Vesiculab’s Post

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

Source: Cell death & disease Study explores HRD1's impact on ER stress-induced apoptosis in intestinal I/R via TMEM2 ubiquitination, revealing a non-canonical pathway. HRD1 regulates ER stress-mediated apoptosis by ubiquitinating TMEM2, inhibiting PI3k/Akt activation. Targeting HRD1/TMEM2 axis could offer new therapeutic avenues for intestinal I/R.

Absolutely thrilled to present our paper accepted for publication in #JMedChem. #Strongyloidiasis is a neglected tropical disease caused primarily by the roundworm Strongyloides stercoralis. It is estimated to infect more than 200 million people worldwide with up to 2.5% of these infections progressing into a hyperinfection syndrome and a mortality rate of 90% if untreated. In preclinical models, the activation of ssDAF-12 receptor was able to suppress infection and to reduce lethality. Despite these remarkable evidences, only scarce efforts have been devoted to the field. In this work, we report the discovery of unprecedented ssDAF-12 modulators that have the potential to interfere with the lifecycle of the nematode Strongyloides stercoralis and to modulate the parasitic infection making it vulnerable to therapeutic intervention. We provide novel insights into the SAR profile and properties of steroidal modulators that might enable progress in targeting ssDAF-12 for treating #parasitism. It has been a long journey with a great collaborative effort from the chemical #synthesis of challenging #steroids to #computationalchemistry, #screening assays and #metabolic studies. Thanx and congrats to all involved Giada Ceccarelli, Laura Goracci, Andrea Carotti, Federico Paccoia, Daniela Passeri, Marco Cipolloni, Stefano Di Bona, Gabriele Cruciani, roberto pellicciari Università degli Studi di Perugia Tes Pharma S.r.l. Molecular Horizon #MedChem #Strongyloidiasis #Parasitism #Drugdiscovery Have a look: https://lnkd.in/gFVpXkpJ

😎 Wow~ Our customers published a paper in Signal Transduct Target Ther! Let's take a look.   👨🔬 👩🔬 Systemic lupus erythematosus (SLE) is a severe autoimmune disease characterized by systemic inflammatory response, accumulation of autoantibodies, and organ damage. Moreover, Neddylation is an important type of protein post-translational modification (PTM) that has been well documented to regulate T cell-mediated immune responses. However, the role of neddylation in SLE remains unclear. 🧐   👏 👏 This paper revealed for the first time the key mechanism by which Neddylation modification regulates the occurrence of SLE and provided a new potential target for the treatment of SLE.   ✔ https://lnkd.in/gb8gQJqH   ✔ For more push, please follow #Twitter: https://lnkd.in/gntQ9qE7   ✔ Related Product Recommendation:   #Pevonedistat (#MLN4924; https://lnkd.in/g737T-UY); #MG132 (#Z-Leu-Leu-Leu-al; https://lnkd.in/gy33AFtQ); #CD4 Antibody (https://lnkd.in/gG_whANd); #IL1β (https://lnkd.in/gZcE-dDH); #TNFα (https://lnkd.in/ge42cc9Z); #JC1 Mitochondrial Membrane Potential Assay Kit (https://lnkd.in/gefEhc3);   #SignalTransductTargetTher #SLE #Neddylation #PTM #MCE #MedChemExpress

MD Bioproducts' Aggrecan monoclonal antibody to N-terminal neoepitope FFGV (Mouse, Clone BC-14): This fragment is rapidly released from tissue when MMP catabolism of aggrecan occurs and has been identified in synovial fluid samples with degenerative joint diseases. Product Link: https://lnkd.in/dVereHDc #cartilage #musculoskeletal #rheumatology

Defects in regulatory T cells (Tregs) have been linked to autoimmune diseases, including systemic lupus erythematosus (SLE). Interleukin 2 (IL-2) is crucial for the development and function of Tregs. Due to the constitutive expression of the alpha chain of the IL-2 receptor (CD25) on Tregs, a preferential CD25 agonist could be beneficial in expanding the Treg population in patients with autoimmune conditions.   This Phase 1b trial evaluates efavaleukin alfa, an Fc-IL-2 mutant that selectively binds to CD25, to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in 35 patients with SLE.   The results showed that the peak expansion of FoxP3+ Tregs occurred eight days post-dose. Treatment with efavaleukin alfa significantly increased the CD25^hi Treg population by 53.8-fold, while conventional T cells expanded by 2.9-fold. #Lupusresearch #Tregresearch #Autoimmunity #SLE #Fusionproteins #Smallmolecules # #Efavaleukinalfa https://lnkd.in/eH_JEeHy

The Role of Complement Blockade in the Management of IgA Nephropathy Exciting developments in treating immunoglobulin A nephropathy (IgAN)! 🌟 Traditional systemic immunosuppression comes with significant toxicities, but new therapies targeting the complement pathway offer hope. Complement activation is a key driver of IgAN, leading to kidney injury and disease progression. Cemdisiran, an RNA interference therapeutic, is one such promising treatment. In a recent phase 2 study, cemdisiran showed significant reduction in proteinuria levels compared to placebo. Common adverse effects were manageable, and no patient discontinued treatment due to side effects. While further research is needed, this study highlights the potential of complement pathway targeting in IgAN management. With new therapies on the horizon, there's renewed optimism for better outcomes in IgAN patients. #IgAN #ComplementPathway #NewTherapies 🩺💊 https://lnkd.in/d2i-y6pf

Exciting advancements in cystic fibrosis treatment! A recent case report and literature review by Tupayachi Ortiz et al. showcases the positive response to Elexacaftor/Tezacaftor/Ivacaftor in individuals with the N1303K mutation. This study sheds light on the promising outcomes of this treatment combination. A must-read for healthcare professionals seeking to stay updated on the latest in CF therapy.

In a world-first, new Blood-group System Discovery Could Save Thousands of Lives Worldwide a team of researchers has identified a new blood group system, called Myelin and Lymphocyte protein MAL. The new MAL system is the 47th to be discovered. Its significance lies in the potential to prevent ARs in people with a rare phenotype known as AnWj-negative. AnWj antigen, is a marker on the surface of RBCs whose genetic origin had been unknown for more than five decades. Although the most well-known blood group systems are ABO and Rh. The landscape is much more complex. Numerous antigens exist on the surface of RBCs and must be considered during transfusions, as an incorrect combination can trigger severe reactions in patients. Mal protein is expressed on RBC membranes of AnWj-positive, but not AnWj-negative, individuals. Individuals who lack this antigen on the surface of their RBCs (due to a genetic mutation or disease), may experience serious complications if they receive AnWj-positive blood. This discovery has Global significance as it represents a major milestone in blood transfusion safety, offering hope to patients with rare blood types and improving global healthcare outcomes. Source: "Blood" #researchanddevelopment #innovation #rarebloodgroup #bloodtransfusion #healthcare #lifesciences #biotechnology

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