🔬 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
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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
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Santa Ana Bio, Inc. launches with $168M in Series A and B funding to develop precision therapies for autoimmune and inflammatory diseases, leveraging advanced proteomic, transcriptomic, and genomic techniques for targeted treatments. Their lead programs include SAB01, a bi-specific antibody targeting mast cells in allergic diseases; SAB03, a PD-1 agonist for rheumatoid arthritis; and SAB05, an antibody-glucocorticoid conjugate for severe inflammatory conditions, all set to enter clinical trials next year. More on this pipeline in our full coverage, link in the comments. #BiotechNews #PrecisionMedicine #AutoimmuneDisease #InflammatoryDiseases #RheumatoidArthritis #BiotechFunding
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R&D, Drug Development & Medical Affairs, Market Access Global Clinical Studies and Product Lifecycle Management, RA, Board of Directors, International Speaker, Development Programs, Strategic Leadership.
RINVOQ® (upadacitinib) Discovered and developed by AbbVie scientists, RINVOQ is a JAK inhibitor that is being studied in several immune-mediated inflammatory diseases. Based on enzymatic and cellular assays, RINVOQ demonstrated greater inhibitory potency for JAK-1 vs JAK-2, JAK-3, and TYK-2.3 The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness and safety is not currently known. Phase 3 trials of #RINVOQ #alopecia areata, #ankylosing spondylitis, atopic #dermatitis, axial spondyloarthritis, #Crohn's disease, giant cell arteritis, hidradenitis suppurativa, psoriatic #arthritis, rheumatoid arthritis, systemic #lupus erythematosus (SLE), Takayasu arteritis, ulcerative colitis and #vitiligo are ongoing.4-17 Use of #upadacitinib in vitiligo is not approved and its safety and efficacy have not been evaluated by regulatory authorities.
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Genome-wide protein quantitative trait locus study identifies genetic influence on inflammation-related proteins. Further insights on disease etiology and pathogenesis, such as shared and distinct effects of specific proteins across immune-mediated diseases, were obtained (i.e. Multiple Sclerosis, Rheumatoid Arthritis, Inflammatory Bowel Disease). Such insights can help prioritize drug targets in the future, as well as identify new targets for future therapies. 🗞 https://lnkd.in/dSayyWB6 #ScienceIsWeAll
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An Oral Interleukin-23–Receptor Antagonist Peptide for Plaque Psoriasis:- •The use of monoclonal antibodies has changed the treatment of several immune-mediated inflammatory diseases, including psoriasis. •However, these large proteins must be administered by injection. •JNJ-77242113 is a novel, orally administered interleukin-23–receptor antagonist peptide that selectively blocks interleukin-23 signaling and downstream cytokine production. •#mabs #cytokines #interleukin
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The #cGAS/STING pathway has become a pivotal focus for autoimmune disease therapy. By targeting #cGAS/STING, there is potential to provide benefits to patients with autoimmune diseases while avoiding widespread disruption of immune response integrity, which could lead to serious safety concerns, such as inhibiting IFN receptors. This pathway's activation in diseases like #SLE and #CLE, while absent in healthy individuals, underscores its crucial role in orchestrating immune system responses, particularly through the activation of type I IFN and NF-kB. Despite a decade of robust biological understanding, the development of potent drugs to effectively suppress the cGAS/STING pathway has posed significant challenges. Exo Therapeutics (https://lnkd.in/ei_SaDza) is working on an oral #TBK1 exosite inhibitor that specifically blocks the #STING/TBK1 signals, showing promise for treating autoimmune conditions such as #SLE, #CLE, and #Systemicsclerosis. For more information on Exo Therapeutics' programs and collaboration opportunities, please contact info@exo-therapeutics.com or send me a direct message. Sources: https://lnkd.in/eH4wSinK, https://lnkd.in/ei_SaDza
Biotechs step on cGAS for autoimmune diseases
nature.com
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NEW STUDY - First biopsy-confirmed case of typical cutaneous sarcoidosis with pulmonary involvement after C-19 injection. 2 days before the onset of the first lesion, she received a second dose of mRNA-1273. Sarcoidosis is an idiopathic granulomatous disease characterized by dense epithelioid, non-necrotizing granulomas in various organs, such as the lungs, skin, eye, heart, joints, and kidneys. "Vacc... likely induces sustained antiviral memory T-cell responses in both CD4+ and CD8+ subsets. Considering that activated CD4+ T helper 1 (Th1) cells and interferon (IFN)-γ are crucial for inflammation and subsequent granuloma formation in sarcoidosis, Th1-type inflammation characteristic of vacc... might trigger sarcoidosis in genetically susceptible populations." "The causal relationship between SARS-CoV-2 mRNA vacc... and the onset of cutaneous sarcoidosis may be considered “possible” according to the Naranjo’s adverse drug reaction probability scale." https://lnkd.in/g2S8cGmC #MFScholar
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This is an interesting study on gut microbiota and metabolites as potential biomarkers to predict outcomes in patients receiving immune checkpoint inhibitors. Did you know, Pythia Biosciences and Biorelate Ltd. has experience working with both these data types? If your interested in exploring this as part of your biomarker strategy we can help. https://lnkd.in/d6bf2sXa
Gut microbiota and metabolites associate with outcomes of immune checkpoint inhibitor–treated unresectable hepatocellular carcinoma
jitc.bmj.com
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This special section highlights the significance of sphingolipids in human disease and the potential for both sphingolipids and their metabolizing enzymes to serve either as therapeutic targets or as prognostic and diagnostic biomarkers. Read them here: https://bit.ly/3IzVT9H. #MolecularPharmacology #pharmacology
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Fatal infections of dogs with SARS-CoV-2 are associated with severe diffuse alveolar damage, pulmonary hyalinization and fibrosis. As with humans, virus, viral genomes, and the viral spike, can be found in multiple tissues and affect multiple organs, including lung, kidney, brain, trachea, tonsil, tracheobronchial lymph node, liver, and intestine. The authors highlight that fatal Covid infections in dogs are infrequent. The study was performed by Thai researchers at Chulalongkorn University (Bangkok) #covid19 #dogs #Thailand #health #globalhealth #publichealth #medicine #biotechnology #pharmaceuticals #FDA #WHO #CDC https://lnkd.in/gmiB2UjN
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