Monoclonal antibody Prasinezumab shows promise in slowing rapid Parkinson's progression. In a recent study published in the journal Nature Medicine , a large, international team of researchers conducted an exploratory analysis to evaluate whether the monoclonal antibody prasinezumab, which had previously been observed to be effective in slowing the progression of motor-associated signs of Parkinson’s disease, did indeed show benefits in subgroups of Parkinson’s disease patients with faster progression of motor degeneration... Check out the full story 👉 https://zurl.co/BKpp (News Medical) #biotech #genetics
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Journey of Innovation continues......
We are excited to announce our latest research “Prolyl hydroxylase inhibitor desidustat attenuates autoimmune hemolytic anemia in mice” has now been published in “International Immunopharmacology, Volume 142, Part A, 2024, 113029” - https://lnkd.in/d2D3CSFS Autoimmune hemolytic anemia (AIHA) is a heterogeneous group of diseases mediated by autoantibody directed against RBCs causing hemolysis and anemia. Scientists at Zydus Research Centre investigated the effect of desidustat in preclinical model of AIHA. Along with significant improvement in hemoglobin and red blood cells, the bone marrow iron was increased and expression of CD71 (cell surface marker for early erythroid progenitor) and TER-119 (cell surface marker for late erythroid progenitor) in bone marrow were found to be elevated by desidustat, and this treatment also suppressed deposition of membrane-bound antibody which destroys the red blood cells. Desidustat treatment ensures the longer life span of good quality red blood cells, due to decrease in the antibody-mediated lysis of RBCs and also reduced oxidative stress in preclinical model of AIHA.. #AutoimmuneHemolyticanemia #Desidustat #AIHA #ComplementSystem #Zydus #ZydusResearchCentre #YouInspireUs
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Sometimes, proteins your body makes can cause or contribute to disease. These proteins can be either be faulty, or healthy. RNAi therapeutics are a type of medicine, pioneered by Alnylam, that treats disease by targeting and disabling specific mRNA (messenger RNA) which tell your body how to make these unwanted proteins. #ScienceCurious to learn more about the science of #RNAi? Click here: https://bit.ly/3oR8r2l #RNAiTherapeutics #siRNA #GeneSilencing #science
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🔎 Company focus: Vesper Bio Vesper is a clinical stage biotech and world leader in sortilin receptor biology. Its lead program uses a sortilin inhibitor to rebalance levels of progranulin in patients where the sortilin receptor would otherwise reduce circulating and extracellular progranulin, contributing to disease. Find out more about Vesper Bio here: #vesperbio #biotechnology #companyfocus
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🔎 Company focus: Vesper Bio Vesper is a clinical stage biotech and world leader in sortilin receptor biology. Its lead program uses a sortilin inhibitor to rebalance levels of progranulin in patients where the sortilin receptor would otherwise reduce circulating and extracellular progranulin, contributing to disease. Find out more about Vesper Bio here: #vesperbio #biotechnology #companyfocus
About Vesper Bio
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It's great to see Axol's products being used in high quality translational research. Hot off the press is another new paper that our team have been involved in, "Activation of IP3R in atrial cardiomyocytes leads to generation of cytosolic cAMP" (Akerman et al, 2024) Excessive stimulation of the IP3-signalling pathway has been linked to atrial fibrillation (AF) through abnormal calcium handling. However, little is known about the mechanisms involved in this process. As part of the study, axoCells™ human iPSC-derived atrial cardiomyocytes were cultured on a microelectrode array (MEA) and Field Action Potentials (FAP) and contractility were recorded while the α-AR agonist, PE was added to the culture at increasing concentrations. The researchers found that indirect activation of the IP3 pathway in atrial myocytes using an alpha adrenergic receptor (α-AR) agonist phenylephrine (PE), and direct activation using IP3-AM, leads to an increase in cAMP and that these changes can be pharmacologically inhibited using IP3R inhibitors. axoCells atrial cardiomyocytes represent a highly validated, physiologically relevant model and have been extensively characterized structurally, functionally and morphologically. Protein and gene expression, beat rate and action potential parameters, along with functionality of the core cardiac and atrial-specific ion channels have been reported. The full paper can be found here: https://hubs.ly/Q02M1bvY0 To find out how iPSC-derived cardiomyocytes can help your studies, contact us at operations@axolbio.com #iPSCs #cardiomyoctes #DrugDiscovery #StemCells #biopharma #MicroelectrodeArray
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One thing we’ve learned at Alltrna is that despite their complexity, tRNAs make natural drugs. In fact, their incredible nucleotide-level diversity and potential post-transcriptional modifications provide an advantage. The incredible flexibility in #tRNA sequence in terms of function and structure allows us to leverage our computational expertise to explore the combined sequence and modification space to design therapeutic molecules optimized for innumerable characteristics important to drug design. Caroline Köhrer does an excellent job explaining this (and more!) in this insightful Drug Discovery World interview.
Not all RNAs are created equal. tRNAs have some incredible therapeutic potential, but also unique challenges in terms of sequencing and chemical synthesis. In this Q&A with Drug Discovery World, Caroline Köhrer, Alltrna's Senior Vice President of Discovery Platform, breaks down the biology and how Alltrna is harnessing it to create tRNA therapeutics that can unify the treatment of diseases based on shared genetic mutations and accelerate rare disease drug development for millions. Read the article here: https://lnkd.in/egAY6_nC #RNA #tRNAmedicines #tRNAbiology #rarediseases #geneticmedicines #programmablemedicines #AI #machinelearning #StopCodonDisease
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Breakthrough Alzheimer's Drug Targets Key 'Hotspots' to Suppress Toxic Tangles. Brains affected by Alzheimer's disease are characterized by damaging clumps and tangles that interfere with neurons – but a new study describes a drug that could potentially suppress half the problem. The drug is RI-AG03, and it's a peptide inhibitor (a blocker of proteins). In tests on fruit flies and human cells, it has shown a positive effect in reducing neuron... Check out the full story 👉 https://zurl.co/pTQF (ScienceAlert) #biotech #genetics
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Not all RNAs are created equal. tRNAs have some incredible therapeutic potential, but also unique challenges in terms of sequencing and chemical synthesis. In this Q&A with Drug Discovery World, Caroline Köhrer, Alltrna's Senior Vice President of Discovery Platform, breaks down the biology and how Alltrna is harnessing it to create tRNA therapeutics that can unify the treatment of diseases based on shared genetic mutations and accelerate rare disease drug development for millions. Read the article here: https://lnkd.in/egAY6_nC #RNA #tRNAmedicines #tRNAbiology #rarediseases #geneticmedicines #programmablemedicines #AI #machinelearning #StopCodonDisease
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Chairman, Ceo and founder EG427: entering clinical stage in precision genetherapy for neurology; Vice Chairman and founder Atias Pharmaceutical
Great review of the safety of non replicative herpes vectors.
We are proud that Trends in Molecular Medicine, a leading peer-reviewed publication, have published an article based on HSV vectors. It is co-authored by Samuel D. Rabkin, of Harvard Medical School and Massachusetts General Hospital and a leading expert in the field, and our Founder and CSO Alberto Epstein. The article, titled Safety of Non-Replicative and Oncolytic Replication-Selective HSV Vectors, explores how herpes simplex virus type 1 (HSV-1), a DNA virus and human pathogen, has been used to construct promising therapeutic vectors. Such vectors have been approved by multiple regulatory agencies and demonstrate a relatively benign safety profile. Most recent non-replicative vectors elicit only limited immune responses. The review highlights vectors translated to the clinic, and host-virus immune-interactions allowing for safety and efficacy of HSVs. Click below for the full article. #genetherapy #biotech #drugdevelopment
Safety of non-replicative and oncolytic replication-selective HSV vectors - TIMM 2024
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🌐 Special Considerations for Clinical Trials with ATMPs Clinical Operations Spotlight - Edition 02 is out now! 🚀 With the second edition of "Clinical Operations Spotlight", we shine a spotlight on the intricacies of Advanced Therapy Medicinal Products (ATMPs) in clinical trials. 💡 In this edition, you will get an overview of specific considerations for successful ATMP clinical trials; from Product Complexity and Logistics and Supply Chain to Patient Selection and Engagement, and more. personalized manufacturing challenges to navigating the evolving regulatory landscape, we've curated insights to empower clinical operations professionals and researchers alike. ⚙️ Why Focus on ATMPs? As this innovative product type is increasingly developed, it's crucial to understand the nuances of ATMP clinical trials, which hold the promise of revolutionizing medicine as we know it. From gene therapies to regenerative medicine, the potential is limitless, but so are the considerations. This newsletter equips you with the knowledge to navigate this transformative landscape. 🚀If you want to read more, subscribe Now to Stay Ahead: 👇 🔗https://lnkd.in/dCPmhmwt 🤝 Connect with me on LinkedIn: Let's continue the conversation! Connect with me on LinkedIn to stay engaged with the latest updates and discussions in the world of Clinical Operations. 🔗 https://lnkd.in/d579JNAC #ClinicalTrials #CellandGeneTherapies #ATMPs #ClinicalExcellence #InnovationInMedicine #SubscribeNow #ConnectWithMe #TogetherStronger
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