Parc Taulí Research and Innovation Institute (I3PT)’s Post

There are so many rodent models available for preclinical target and drug discovery for #MASH. But which model offers the highest resemblance of the human disease?   The answer is simple. In the recent issue of Nature Metabolism, the renowned #LITMUS consortium concludes that Gubra’s GAN DIO-MASH mouse model ranks #1 for human proximity, a key indicator of clinical translatability, in terms of both metabolic relevance and ability to induce MASH-fibrosis. LITMUS compared a wide range of the most commonly used preclinical MASH models, evaluating their metabolic phenotype, liver histopathology and transcriptomics against corresponding MASH patient data.    We are very proud and grateful to be recognized by LITMUS as the provider of the most translational preclinical model.   Check out the paper and explore the objective ranking of preclinical models of MASH in the industry. #PreclinicalModel #PreclincalResearch #ClinicalTranslatability #MASLD #Publication #PreclinicalCRO #ResearchModels

#ASLTreatment - Motor Neuron Disease - recent scientific research showed that Allogeneic B cell immunomodulatory therapy benefitted lab models, i.e. laboratory animals. As a result, the researches got permission from the FDA and Massachusetts General Hospital to try this therapeutic approach in an individual w/ #ALS. There are a range of alternative approaches to developing ALS therapies. We particularly like MSCs including $BCLI's NurOwn, its P3b trial has just begun rollout - of course $BCLI needs further funding to complete, but it is an interesting area with some promise of marked progress in therapy effectiveness for early to mid-stage ALS sufferers, if BCLI's P3b is a success. To see where $BCLI is right now you can read our #ACFResearch #NurOwn Update https://lnkd.in/dFgfxHpk $AZN $AMGN $SNY $GILD $VRTX $ABBV $AMRX $ROG.SW $MRK $PFE $TEVA $NVS $BIIB. Brainstorm Cell Therapeutics Pfizer AbbVie ALS TDI The ALS Association ALS CURE Project

#StemCells Oral #ALStherapy by #TransposonTherapeutics (TPN-101 PIIa trial) shows safety & slows lung function decline over w/ 1yr’s treatment in ALS C9orf72 mutations. Also see NurOwn PIIIb - Read our #ACFResearch update on $BCLI https://lnkd.in/e2sThM9S The ALS Association ALS TDI

💡 Unlocking Potential: Risdiplam (discovered and developed by PTC Therapeutics, Inc. and Roche) marks a milestone in medical history as one of the first small molecule therapies designed to target RNA. More specifically, it was developed as a treatment for Spinal muscular Atrophy (SMA) and works by targeting the RNA splicing mechanism affected in this disease. Using Risdiplam as a case-study, learn more about the science behind this game-changing innovation and its implications for future treatments. This is a continuation of a special 3-part series that we started at the end of 2023, to introduce the emerging field of using small molecule drug compounds to target RNA, as well as EDDC's in-house efforts in this field. As was presented in the previous installment, the illustrations in this were meticulously drawn by our talented chemist Juliana Mohammad. #EDDCsg #DrugDiscovery #DrugDevelopment #Risdiplam #MedicalInnovation #RNATherapy #SpinalMuscularAtrophy

With all the publicity around cell and gene therapies for genetic disorders, a less known strategen is to use small molecules to target gene products. With this approach, the disease process is alleviated and relief provided to the patient. This has the benefit that the cost of goods will be typically much lower than traditional cell and gene therapies, and also unlike CGT for which the effects can be permanent, there is an "off switch" in that cessation of treatment should reverse its effects. EDDC is pursuing efforts here, and this is an exciting space to watch!

Don’t miss the opportunity to connect with our CEO Anker Lundemose at #BioEquity Europe 2024, from 12 - 14 May in San Sebastian, Spain! It’s an ideal time to meet as the clinical trial for our lead asset #MTX325 is now underway. MTX325, is a potentially disease-modifying treatment for #Parkinsons Disease, a progressive neurological condition that affects over 10 million people worldwide. 📧 Hear the latest developments on Mission Therapeutics’ innovative #mitophagy targeting pipeline in a 1-1 meeting using the link in the comments below. @Mission Therapeutics is committed to advancing its pipeline of deubiquitylating enzyme (#DUBs) inhibitor drug candidates for conditions driven by a build-up of dysfunctional mitochondria in cells, including #ParkinsonsDisease, #HeartFailure, #KidneyDisease, #DuchennesMuscularDystrophy (#DMD), #idiopathicpulmonaryfibrosis (#IPF) and #Alzheimers. #BioEquityEurope #BioEquity2024

Data we presented this spring at #ASGCT24 showed the promise of our lead candidate, EPI-321, to uniquely address the epigenetic root cause of #FSHD muscular dystrophy. In this interview with CGTLive, our Head of Therapeutics Alexandra Collin de l'Hortet describes the mode of action of EPI-321 and the robust preclinical dataset that supports its potential to have a transformative impact for people with FSHD: https://lnkd.in/e-uufmYv

Several factors contribute to the development and progression of #Alzheimer's disease. It is increasingly evident that a multi-targeted approach is needed to slow and reverse the destruction caused by this complex disease. While amyloid-beta buildup and tau tangles have been the focus of research and drug development, attention has been turning to other pathological factors affecting disease progression, including neuroprotection and synaptic dysfunction. InMed's INM-901 is a novel drug candidate that may target several biological pathways associated with #Alzheimers disease. Preclinical studies of INM-901 demonstrate neuroprotective effects, reduced neuroinflammation and an ability to promote the growth of neurites. More about INM-901: https://ow.ly/ozUo50SNJo7 $INM

Comparing activation methods to yield clinical-scale expansion of γδ #Tcells Some #Tcelltherapies have been approved to treat patients with conditions like #Bcellmalignancies and #multiplemyeloma, but their use for #solidtumors remains challenging. Gamma delta (γδ) T cells have the inherent ability to infiltrate solid tumors and kill transformed cells, but their low prevalence in peripheral blood poses a challenge when generating enough cells to produce a clinical dose. Next month, Lonza Bioscience Solutions’ Chengkang Zhang (Associate Director, R&D) will present two novel methods for the expansion of #gammadeltaTcells from peripheral blood #mononuclearcells (PBMCs) and present data showcasing their suitability for clinical applications Register for this upcoming #webinar below:

#nanoemulsions #CNS #RNA | 𝗔𝗿𝗲 𝗜𝗼𝗻𝗶𝘇𝗮𝗯𝗹𝗲 𝗡𝗮𝗻𝗼𝗲𝗺𝘂𝗹𝘀𝗶𝗼𝗻𝘀 𝘁𝗵𝗲 𝗙𝘂𝘁𝘂𝗿𝗲 𝗶𝗻 𝗖𝗡𝗦 𝗱𝗿𝘂𝗴 𝗱𝗲𝗹𝗶𝘃𝗲𝗿𝘆? Lipid nanoparticles (LNPs) currently dominate the RNA delivery landscape; however, their limited diffusivity hampers targeted tissue dissemination, and, hence, their capacity for intracellular drug delivery. This is especially relevant for tissues such as the central nervous system (CNS), where overcoming proactive brain barriers is crucial for the efficacy of genetic therapeutics. This research aimed to create ionizable #nanoemulsions (#iNEs), a new generation of #RNA delivery systems with enhanced diffusivity. The developed iNEs, consisting of a combination of C12–200, DOPE, Vitamin E, and DMG-PEG, 𝘄𝗶𝘁𝗵 𝗮 𝘀𝗶𝘇𝗲 𝗯𝗲𝗹𝗼𝘄 𝟭𝟬𝟬 𝗻𝗺, 𝗻𝗲𝘂𝘁𝗿𝗮𝗹 𝘀𝘂𝗿𝗳𝗮𝗰𝗲 𝗰𝗵𝗮𝗿𝗴𝗲, 𝗮𝗻𝗱 𝗵𝗶𝗴𝗵 𝗥𝗡𝗔 𝗹𝗼𝗮𝗱𝗶𝗻𝗴 𝗰𝗮𝗽𝗮𝗰𝗶𝘁𝘆, 𝘀𝗵𝗼𝘄𝗲𝗱 𝗲𝘅𝗰𝗲𝗹𝗹𝗲𝗻𝘁 𝗰𝗲𝗹𝗹 𝘃𝗶𝗮𝗯𝗶𝗹𝗶𝘁𝘆 𝗮𝗻𝗱 𝘁𝗿𝗮𝗻𝘀𝗳𝗲𝗰𝘁𝗶𝗼𝗻 𝗲𝗳𝗳𝗶𝗰𝗶𝗲𝗻𝗰𝘆 𝗶𝗻 𝘃𝗮𝗿𝗶𝗼𝘂𝘀 𝗰𝗲𝗹𝗹𝘂𝗹𝗮𝗿 𝗺𝗼𝗱𝗲𝗹𝘀, 𝗶𝗻𝗰𝗹𝘂𝗱𝗶𝗻𝗴 𝗻𝗲𝘂𝗿𝗼𝗻𝘀, 𝗮𝘀𝘁𝗿𝗼𝗰𝘆𝘁𝗲𝘀, 𝗮𝗻𝗱 𝗺𝗶𝗰𝗿𝗼𝗴𝗹𝗶𝗮. 𝗦𝘂𝗯𝘀𝗲𝗾𝘂𝗲𝗻𝘁𝗹𝘆, #𝗶𝗡𝗘𝘀 𝗰𝗼𝗻𝘁𝗮𝗶𝗻𝗶𝗻𝗴 #𝗺𝗥𝗡𝗔 #𝗚𝗙𝗣 𝘄𝗲𝗿𝗲 𝘁𝗲𝘀𝘁𝗲𝗱 𝗳𝗼𝗿 #𝗖𝗡𝗦 𝘁𝗿𝗮𝗻𝘀𝗳𝗲𝗰𝘁𝗶𝗼𝗻, 𝗵𝗶𝗴𝗵𝗹𝗶𝗴𝗵𝘁𝗶𝗻𝗴 𝘁𝗵𝗲𝗶𝗿 𝗲𝘅𝗰𝗲𝗽𝘁𝗶𝗼𝗻𝗮𝗹 𝗱𝗶𝗳𝗳𝘂𝘀𝗶𝘃𝗶𝘁𝘆 𝗮𝗻𝗱 𝘀𝗲𝗹𝗲𝗰𝘁𝗶𝘃𝗲 𝘁𝗿𝗮𝗻𝘀𝗳𝗲𝗰𝘁𝗶𝗼𝗻 𝗼𝗳 𝗻𝗲𝘂𝗿𝗼𝗻𝘀 𝗳𝗼𝗹𝗹𝗼𝘄𝗶𝗻𝗴 𝗶𝗻𝘁𝗿𝗮-𝗽𝗮𝗿𝗲𝗻𝗰𝗵𝘆𝗺𝗮𝗹 𝗮𝗱𝗺𝗶𝗻𝗶𝘀𝘁𝗿𝗮𝘁𝗶𝗼𝗻. Kudos to authors Mireya L. Borrajo, Aloia Quijano Ocampo, Philipp Lapuhs, @Ana Rodriguez-Perez, Shubaash Anthiya, @José Labandeira-Garcia, @Rita Valenzuela, and Maria Jose Alonso. Check here for more: https://lnkd.in/gNXuWQ6h