DefiniGEN’s Post

NMD Pharma novel skeletal muscle-targeted ClC-1 ion channel has been featured in an article published by @Labiotech.eu, exploring the latest clinical developments that aim to enable those living with Charcot-Marie-Tooth disease (#CMT) to live better, more independent lives. The article highlights our recently initiated Phase 2 #ClinicalTrial of NMD670, an oral first-in-class small molecule inhibitor of the skeletal muscle specific ClC-1 ion channel, in patients with CMT types 1 and 2. By inhibiting this ion channel in those with neuromuscular deficits and disease,  NMD670 has the potential to increase neuromuscular transmission and improve muscle function to address the muscle weakness and fatigue that are characteristic of this #RareDisease, helping muscles to contract in order to provide increased muscle strength and endurance. To find out more, read the full article here: https://lnkd.in/dTJXq7Vz #ICYMI #NeuromuscularDisease #CharcotMarieTooth #Biotech #Innovation

🔬 Unlock New Possibilities with Complement System Humanized Mouse Models! 🧬 The complement system, one of the most ancient components of the immune system, is increasingly recognized for its roles beyond traditional immunity, including maintaining immune homeostasis, supporting development, and regulating synaptic pruning. Since the FDA's approval of eculizumab—the first complement-specific inhibitor—in 2007, interest in complement-targeting therapies has grown exponentially. At Biocytogen, we are driving this innovation forward with a suite of complement system-related humanized mouse models, featuring humanized genes such as C3, C5, and C5AR1/2. These advanced tools enable robust preclinical in vivo evaluation of your drug candidates against human targets, helping you explore the untapped potential of complement system therapeutics with confidence and precision. #ComplementTherapeutics #DrugDevelopment #HumanizedModels #PreclinicalResearch #Biocytogen #ImmuneSystem #Innovation

Meet the Conigen Experts Have questions about how advanced proteins can be used as antigens or immunogens? Looking for soluble dimeric proteins to develop deeper insights into your research? Wondering how your specific membrane proteins can be generated as an immunogen?   Meet the Conigen team of experts at AAI booth #337 and get insights into the latest strategies empowering translational research and drug discovery. Let's push the boundaries of research together! https://lnkd.in/eyZgGVwu   #AAI2024 #Immunology #InnovateWithConigen #AdvancedProteins

Introducing Hornet Therapeutics: Pioneering Treatment for EBV-Driven Diseases 🚀 Exciting news for Hornet Therapeutics, as they emerge from stealth mode with groundbreaking data published in Science! 📰 This marks a significant milestone in the fight against Epstein-Barr Virus (EBV) and its related diseases. 🔑Key Highlights🔑 🐝 Revolutionary Treatment: First-ever small molecule drug for EBV-driven diseases. 🐝 Published Data: Demonstrates decreased EBV latency with IDO-1 inhibition. 🐝 Strategic Partnership: Collaboration with Kyowa Kirin. 🐝 Clinical Trials Ahead: Proof-of-concept studies for EBV-driven PTLD in transplant patients. 🌟 What Makes the Lead Asset (HTX-201) & This Antiviral Approach Significant? 🌟 👉 Addresses a significant unmet need in solid organ transplant populations. 👉 Broad Potential: Future targets include Multiple Sclerosis, Mononucleosis, and long-COVID. #Biotech #MedicalInnovation #EBV #HealthTech #Science #Transplantation #MS #LongCOVID #AntiviralTherapy #Healthcare

In just two days, don’t miss our late-breaking abstract on inherited retinal disease caused by RPE65 mutations at the European Society of Retina Specialists (EURETINA) 2024 (#EURETINA24). Our Dr. Alvin Luk, Co-founder and CEO at HuidaGene Therapeutics , will be orally presenting the #CRISPR /Cas13 RNA-targeting therapy (HG202) clinical study “SIGHT-I” (NCT06031727) in AMD patients who have shown poor response or have developed tachyphylaxis with anti-VEGF therapies. This is the first #geneediting trial dosing humans beyond Cas9. Additionally, Dr. Luk will also orally present the AAV9-RPE65 #genetherapy (HG004) clinical trial “LIGHT” (NCT06088992) in LCA2 patients. With 25-7.5 folds lower than the approved Luxturna, the LIGHT study shows a superior safety profile and visual improvement than the approved Luxturna without progressive chorioretinal atrophy. Also, don’t miss our two audio-narrated free papers on preclinical studies in mice and non-human primates on RPE65-mediated inherited retinal dystrophy and age-related #maculardegeneration. #blindness #inheritedblindness #biotechnology #medicine #future #health #healthcare #innovation #pharmaceuticals #retina #technology

Inoviv is excited to announce the launch of FibroKey™, a non-invasive, mass spectrometry-based multiplex assay designed to monitor fibrosis-related #biomarkers with precision. FibroKey™ delivers valuable insights into the progression of chronic kidney disease (#CKD) and other #fibrotic conditions, supporting therapeutic developers and #biopharma in advancing their drug development efforts. Learn more about how FibroKey™ can drive your #fibrosis research forward - https://lnkd.in/g8YB2zZp #FibrosisResearch #BiomarkerDiscovery #FibrosisTreatment #ChronicKidneyDisease #PharmaResearch #ClinicalResearch #BiotechInnovation #DrugDevelopment #Proteomics #FibrosisBiomarkers

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Merck partners with small UK biotech to drug fibroblasts for fibrosis, I&I diseases: As Merck builds out its immunology research lab in London, it’s partnering with a local biotech startup that has remained relatively quiet since its seed round in 2021. The pharma giant said Tuesday morning it is pairing up with Cambridge, UK-based Mestag Therapeutics to find new targets for fibrosis and inflammatory disease treatments. Merck will deliver an upfront payment of undisclosed size to the nearly four-year-old Mestag, which is working on drugging fibroblasts. These cells are integral to the formation of connective tissue and also help communicate with immune cells and give them navigation orders. If all goes to plan, Mestag stands to gain as much as $1.9 billion from the yearslong partnership that the duo has inked. Mestag could deliver a “number of targets,” but the startup’s CEO, Susan Hill, declined to disclose how many to Endpoints News. Merck will be responsible for developing any drugs that arise from the pact. The move comes as Merck makes bold bets on the I&I field, dishing out $10.8 billion to buy Prometheus Biosciences last year and $700 million upfront to buy an early clinical-stage T cell engager from Curon Biopharmaceutical this summer. “We’re also very happy to work with a partner that is very actively and demonstrably building out inflammatory disease and autoimmune discovery capabilities,” Hill said in an interview. For Mestag, the money infusion gives runway to get closer to the clinic. It disclosed a $45 million seed round in 2021 from SV Health Investors, Johnson & Johnson Innovation, GV, Northpond Ventures and Forbion. It also worked on an inflammatory disease collaboration with J&J for undisclosed terms, but that tie-up is “completing” and coming to an end, Hill said. The three dozen-employee startup will likely begin fundraising a Series A in the first half of 2025 so it can get into the clinic with its first experimental drug about 15 months from now, Hill said. The company’s first program, MST-0300, is a bispecific antibody that is meant to agonize lymphotoxin beta receptors in solid tumors and co-engage fibroblast activation protein, or FAP. It aims to induce tertiary lymphoid structures, or TLS, in solid tumors to drum up an immune response to the cancer. “There’s an incredible amount of data in the literature now showing that patients that have TLS in their tumors do better on overall survival and PFS,” Hill said. Behind that project, Mestag has inflammatory and autoimmune programs, including checkpoint agonists that target myeloid biology rather than T cell biology that other hot I&I drug modalities are going after, the CEO said. For example, multiple drugmakers are attempting to turn CAR-T cell therapies and T cell engagers into I&I medicines. Hill said…

The September edition of our Literature Highlights & detailed slide decks are now available on the Lupus-Forum, featuring key advancements in lupus research and therapeutics. This month includes a first-in-human, double-blind, randomized, and placebo-controlled study evaluating the safety, pharmacokinetics, immunogenicity, and pharmacodynamics of single ascending intravenous (IV) and subcutaneous (SC) doses of DS-7011a in healthy subjects. 🔗https://ow.ly/tFp150Tw8WU

ELRIG UK Drug Discovery 2024 starts tomorrow! If you're looking for efficacy screening solutions, evaluating hepatic safety, or need iPSC-derived disease models for #MASLD, #A1ATD, Wilson's disease, or #PFIC2, we've got you covered! Stop by booth D24 and chat with Nicholas Clare, Malek Haddad, Haris Choudhery, and our CEO Chris Kirton. They'll be happy to show you how our advanced iPSC-derived models and screening services can help de-risk your drug discovery efforts. Also, make sure to catch Celine Gomez’s presentation (Poster 219) on iPSC-derived hepatocytes, highlighting their potential in modeling Metabolic dysfunction-Associated Steatotic Liver Disease (MASLD) in vitro. We can't wait to see you at booth D24! #DrugDiscovery #iPSC #CRISPR #Biotech #elrig #elrigdd24 #hepatocytes #liver #liverdisease #DefiniGEN

Tomorrow, our CEO Anker Lundemose will be at the Annual Seed🌱 Summit, the premier event welcomes portfolio companies, investors and pharma BD from across Europe and US for a full day of networking and discussions around company creation at the Novo Holdings HQ in Copenhagen. Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria which are significant drivers of disease pathophysiology in #acutekidneyinjury (#AKI), #ParkinsonsDisease (PD), #heartfailure, #DuchennesMuscularDystrophy (#DMD), #IPF, mitochondrial diseases and #alzheimers. Keep up to date with Mission Therapeutics by following us at Mission Therapeutics on LinkedIn or @MISSIONTherapeu on X / Twitter. You can also visit our website using the link in the comments below. #NovoHoldings #mitophagy #DUBInhibitors #parkinsons #alzheimersdisease