Beond Ibogaine Treatment ’s Post

I’m thrilled to announce the start of Kariya Pharmaceuticals' Phase 1 trial and successful dosing of the first cohort with KP405, a novel, brain-penetrant, dual incretin (GLP-1/GIP) receptor agonist for the treatment of Parkinson’s Disease. I am beyond proud of our team Mikael Thomsen, Christian Hölscher, René Egebro, and Kaare Gyberg Rasmussen, and wish to also thank Mark Dale, John Connell, Grace Hawes, and the rest of the MAC Clinical Research team, as well as our partners (Syngene International Limited, ERBC GROUP, Bachem, Bioneer A/S, Porton Pharma Solutions Ltd., Alderley Analytical Ltd), and our financiers (BioInnovation Institute, BNC KOREA, Inc., and Innovation Fund Denmark), who each played a critical part in getting us here. KP405 is poised to be first to market in this promising new drug class, and we celebrate this milestone as we bring this life-changing drug one step closer to patients in need. https://lnkd.in/dsMxnuh9 #PhaseI #firstinhuman #clinicaldevelopment #endparkinsonsdisease #cureparkinsonsdisease #parkinsonsresearch #parkinsonsdisease #movementdisorders #patientsarewaiting #hope #BioInnovationInstitute #DKlifescience #DKBiotek #neuroscience #CNS #onestepcloser

Kyle Landskroner, Chief Scientific Officer at AZAFAROS B.V., last week gave an oral presentation entitled: 𝘕𝘪𝘻𝘶𝘣𝘢𝘨𝘭𝘶𝘴𝘵𝘢𝘵, 𝘢 𝘯𝘰𝘷𝘦𝘭 𝘓-𝘪𝘥𝘰 𝘢𝘻𝘢𝘴𝘶𝘨𝘢𝘳 𝘪𝘯 𝘥𝘦𝘷𝘦𝘭𝘰𝘱𝘮𝘦𝘯𝘵 𝘧𝘰𝘳 𝘎𝘔1 𝘢𝘯𝘥 𝘎𝘔2 𝘨𝘢𝘯𝘨𝘭𝘪𝘰𝘴𝘪𝘥𝘰𝘴𝘦𝘴, 𝘢𝘯𝘥 𝘕𝘪𝘦𝘮𝘢𝘯𝘯-𝘗𝘪𝘤𝘬 𝘥𝘪𝘴𝘦𝘢𝘴𝘦 𝘵𝘺𝘱𝘦 𝘊 at the EFMC-ACSMEDI Medicinal Chemistry Frontiers 2024 conference. Dr. Landskroner discussed our novel L-ido biphenyl-containing azasugar, which recently completed a 12-week Phase 2 study, 🌈 RAINBOW (Phase 2 NCT05758922). He also presented data on the effectiveness of #nizubaglustat in preclinical mouse models on Sandhoff and NPC diseases, its ability to reduce GM1 in a human organoid model of GM1 gangliosidosis, and nizubaglustat’s effects in our completed Phase 1 study. Nizubaglustat is a potent and selective inhibitor of both glucosylceramide synthase (GCS) and non-lysosomal glucosylceramidase (NLGcase), two enzymes involved in glycosphingolipid metabolism.  Its unique mode of action and brain-penetrant properties make nizubaglustat a promising candidate to treat a variety of glycosphingolipid (GSL) neurologic disorders, including #GM1 and #GM2 gangliosidoses, and Niemann-Pick disease type C (#NPC).  #medicinalchemistry #smallmolecules, #Biotech #DrugDiscovery #MedChem #EFMC #ACSMEDI, #rarediseases, #Azafaros 

☑️ *READ DESCRIPTION BELOW:* Part 1: llustration of the miRNA-based drug discovery and development process beginning from target identification and miRNA discovery to FDA-approved miRNA therapeutics on the market. In the traditional drug development process, the timeline from target identification and drug discovery to phase 1–3 human clinical trials and, ultimately, FDA approval, followed by phase 4 studies, can go on for several years. Conversely, RNA-based and, more specifically, miRNA-based drug discovery and development can potentially mitigate attrition rates, time constraints, and costs. The initial phase in developing miRNA therapeutics involves systematically selecting potential miRNA candidates by analyzing patient samples and validating their relevance to a particular disease of interest through tissue culture and in vivo models. Various publicly available genomic and proteomic databases from diverse healthy and diseased tissues can aid in identifying promising miRNA candidates when combined with biological validation(...) https://lnkd.in/e38EDpjA #Gesundheit #Bildung #Fuehrung #Coaching #Mindset #Motivation #Gehirn #Neuroscience #Psychologie #Persoenlichkeitsentwicklung #Kindheit #KeyNoteSpeaker #Humangenetik #Biochemie #Neuroleadership #Ernaehrung #Transformation #Stress #Demografie #Gender #Age #interkulturelleKompetenz #Epigenetik #Veraenderung #EmotionaleIntelligenz #Change #Gesellschaft #Organisationsentwicklung #Philosophie #Beratung # Quantum

A nice overview of Fragment Based Drug Discovery (FBDD) with a focus on diseases of the Central Nervous System (CNS). This review highlights the strengths of FBDD in terms of generating smaller, more ligand efficient molecules with generally better pharmacokinetic profiles. It also highlights the importance of choosing the right technique to measure binding of these smaller (and weaker) fragments - for example crystallography, NMR, SPR and DSF. The review also highlights examples of recent CNS targeting compounds developed using FBDD. University of Sydney, CSIRO Manufacturing & Infrastructure Technology, Vast Bioscience #drugdiscovery #smallmolecule #pharmacokinetics #FBDD #structuralbiology #biophysics #crystallography #surfaceplasmonresonance #CNS #Alzheimers #Parkinsons

It's Day 3 of Axol Advent- and today, we're bringing you gifts of three. Tri-culture models (incorporating three different iPSC-derived cell types) have exciting potential in drug discovery as they enable human-relevant models with greater complexity than simple mono-culture. By mixing three different cell types, you can incorporate cell-cell crosstalk and better recapitulate human physiology. We recently validated an exciting tri-culture axoModel™ for neurotoxicity screening, comprising axoCells™ cortical excitatory neurons, inhibitory interneurons and astrocytes. The axoModel was validated via a blinded compound study in partnership with Sumitomo Pharma America Inc. Our Senior Scientific Advisor (and first author) Dr Steven Broadbent presented this work at Neuroscience 2023, and you download the poster by clicking this link: https://hubs.la/Q02bDYq30 It's not quite gold, frankincense and myrrh, but it does demonstrate the exciting potential of advanced in vitro models for drug discovery and safety pharmacology. #iPSCs #TriCulture #Neurotoxicity #PluriSnowtent

It's Day 3 of Axol Advent- and today, we're bringing you gifts of three. Tri-culture models (incorporating three different iPSC-derived cell types) have exciting potential in drug discovery as they enable human-relevant models with greater complexity than simple mono-culture. By mixing three different cell types, you can incorporate cell-cell crosstalk and better recapitulate human physiology. We recently validated an exciting tri-culture axoModel™ for neurotoxicity screening, comprising axoCells™ cortical excitatory neurons, inhibitory interneurons and astrocytes. The axoModel was validated via a blinded compound study in partnership with Sumitomo Pharma America Inc. Our Senior Scientific Advisor (and first author) Dr Steven Broadbent presented this work at Neuroscience 2023, and you download the poster by clicking this link: https://hubs.la/Q02bDWR70 It's not quite gold, frankincense and myrrh, but it does demonstrate the exciting potential of advanced in vitro models for drug discovery and safety pharmacology. #iPSCs #TriCulture #Neurotoxicity #PluriSnowtent

#PROTAC Series: E3 ligase selection and specificity (now towards #drugdesign) The importance of E3 ligase selection and specificity, and the inclusion of specific details about Cereblon (CRBN) and its ligands add depth to our earlier discussion. In my last LinkedIn post, we discussed the critical role of E3 ligase selection and specificity, underscoring the importance of choosing the right E3 ligase from the vast E3 family. This decision is pivotal and hinges on the availability of specific ligands tailored for recognized E3 ligase. Today, let's delve into a specific E3 ligase, Cereblon (CRBN), and explore the corresponding ligands. This will shed light on the molecules capable (minimum pharmacophore) of interacting with and modulating the activity of this ligase. 1. Cereblon (CRBN):   - Ligands: Thalidomide, pomalidomide, and lenalidomide.   - Thalidomide, lenalidomide, and pomalidomide share a common structure with a conserved glutarimide ring and a variable phthaloyl ring (shown in the picture attached). Thalidomide and its analogues are called immunomodulatory drugs (IMiDs). If you would like to see more analogues (designs) that can interact with CRBN, and help you design new #PROTACs. Let me know. A groundbreaking study in 2010 (Science, 2010, 327, 5971, 1345–1350) revealed that the primary target of thalidomide is CRBN. CRBN acts as a substrate receptor of cullin-RING ligase 4 (CRL4), an E3 ubiquitin ligase complex composed of Cul4, DDB1, and RBX1 (shown in the picture attached). This complex plays a pivotal role in regulating the cell cycle, DNA damage repair, and chromatin replication by targeting cellular substrates for ubiquitination. This additional information enriches our understanding of the "pharmacophore" of E3 ligase CRBN ligands. Understand and remember these structures, this will help us in our future discussions, stay tuned. If you have not followed us yet, please follow MetaG1 Research Consortium and CEO Purushottam Dewang. Hit the like 👍 button (double tap the picture), shares and comments are encouragements to keep talking to you 😊 ! #protac #protacs #medicinalchemistry #drugdesign #innovation #MetaG1  Figure ref: Biomedicine & Pharmacotherapy 127 (2020) 110114

𝗦𝘆𝗻𝗖𝗥𝗢𝗣𝗮𝘁𝗰𝗵 𝟯𝟴𝟰: 𝗗𝗿𝗶𝘃𝗶𝗻𝗴 𝗜𝗻𝗻𝗼𝘃𝗮𝘁𝗶𝗼𝗻 𝗶𝗻 𝗜𝗼𝗻 𝗖𝗵𝗮𝗻𝗻𝗲𝗹 𝗥𝗲𝘀𝗲𝗮𝗿𝗰𝗵 ✅🔬👩🔬 SB Drug Discovery specializes in ion channel and receptor drug discovery services, offering customized solutions from cell line and assay development to high-throughput screening and lead optimization. To keep up with the growing demands and rapid progress of their field, SB Drug Discovery employs Nanion's sophisticated electrophysiology platforms. The SyncroPatch 384 exemplifies this, allowing for recordings from up to 384 cells simultaneously, which minimizes compound consumption and manual labor. In partnership with Nanion, SB Drug Discovery stands at the cutting edge of innovative scientific research. They are investigating the lysosomal cation channel TMEM175, recognized as a promising therapeutic target for Parkinson's disease. Their joint research emphasizes the effectiveness and versatility of Nanion's tools, notably the SyncroPatch 384 and SURFE²R 96SE, highlighting the significance of a comprehensive approach to the analysis of complex biological systems. For an in-depth look at SB Drug Discovery’s work, download the case study here: https://ow.ly/MtKM50RjSPO We are proud of our lasting partnership and look forward to more groundbreaking discoveries in the future! 👏 David Dalrymple #IonChannels #DrugDiscoveryServices #Electrophysiology #Innovation #CollaborativeScience #CRO #SyncroPatch384 #SURFE2R #APC

Some biopharma M&A for the holidays: Bristol Myers Squibb plans to buy neuroscience biotech Karuna Therapeutics in cash at a $14 billion deal value just a few weeks after AbbVie dished out $8.7 billion to get its hands on schizophrenia drugmaker Cerevel Therapeutics. #acquistions #deals #pharma #biotech #neuroscienceresearch #neuroscience #cns #schizophrenia #mergersandacquisitions Endpoints News

🔬 Exciting news! Our latest scientific paper, co-authored with Jana Kejíková and other esteemed colleagues, has been published in the Molecular Pharmaceutics journal! Titled "Nebulization and In Vitro Upper Airway Deposition of Liposomal Carrier Systems," this publication delves into the fascinating realm of liposomal carrier systems and their potential for pulmonary drug delivery, and is now available online! 📰 https://lnkd.in/eaex-hKv In this study, we explore the stability of liposomal systems against nebulization and investigate their potential in delivering drugs to the lungs. Our research underscores the importance of considering realistic breathing profiles. By simulating these conditions, we aim to provide a more accurate understanding of aerosol deposition in the lungs, paving the way for personalized drug delivery solutions. 🙏 A heartfelt acknowledgment to all our co-authors for their invaluable contributions to this work! #PharmaceuticalResearch #DrugDelivery #LiposomalSystems #ScientificPublication #MolecularPharmaceutics