We are delighted to kick off an exciting new collaboration with Christine Bear, PhD from Molecular Medicine at The Hospital for Sick Children (SickKids), to unlock targeted therapies for people with Cystic Fibrosis (CF). The recent breakthrough drug Trikafta™ has been transformative for patients with the F508del mutation on the CFTR gene: the most common cause of CF. But there are over 400 disease-causing gene variants of the CFTR gene, and not all patients with the F508del mutation respond to Trikafta™. Similarly, the SickKids CF Centre found that tissue samples carrying CF-causing mutations show considerable variability in Trikafta™ response size in the lab. This collaboration will use machine learning to sift through phenotypic and genetic data for responders and non-responders, to help identify those people who are most likely to benefit from Trikafta™ and emerging therapies. “The results of this project could be so impactful to the lives of those living with Cystic Fibrosis. I’m gratified to be involved in this important work.” —Victoria Catterson, VP of Data Science Research at BioSymetrics #CysticFibrosis #MachineLearning
BioSymetrics’ Post
More Relevant Posts
-
Phacilitate's Advanced Therapies Europe is next week and we can't wait. But what makes it Europe-Focused? A majority of the sessions talk directly to the challenges of ATMPs and their development within Europe - including how we support the capital ecosystem, the manufacturing and logistics infrastructure, and the patient need throughout European states. I realise I've shouted about these before, but we also have several sessions highlight specific European regions that have become hubs of scientific and technical innovation in cell and gene therapies. These are: 💶 Spotlight on Cell Therapy in Iberia - Future of ATMPs in Spain & Portugal Chaired by Antonio Salgado (ICVS - Life and Health Sciences Research Institute) Joined by presenters: Stefanos Theoharis (OneChain Immunotherapeutics) Rui A. Sousa (Stemmatters) Claudio Santos (Gyala Therapeutics) And panel participation from Miguel Forte (Kiji Therapeutics & ISCT, International Society for Cell & Gene Therapy) & Ricardo Baptista (SmartCella Holding AB) 💶 Spotlight on Cell and Gene Therapy Developments in Northern Europe Chaired by Heather Main (ATMP Sweden) Joined by presenters: Tim Desmet (Ghent University/CEVAC - Ghent University and Ghent University Hospital) Philippine Doekes (European Hub for Advanced Development in Regenerative Medicine and Biomaterials (EHAB) & panel participation from Ricardo Baptista (SmartCella Holding AB) 💷 ATMPs in the UK: Spotlight on the UK's Innovations and Initiatives Chaired by Rosie Lindup (BioIndustry Association (BIA) Presentations from: Faraz Farooqi, (NICE - National Institute for Health and Care Excellence) Panos Kefalas (Cell and Gene Therapy Catapult) Samantha Barber (Gene People) and joined by panelist Zoe Hewitt (British Society for Gene and Cell Therapy (BSGCT) This is also supported by the BioIndustry Association (BIA) pavilion on the exhibition floor, showcasing UK biotech excellence in ATMPs. We'll be celebrating success and highlighting potential solutions to major European challenges, and these segments are particularly exciting and something I'm keen to develop further at future Phacilitate events! #ATE24 #Advancedtherapieseurope #cellandgenetherapy #genetherapy #celltherapy #biotech #bia #europeanbiotech #atmpsweden #ISCT #procella #smartcella #genepeople #NICE
To view or add a comment, sign in
-
🔬📊 Exploring the Depths of Single-Cell Technologies: Introducing SCORPION 🧪 Are you ready to dive deep into the fascinating world of single-cell technologies? Today, we're thrilled to introduce you to SCORPION - a groundbreaking tool that's revolutionizing our understanding of molecular mechanisms at the single-cell level. It is a game-changer in the field of gene regulatory network reconstruction from single-cell/nuclei RNA-sequencing data. Leveraging a sophisticated message-passing algorithm, SCORPION reconstructs comparable gene regulatory networks, paving the way for population-level comparisons while maintaining baseline priors. Through supervised experiments, SCORPION demonstrated its ability to pinpoint differences in regulatory networks between wild-type and transcription factor-perturbed cells with remarkable precision. With its impressive scalability, SCORPION seamlessly transitions to population-level analyses. By detecting differences between tumor regions consistently across multiple cohorts, SCORPION sheds light on disease progression and highlights phenotypic regulators that could impact patient survival. Case in point: a single-cell RNA-sequencing atlas featuring a staggering 200,436 cells from colorectal cancer and adjacent healthy tissues. Stay tuned for more updates and insights from the world of single-cell technologies. Keep exploring, keep innovating, and keep pushing the boundaries of scientific knowledge! 🌟🔬 Link to the publication: https://lnkd.in/g_U8zybG #SCORPION #SingleCellTechnologies #GeneRegulatoryNetworks #ScientificRevolution
To view or add a comment, sign in
-
🔬📚 Exciting News Alert! 📚🔬 🎉 I'm thrilled to announce the publication of our latest paper "Towards the Development of a DNA Automaton: Modular RNA-cleaving Deoxyribozyme Logic Gates Regulated by miRNAs"! 🎉 💡 Inspired by natural riboswitches we created innovative, modular designs of DNAzyme-based logic gates for the precise and efficient identification of specific miRNAs within a panel, holding immense potential for advancements in diagnostics and therapeutics of various of diseases. 🔍 MicroRNAs play a pivotal role in regulating gene expression and have been implicated in disease pathogenesis. Therefore, the ability to accurately detect and quantify these molecules is paramount in disease diagnosis, prognosis, as well as in developing targeted interventions. 🔗 Click the link below to get familiar with our work. Your support and engagement are invaluable as we continue to push the boundaries of scientific innovation. 🌟 https://lnkd.in/d3TbwiKs #DNAnanotechnology #miRNA #biotechnology #research #innovation #molecularbiology #scientificadvancement
To view or add a comment, sign in
-
Congratulations to Victor Ambros and Gary Ruvkun for the The Nobel Prize in Medicine for the discovery of microRNA! Previously thought to be “evolutionary debris”, the discovery of microRNA uncovered a whole new mechanism of gene regulation. These molecules bind to mRNA (messenger RNA), inhibiting protein expression by recycling/destroying the mRNA transcript or preserve for future translation. This discovery led to breakthroughs in- 1. Understanding of cell differentiation. 2. Involvement of damaged miRNAs in cancer development and progression. 3. Development of gene editing technologies involving gene silencing. Sources and Further Reading: 1. https://lnkd.in/dtJRSrVZ 2. https://lnkd.in/der6XTd6 3. https://lnkd.in/dQZPSDMJ This day is dedicated to celebrating these brilliant scientists and the teams that were behind their experiments, lab work and publication. The discovery of miRNA has transformed a large number of lives, diagnoses (and curriculums!). #NobelPrize, #microRNA, #pharma, #biotech, #medicalresearch, #research, #publications, #nobelprizeinmedicine, #cancerbiology, #cancer, #genetics, #molecularbiology
To view or add a comment, sign in
-
🚀 𝗨𝗽𝗰𝗼𝗺𝗶𝗻𝗴 𝗪𝗲𝗯𝗶𝗻𝗮𝗿: Unlocking CRISPR: Advances in Base Editing, Prime Editing and Future Applications Join our scientist to explore the future of gene editing with cutting-edge #CRISPR technologies. This webinar will also provide you insights into recent breakthroughs, enhanced delivery methods, and efforts to minimize off-target effects. Discover how these advancements are paving the way for transformative applications, from correcting hereditary mutations to developing more effective cancer therapies 📅Register here: https://bit.ly/4fd7Mk6 What you will learn: ✔ The mechanisms behind BE and PE technologies ✔ The latest research advancements and breakthroughs in CRISPR-based gene editing ✔ Potential therapeutic applications and the future impact of these technologies in medicine #GeneEditing #Biotechnology #Genomics #CancerResearch #LifeSciences #ScientificBreakthroughs #MolecularBiology #VirtualEvent #MedicalInnovation #CRISPRApplications #GenScript #Webinar
To view or add a comment, sign in
-
The Summer issue of Drug Discovery World (DDW) is out now! The DDW Summer issue is out now and looks at where the global biotech hubs are for drug discovery and development, asks how 3D genomics can accelerate personalised medicine, and looks at the technological advances in mass spectrometry. It also includes an exclusive global cancer research guide examining the innovation happening in this sector. Other features in the Summer issue include: - How AI can advance the hit-to-lead process - GSK’s efforts in the autoimmune sector - Improving viral vector safety for cell & gene therapy Read the digital version here: https://lnkd.in/eEiBFnsz #DrugDiscovery #DrugDevelopment #LifeSciences
To view or add a comment, sign in
-
🧬 NTx Spotlight at ASGCT 2024: Advancing Genetic Medicines 🧬 Jessica Silva, a dedicated scientist at NanoVation Therapeutics, is gearing up to present our latest research at the American Society of Gene & Cell Therapy Annual Meeting. She will be diving into our sophisticated use of Long-Circulating Lipid Nanoparticles (LCLNP) for targeted delivery of nucleic acids — a key innovation in our genetic medicine toolbox. 📅 Date & Time: May 9, 2024, at 12:00 PM ET 🔍 Abstract #0032 🔬 Title: "Long-Circulating Lipid Nanoparticles (LCLNP) Effectively Deliver Nucleic Acids to Hematopoietic Stem and Progenitor Cells in the Bone Marrow" This presentation will explore the forefront of LCLNP technology and its potential to transform genetic medicine. Jess’s insights are paving the way for innovative treatments targeting bone marrow cells, crucial in combating various diseases. Don't miss the chance to see how NTx is shaping the landscape of advanced therapeutics. #ASGCT2024 #InnovativeResearch #Biotech #genetherapy #RNA #Nucleicacid
To view or add a comment, sign in
-
CRISPR-based diagnostics and platforms, a groundbreaking innovation that emerged in 1987, have revolutionised the field of genetic engineering and molecular biology. CRISPR technology, initially a mysterious part of bacterial immunity, enables precise and efficient gene editing and has given rise to a new era of diagnostics. With the ability to target specific DNA sequences, it has paved the way for rapid, cost-effective, and highly accurate genetic testing. This has transformed the landscape of medicine, as it allows for the early detection of genetic disorders, infectious diseases, and even cancer markers. Who developed this? While the foundational discovery of clustered regularly interspaced short palindromic repeats (CRISPR) was made by Yoshizumi Ishino and his team in Japan that year, it wasn't until decades later that scientists realised its full potential. How has CRISPR revolutionised biotech? The versatility of CRISPR-based diagnostics is remarkable. It can identify viral RNA, single nucleotide polymorphisms (SNPs), and much more. It's a testament to human ingenuity, promising a future where early disease detection and personalised medicine are the norm. Which are the companies leveraging this innovation? Some top players in the CRISPR space are CRISPR Therapeutics, Beam Therapeutics, Intellia Therapeutics, Inc., Editas Medicine, and Verve Therapeutics. The journey from Ishino's initial discovery in 1987 to today's CRISPR-based diagnostic platforms is a testament to the power of scientific exploration and its impact on our world. #BioAngelsBiotechChronicles #BioAngels #HealthcareInnovations #HealthcareSolutions
To view or add a comment, sign in
-
Don't miss our poster presentations at Discovery on Target in Boston, MA! 📃 "Identification and characterization of small molecules inhibiting RNA targets: miRNA" 🏷 Session A, poster A39 📆 October 1st to 2nd a.m. 👩🏼🔬 Paola C., PhD, Director Alliance Management Abstract: RNA plays a key role in transcription and translation: gene regulation, proliferation, apoptosis, and development. The biological function is linked to the unique secondary and tertiary structures. Many diseases are associated with dysregulation of RNA functions, so there is a growing interest in RNAs as drug targets for novel therapies or targeting undruggable proteins. The most used modalities are antisense oligonucleotides (ASOs), CRISPR gene editing, and more recently small molecules. [...] Discover more about our approach that demonstrates a miRNA targeting platform that could be applied to other oncogenic miRNA targets: https://lnkd.in/edM4ecJj For more questions and details, come and meet our team at our booth 508! #DrugDiscovery #Biophysics #RNA #SPR #microRNA #DiscoveryOnTarget #BostonDOT24
To view or add a comment, sign in
-
🚀 Accelerating Genomics Research with Cutting-Edge Technology from Element! 🚀 Dr. William Lai, Assistant Research Professor and Director of The Epigenomics Core Facility at Cornell University, is making waves! At the Element Biosciences "Shift Your Science" Roadshow in NYC, Dr. Lai showcased his research in gene regulation, leveraging the powerful AVITI™ system. 🔬 By switching from Illumina to AVITI sequencing, Dr. Lai's lab has significantly advanced its research capabilities: • 24-hour turnaround and better readouts • Cost reduction from $130,000 to $15,000 for the same number of reads • 1.2 billion reads in one go, enabling rapid, simultaneous testing of multiple factors Dr. Lai's work focuses on understanding the complex interactions of genes, RNA, and proteins, critical for developing effective cancer therapies. His recent studies on anti-cancer drugs flavopiridol and triptolide reveal how different mechanisms of RNA expression inhibition impact therapeutic outcomes. ➡️ To learn more read the blog: https://bit.ly/3wU996W 🚀🔍 This post is part of our Elemental Insights series, focused on celebrating our customer success stories and the groundbreaking work they are accomplishing with Element's technology. 🌟 Want to be featured? Tell us your story: https://bit.ly/3RfzUJP #ElemBio #AVITI #Genomics #Epigenomics #ResearchInnovation #CornellUniversity #CancerResearch #DrugDiscovery
To view or add a comment, sign in
3,079 followers