Advancing Precision Medicine with ASO & siRNA Technologies ?? Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are at the forefront of gene silencing technologies, offering innovative solutions for targeting diseases linked to aberrant gene expression. Here's why these tools are game-changers: ?? High Specificity: Precisely target mRNA to block disease-related protein production. ?? Broad Therapeutic Potential: Effective for conditions like cancer, rare genetic disorders, and viral infections. ?? Non-Permanent & Reversible: Unlike gene editing, their effects can be fine-tuned with dosing adjustments. ?? Cost-Effective & Scalable: Synthesized rapidly, enabling faster development and large-scale applications. ?? Breakthrough in Delivery: Recent advancements allow crossing the blood-brain barrier, expanding use in CNS diseases. ?? Market Insights: The global ASO and siRNA therapeutics market is projected to grow from $4.38B in 2023 to $14.35B by 2030 (CAGR of 18.72%), driven by advancements in molecular biology and the demand for personalized medicine. As molecular therapies evolve, ASOs and siRNAs provide hope for undruggable targets and diseases with limited treatment options. Let’s connect to discuss how these innovations can transform your research! ?? hashtag #GenomicMedicine hashtag #ASOTechnology hashtag #PrecisionTherapeutics If you like our content creation, get in touch with us to see how we might be able to assist you.
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?? Unveiling the Future of Medicine: Cell and Gene Therapy Tools and Reagents Market! ?? The global market for Cell and Gene Therapy (CGT) tools and reagents is set to revolutionize healthcare, growing from $10.0 billion in 2024 to an impressive $16.7 billion by 2029, with a robust CAGR of 10.8%. This growth highlights the pivotal role of CGT tools in advancing cutting-edge treatments. Cell and gene therapy tools and reagents are essential in developing transformative therapies that target genetic disorders, cancers, and rare diseases. These technologies enable precise gene editing, cell modification, and therapeutic development, making previously untreatable conditions manageable or curable. Key drivers include increasing investments in biotechnology, advancements in gene-editing platforms like CRISPR, and the rising prevalence of chronic diseases. As research progresses, CGT tools will continue to pave the way for personalized medicine, unlocking unparalleled opportunities in healthcare. https://bit.ly/4hRnXWm ?? #CellAndGeneTherapy #Biotechnology #HealthcareInnovation #MarketTrends #FutureOfMedicine
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???#PolishBiotech: How Cell and Gene Therapies Are Transforming Healthcare??? Cell and gene therapies represent one of the most groundbreaking advancements in modern #medicine, offering hope for diseases once considered untreatable. By leveraging cutting-edge technology, these therapies use?cells?or #genetic material?to treat, prevent, or even cure complex conditions like #cancer, genetic disorders, and #degenerative diseases. ?? What makes?cell and gene #therapies?so revolutionary? ???Cell Therapies:?Using or modifying living cells or proteins to restore health. Examples include #CAR-T therapies, which reprogram #immune cells to fight cancer. ???Gene Therapies:?Introducing, #editing, or replacing genes to correct genetic disorders or provide therapeutic benefits. Technologies like #CRISPR have unlocked incredible potential in this field. ?? Poland is home to a growing number of biotech #innovators exploring this transformative space. From?cancer #immunotherapy?to #regenerative medicine, these companies are pushing the boundaries of #science and medicine. ?? Swipe below to explore leading Polish companies shaping the future of cell and gene therapies: PolTREG S.A., Polpharma Biologics, Celon Pharma S.A. , Synthaverse. ???This post is part of our series?#PolishBiotech: Companies Shaping the Future, showcasing the dynamic ecosystem of Polish biotech innovators. Dive deeper at?https://lnkd.in/dHy3cPme?and explore our #polishbiotech database! ?? Join us at?CEBioForum 2025?on April 2-3 in Warsaw to discover the full potential of cell and gene therapies and connect with the leaders driving this transformation. Together, let’s shape the future of healthcare! ?? Follow us for more insights into Poland’s thriving biotech industry. #BiotechSpectrum #CellAndGeneTherapies #PolishBiotech #CEBioForum2025 #Innovation #LifeSciences #Biotechnology
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?? This is the topic about which I talked on the radio UMH last Tuesday. ?????? A recent clinical trial, BRILLIANCE, was the first to use gene editing to treat hereditary blindness, more specifically for Leber Congenital Amaurosis (LCA) caused by the CEP290 gene. ?? This trial involved 14 participants who received a single injection of EDIT-101, a CRISPR/Cas9-based medicine. The injection, performed subretinally to reach the patients' photoreceptors, introduced a viral vector based on adeno-associated virus serotype 5 (AAV5), which encoded for the CRISPR/Cas9 system. This system flanked a mutation in intron 26 of the CEP290 gene, cutting it out of the gene and restoring its function. ?? For the injection, different doses were used in different participants, with a low dose in 2 participants, an intermediate dose in 7 participants, and a high dose in 5 participants. To measure the patients' vision, 4 visual tests were performed: -Visual acuity -Participants' ability to see coloured dots of light by looking at a specialised device (full-field test). - Navigation of a maze with physical objects and varying amounts of light - Improvement in their quality of life -Improvement in their quality of life. ??? Among all patients, 11 showed improvement in at least one of the tests, 6 showed improvements in two or more visual tests, 6 showed improved vision-related quality of life, and 4 experienced clinically significant improvement in visual acuity, more specifically the ability to identify objects or letters on a chart. ? In this trial, not only were no dose-related toxic effects observed, but no serious adverse effects were seen in any participant, demonstrating that this therapy is not only effective, but also safe. ?? Source: Gene Editing for CEP290-Associated Retinal Degeneration, The New England Journal of Medicine (DOI: 10.1056/NEJMoa2309915). ??? Image created with Biorender.com. #science #sciencecommunication #medicine
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??????????: ?? ???????? ???? ?????????????????????????? ???? ???????? ?????????????????? As we look ahead to 2025, it's clear that the Life Sciences sector continues to experience transformative growth, driven by innovation across various fields. From advancements in gene therapy to AI-powered drug discovery, companies are redefining what's possible in healthcare. ?????????????????? ?????????????? ?????? ????????????: ? Kriya Therapeutics, Inc. is revolutionising gene therapy with its $600M investment, focusing on rare and chronic diseases, and advancing its capabilities through cutting-edge computational platforms ? ElevateBio is making waves in gene editing, with a flexible post-CRISPR platform that could reshape the treatment of genetic disorders ? Moderna continues to lead the charge in mRNA technology, expanding its platform beyond COVID-19 vaccines to target other diseases, including cancer and rare genetic disorders ? Gilead Sciences is focusing on groundbreaking treatments for chronic diseases, such as HIV and liver diseases, using gene therapy and antiviral technologies ? ?????? ???????? ??????????????: These advancements are not just about creating new drugs, but also about improving patient outcomes through precision medicine, faster drug development, and novel therapies that tackle diseases previously thought to be intractable. With the rapid pace of innovation, what will be the most significant impact on the future of healthcare? #LifeSciences #Innovation #Biotech #AIinHealthcare #GeneTherapy #MedicalAdvancements ? ???? Eireann E.
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The recent ESGCT Conference in Rome was a great industry snapshot showcasing Cell and Gene Therapy advancements! Our experts highlight are 3 key takeaways from the show and a few notable talks: ??Growing Scope and Demand for CGT: Expansion to treat new disease indications and larger patient populations, with ESGCT attendees showcasing promising pre-clinical innovations worldwide. ??An interesting talk explored using a single-course gene editing technology to treat atherosclerotic cardiovascular disease (ASCVD). These diseases are caused by plaque buildup in arteries leading to coronary heart disease (CHD). Highlights immense potential for global therapeutic impact and future preventive use in a large patient population. ??Another talk showcased curative results for the first human clinical trial using AAV1-hOTOF gene therapy to treat congenital deafness. The treated children regained the ability to hear, speak and respond to auditory stimuli. ??Addressing Cost and Accessibility Challenges: High costs and limited accessibility are major hurdles, leading to the adoption of novel technologies aimed at increasing yields, improving quality, and reducing costs. AI/ML, automation and DoE will be instrumental in accelerating development and reducing costs. ??Talk titled “Manufacturing AAV Vectors: Every Step Matters” highlighted that AAV production is a complex process with many interconnected steps, each of which can be highly optimized. DoE as a tool can accelerate this process and enhance scalability. ??Technological and Sustainable Advancements: Efforts are focused on advancing manufacturing with stem and immune-based cell therapies, including enhancing transduction, and moving toward allogenic platforms to ensure sustainable CGT production. ??An interesting talk explored novel green methods for the production of lipid nanoparticles. The team isolated precursors for LNPs from cashew nut shells (a common regional waste product) to move away from the commonly used petroleum-based sources. Ultimately, there is an acute need to scale life-saving CGTs sustainably and ensure patient affordability and accessibility. ?? Looking to drive up CGT manufacturing yield and drive down manufacturing COGS? ?? Collaborate with us! Check out our technology: https://hubs.la/Q02Wn4ng0 Connect with us to learn more: https://hubs.la/Q02Wn7Nf0 #biomanufacturing #innovation #ESGCT2024
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?? Don't miss this special event hosted by MTIF and CGT Catapult! Are you interested in exploring the latest in cell and gene therapy applications and overcoming the challenges of ATMP manufacturing? This is your chance to learn how MTIF and CGT Catapult are driving advancements in the cell and gene therapy sector and how they can support you in developing clinically and commercially viable ATMPs. Check out the post below for information on how to sign up for the event. #CellandGeneTherapy #Research #Conference #Networking #CGT #ATMP #TranslationalResearch
?? Join Us for an Insightful Event on Translational Pathways and ATMP Manufacturing! ?? ? Are you interested in exploring the latest in cell and gene therapy applications and overcoming the challenges of ATMP manufacturing? ?? We invite you to attend our interactive event, a collaboration between the Medical Technologies Innovation Facility (MTIF) and CGT Catapult at Nottingham Trent University. This is your chance to learn how MTIF and CGT Catapult are driving advancements in the cell and gene therapy sector and how we can support you in developing clinically and commercially viable ATMPs. ?? Event Highlights:? -> Translational non-clinical considerations for ATMP development? -> Innovations in Cell and Gene Therapy Process Development? -> CAR-T Therapy: Breakthroughs and Patient Impact? -> Exploring Cell Therapy Applications in Musculoskeletal Conditions? -> Tackling ATMP Manufacturing Challenges in Academic Settings ?? This event is perfect for academics, clinicians, and industry researchers eager to stay at the forefront of the cell and gene therapy industry. Don't miss the opportunity to connect with experts from MTIF, Catapult CGT, and other key stakeholders in the field. Learn more about the event and how to register via our website - https://lnkd.in/gdY62gNu ?? Date: October 9th? ?? Location: Nottingham Trent University, Clifton Campus? ?? Register now https://lnkd.in/gv4Cy9rU and be a part of the future of cell and gene therapy! #CellTherapy #GeneTherapy #ATMP #TranslationalMedicine #MTIF #CGTCatapult #Biotech #Pharma #HealthcareInnovation?#RTOgrant #Catapultfunding #UKInnovation #InnovateUK ?
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????????????????: ?????? ???????? ?????????????????? ?????????????????????????????? ???????????????? Gary Ruvkun and Victor Ambrose's Nobel Prize-winning discovery of microRNA has unveiled a new frontier in gene regulation, promising to transform how we approach disease treatment. ?????? ?????????? ???? ???????????????? ?????????????????? MicroRNAs offer unprecedented precision in targeting multiple genes simultaneously, surpassing traditional therapies in their potential for comprehensive disease management. These tiny molecules can: ? Fine-tune entire cellular pathways ? Serve as both therapeutic targets and diagnostic biomarkers ? Provide multifaceted regulation of complex diseases ?????? ???? ???????? ?????????? ???? ?????????????? ?????????????? ????????????????????? ???????? ??????????????: Offers more nuanced gene expression control. ???????????????? ??????????????????: Can target intracellular molecules, unlike traditional antibodies. ?????????? ???????????????? ??????????: Provides greater specificity with potentially fewer side effects. ???????????????????? ???? ?????? ?????????????? 1. Developing efficient delivery systems 2. Controlling off-target effects 3. Enhancing stability and longevity in the body ?????? ???????? ?????????? ? Exploring nanoparticle and exosome-mediated delivery ? Utilizing chemical modifications for improved specificity ? Investigating combination therapies with existing treatments As we stand at this exciting crossroads of medical innovation, one question looms large: Will microRNA therapies be the key to unlocking personalized medicine for previously untreatable diseases? What are your thoughts on the potential of microRNA in reshaping the future of healthcare? Share your insights below! ?? #NobelPrize #Genetics #MicroRNA #FutureOfMedicine #Biotech https://lnkd.in/exHySBcK
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???Excited to Share Our Latest Publication!??? Happy to announce that our review article has been published in the?Life Sciences?journal, titled?"Personalized Medicine in Cystic Fibrosis: Exploring Genetic Foundations and Precision Therapeutics." This review explores into the transformative role of personalized medicine in?cystic fibrosis (CF)?treatment, highlighting: ??Genetic Foundations: Exploring the impact of CFTR gene mutations and their correlation with disease severity. ??CFTR Modulators: Advances with Ivacaftor, Lumacaftor, and Tezacaftor, tailored to individual genetic profiles. ??Emerging Therapies: The potential of gene therapy, genetic manipulation, and CRISPR-Cas9 for mutation correction. ??Biomarkers: Novel inflammatory markers and omics technologies for early detection and disease progression monitoring. ??Pharmacogenomics: Personalized approaches to optimize drug efficacy and minimize adverse effects. We also address the ethical and regulatory challenges surrounding equitable access and long-term sustainability of these groundbreaking therapies. As CF care transitions towards precision medicine, we hope this article inspires further research and innovation in the field.??? I thank and congratulate my coauthors Dr. Meghana G S and Dr. Akhila Akkihebbal Ravikumar and Good work Abinesh R S You can read the full article here:?https://lnkd.in/ddGAPduH Looking forward to engaging discussions and collaborations in the area of personalized medicine and CF treatment!??? #CysticFibrosis #PersonalizedMedicine #PrecisionTherapies #GeneTherapy #Pharmacogenomics #LifeSciences #Research #Elsevier
Precision medicine advances in cystic fibrosis: Exploring genetic pathways for targeted therapies
sciencedirect.com
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?? Breaking: Purespring Therapeutics Secures $105M Series B for Kidney Disease Gene Therapy Key Updates: ? Major funding round to advance kidney-focused gene therapy program ? Moving closer to clinical trials ? Strong investor backing validates gene therapy approach Why This Matters for Biotech: This raise signals a pivotal shift in kidney disease treatment, traditionally an area with limited therapeutic innovation. The strong investor appetite demonstrates growing confidence in gene therapy's potential to transform chronic disease management. Strategic Takeaways: 1. Gene therapy is gaining momentum in previously untapped therapeutic areas 2. Precision medicine continues to attract significant investment 3. Nephrology sector is ripe for innovation and disruption What's Next: Watch for emerging opportunities in: ? Gene therapy development ? Strategic partnerships ? Personalized medicine platforms ? Clinical trial developments ?? Question for the community: How do you see gene therapy reshaping treatment paradigms for chronic diseases in the next 5 years? #Biotech #GeneTherapy #KidneyDisease #PrecisionMedicine #BiotechInnovation
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?? ???????????????? ???? ??????????????????’?? ?????????????? ?????????????????? ?? ?? Biomarkers on the Rise: Exciting developments in identifying biomarkers pave the way for earlier and more accurate diagnosis of Alzheimer’s. Such precision can improve treatment outcomes. ?? ?? Innovative Drug Therapies: The emergence of novel drugs, including disease-modifying therapies, offers hope for slowing the progression of Alzheimer's and enhancing quality of life. ?? ?? Gene Therapy Exploration: Cutting-edge research into gene therapy holds promise for targeting the underlying genetic causes, potentially transforming patient care. ?? ?? Non-Pharmacological Innovations: From cognitive training to lifestyle interventions, non-drug approaches are gaining traction, underscoring the power of holistic treatment strategies. ?? For even more insights, check out the latest biomedical literature reviews at https://www.sciqst.com! #AlzheimersResearch #Biotech #MedicalInnovation #SciQst
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