Otago Innovation Limited’s Post

The CTAD Clinical Trials on Alzheimer's Disease conference 2024 in Madrid provided so much food for thought! Precision medicine is quite a way off to become precise. Where does an individual track regarding Alzheimer's Disease (AD) progression? How do biomarkers and imaging help to understand that individual journey to improve diagnosis and treatment? Trajectory analysis continues to evolve including AI approaches. ApoE4 mutation carrier status remains an important factor regarding AD progression and treatment response. Inroads are made with protein biomarkers including p-Tau 217 in blood and in Cerebral Spinal Fluid (CSF) as well as imaging technology especially with specific tracers. How important is the location of amyloid plaque formation in the brain compared to amyloid load in CSF and/or blood? What insights do tau seeds and tau spread and their locations provide? If amyloid plaque can be depleted with anti-amyloid therapy (and continue to prevent new accumulation), what other treatments could be added to STOP AD progression? Will gene therapy take care of patients that are not suitable for or don't respond to anti-amyloid therapy? What other biomarkers could aid in treatment response monitoring also in clinical practice? We are hopeful that our miRNA biomarkers will add value. They bring AD specific aspects beyond amyloid and tau, and continue to be validated with accepted protein biomarkers. We are looking forward to include other diagnostic and therapeutic approaches!

The Foundation For Rare Diseases (FFRD) @https://lnkd.in/eZSZBe56 has co-developed with the European Reference Network Transplant Child and CVBF (Consorzio per Valutazioni Biologiche e Farmacologiche/Clinical Validation from Biopharmaceutical Findings) @https://www.cvbf.net/ , a MOOC (Massive Open Online Course) titled “Innovative Therapies and Personalized Medicine for Rare Diseases”.   This course has been developed in the context of the European Joint Programme for Rare Diseases EJPRD @https://lnkd.in/gqATb6h, a programme funded under the European Union’s Horizon 2020 research and innovation programme, aiming to create an effective rare diseases research ecosystem for progress, innovation and for the benefit of everyone with a rare disease   The course's objectives are to: Introduce the latest therapeutic treatments developed through medical research, such as gene therapy, protein-based treatments, regenerative medicine, cell therapy, and tissue engineering, and their potential applications in the field of rare diseases and discuss how these treatments offer unprecedented opportunities to treat, modify, reverse, or even cure diseases that were previously deemed incurable. Define personalized medicine and explain how it differs from the traditional "one size fits all" approach. Discuss the current real-world challenges in developing personalized treatments and ways to overcome them.   The MOOC will start on Monday July 8th, and enrollments is now open at: https://lnkd.in/dsmY_-Pj   The access to the content is free for the first 5 weeks after the enrollment.   This course is designed for anyone interested in knowing how recent advances in the medical research field can impact the treatment of rare disorders. The MOOC will be particularly beneficial for medical students, researchers, patient advocacy groups, healthcare professionals, and biotech developers. However, anyone with a curiosity about science, medicine, and rare conditions is encouraged to participate.

🔬 Presenting Breakthroughs in Multiple Myeloma Diagnostics! 🔬 Excited to have had the opportunity to present groundbreaking research on ISM-FISH for detecting IGH translocations in multiple myeloma (MM). Key findings from the research by Tsukamoto et al. (2023) include: - ISM-FISH's remarkable sensitivity, capable of identifying chromosomal translocations with a detection rate of at least up to 1%. - Simultaneous evaluation of three major translocations: t(11;14), t(4;14) and t(14;16) that influence prognosis and treatment options. - Enhanced diagnostic sensitivity over traditional techniques like DC-FISH or G-banding, offering more robust clinical utility. This work provides a promising path forward in MM diagnostics, improving the accuracy of detection and ultimately patient care. This technique could be also useful for evaluating /monitoring the presence of simultaneous hematologic diseases like: B-cell lymphoma or Chronic lymphocytic leukemia. Please check the article to know more. 📄 Study Reference: Tsukamoto, T., Kinoshita, M., Yamada, K. et al. Imaging flow cytometry-based multiplex FISH for three IGH translocations in multiple myeloma. J Hum Genet 68, 507–514 (2023). https://lnkd.in/dGAvt5vK #MultipleMyeloma #Diagnostics #FISH #CancerResearch #Biochemistry #Hematology

Insights on cell therapies for Rare Diseases in Europe! In this short but insightful blog post, Professor Boris Kramer, a renowned expert in neonatology and translational medicine with two European Orphan Drug Designations for spinal cord injury (SCI) and frontotemporal dementia, sheds light on the transformative potential of cell therapies for Rare Diseases, accompanied with a fantastic graphic! 💚 Discover how these innovative treatments can target the root causes of genetic disorders and offer hope to patients with limited options. Professor Kramer shares his vast experience in this field, from preclinical research to clinical trials and regulatory approvals. Read the full article on our website and gain valuable insights into the future of Rare Disease treatment: https://lnkd.in/ehyTHV3T Interested in exploring cell therapy development for your Rare Disease program? Contact us today to speak with one of our experts and learn how we can help bring your therapy to patients. #celltherapy #rarediseases #biotech #innovation #patientcare #EMA

Genomic medicine can improve lives and make health systems more efficient. But as an industry, much more work remains to be done. A widening gap between exists between what is possible and what is actually practiced in modern medicine. To quote from the article: “In a paper published in 2022, researchers showed that genetically based tests and treatments were reaching fewer than 40% of the people with advanced non-small cell lung cancer who may have benefitted from them. For multiple reasons, the other 60% of patients have gained little from the advancement of personalized medicine.” In other words, as this article below explains, building a better personalized medicine system would make these extraordinary benefits much more accessible to larger numbers of patients. Time to innovate. https://lnkd.in/g_XzDz3f

As iPSC leaders, we've applied our decade of expertise to become the first choice for AD researchers looking to benefit from iPSC technology. Our new 2024 "Focus on Alzheimer's Disease" document highlights the latest developments in iPSC-derived Alzheimer's Disease modelling, and gives information on the products and services we provide to power AD research. Read more about our new document and download it here: https://hubs.la/Q02HXYyG0 We can power your AD research with: • axoLines™: we've done the hard work developing 7 iPSC lines (axoLines) reprogrammed from patients with AD. These axoLines can be used for custom differentiation to endpoint neurons and neuroinflammatory cells to build advanced in vitro AD models. • axoCells™: fuel your AD research with functional cells manufactured in our ISO 9001-accredited production facility • axoServices™: let us do the "heavy lifting" with a comprehensive suite of outsourced lab services, including differentiation, gene editing and AD compound screening • axoModels™: let's collaborate as we work to develop a "clinical trial in a dish" (CTIAD) model If you have any questions or would like to discuss a project, email us at [email protected] and let us know what we can do to help! #iPSCs #AD #StemCells #Alzheimer's Disease

🔬 MicroRNAs (miRNAs)! 🔬 miRNAs are small non-coding RNA molecules that play a crucial role in gene regulation. Despite their small size, they have a big impact on cellular processes. miRNAs are involved in the regulation of over 60% of human protein-coding genes. That's a significant portion of our genome under their control! miRNAs act as post-transcriptional regulators, fine-tuning gene expression by binding to target messenger RNAs (mRNAs) and either inhibiting translation or promoting degradation. Dysregulation of miRNAs has been implicated in various diseases, including cancer, cardiovascular disorders, and neurological conditions. Understanding their role in disease pathology is key to developing targeted therapies. miRNAs are incredibly versatile and can function as both oncogenes (promoting tumor growth) and tumor suppressors (inhibiting tumor progression), depending on the context of the cancer type and the target genes involved. Research in miRNA biology is rapidly expanding, with new miRNAs being discovered and characterized regularly. This dynamic field holds promise for uncovering novel insights into gene regulation and disease mechanisms. Harnessing the power of miRNAs opens up exciting opportunities for therapeutic intervention. miRNA-based therapies, including miRNA mimics and antagonists, are being explored as potential treatments for various diseases, offering new avenues for precision medicine. World of miRNAs! 🌟🔍 #miRNA #GeneRegulation #BiomedicalResearch #sciencefacts

As iPSC leaders, we've applied our decade of expertise to become the first choice for AD researchers looking to benefit from iPSC technology. Our new 2024 "Focus on Alzheimer's Disease" document highlights the latest developments in iPSC-derived Alzheimer's Disease modelling, and gives information on the products and services we provide to power AD research. Read more about our new document and download it here: https://hubs.la/Q02HXY3G0 We can power your AD research with: • axoLines™: we've done the hard work developing 7 iPSC lines (axoLines) reprogrammed from patients with AD. These axoLines can be used for custom differentiation to endpoint neurons and neuroinflammatory cells to build advanced in vitro AD models. • axoCells™: fuel your AD research with functional cells manufactured in our ISO 9001-accredited production facility • axoServices™: let us do the "heavy lifting" with a comprehensive suite of outsourced lab services, including differentiation, gene editing and AD compound screening • axoModels™: let's collaborate as we work to develop a "clinical trial in a dish" (CTIAD) model If you have any questions or would like to discuss a project, email us at [email protected] and let us know what we can do to help! #iPSCs #AD #StemCells #Alzheimer's Disease

📅 Upcoming Session Alert! Daniel Chancellor and Isma Hachi, PhD have been sharing a series of teasers leading up to their highly anticipated speaking session, “Knowing Me, Knowing You: A Tête-à-Tête on Global Trends and the Next Wave of Biopharma Innovation” happening on November 4th at 1 PM CET, at BIO-Europe. In this third and final teaser, we delve into the latest biopharma trends across therapeutic areas and modalities: 💡 GLP-1s & Novel Immunology Targets: Tremendous opportunities to build new markets. 📈 Oncology: Maintaining double-digit growth, projected to be worth $375bn by 2030. 🔄 Resurging TAs: CNS and CV are making a comeback. 🧬 Cell & Gene Therapies: 90% of trials run by academic institutions and startups across various diseases and modalities 🛡️ Cell-Based Immunotherapies: CAR T therapies show success in hematological cancers; potential in solid tumors under investigation. 🧪 Stem Cell Therapy Trials: 53% focus on immunology, neurology, and metabolic/endocrinology diseases. 🧠 Gene Therapies: Promising for inherited diseases, with notable success in restoring hearing in children. 🏢 Sponsor Dynamics: Emerging biopharma leads in cell-based immunotherapy (55%) and gene therapy (59%) trials; larger pharma companies have a smaller share but are involved in key areas. 💡 Have questions you’d like us to cover during the BIO-Europe session? Drop them in the comments below, and we’ll address them during the event! Join us on Monday, November 4th at 1 PM CET for more insights. #TherapeuticArea #NewModalities #BIOEurope #BiopharmaInnovation #EBDGroup #Norstella #IQVIA

#RNA-based therapies are reshaping the landscape of modern medicine, offering unprecedented opportunities to treat complex conditions with precision and efficiency. From mRNA vaccines to gene editing tools, RNA technologies are paving the way for innovative, personalized treatments. In our latest review, we explore how these therapies are perfectly suited for musculoskeletal conditions, where the intricate balance of regeneration and repair requires advanced, adaptive solutions. The ability of RNA-based treatments to modulate gene expression, target specific pathways, and promote healing aligns seamlessly with the needs of these challenging conditions. However, to unlock their full potential, pushing research and regulatory frameworks is essential. Progress in preclinical and clinical trials, coupled with optimized delivery methods, will not only enhance therapeutic efficacy but also ensure their widespread adoption in clinical practice. Our perspective highlights the current advancements, challenges, and future directions of RNA-based therapies in orthopedics and regenerative medicine. We’re proud to contribute to the ongoing conversation about how these cutting-edge therapies can transform patient outcomes. thanks to Chiara Mancino, PhDMadeline Franke, MD, MEngClaudia Corbo and the other co-authors! Houston Methodist