-selective antisense oligonucleotide for Huntington disease, showed a significant reduction in mutant huntingtin protein and preserved healthy protein huntingtin protein in patients with HD, with no serious adverse events. https://lnkd.in/eb-taUTZ