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Check out BioSpace's latest article on 6 Recently Approved Cell & Gene Therapies that are revolutionising medicine as we know it! From tackling rare diseases to offering hope for previously untreatable conditions, these breakthroughs are shaping the future of healthcare. Advanced Therapies

It’s Sickle Cell Awareness Month Know the facts about the science behind the corporations research… “Q: Can you comment briefly about the potential risks involved in gene therapies and the long-term effects of the SCD? Gibbons: We still need to better understand and address the potential risks of this curative option. For example, leukemia is a very rare complication of gene therapy in adults with SCD. NHLBI investigators are working to identify the molecular basis of, and advance possible solutions for, blood cancers associated with gene therapy. Panepinto: We don’t know yet if the long-term or chronic complications of SCD will be prevented, such as damage to the kidneys, lung, and brain. And the therapy may not be 100% effective at preventing the most common acute complication, such as pain crises, for all individuals. In addition, the medicine that is needed to allow for the new gene expression can have serious side effects. But building on what is currently known, and working to improve on what we know, will help provide improved therapeutic options and will advance the field and improve the lives of individuals living with SCD.” https://lnkd.in/eMD8itmT

🚀 Thrilling Milestone in the Fight Against Huntington's Disease! 🚀 This past week, UniQure made history by receiving the first-ever FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Huntington’s Disease. This came following impressive Phase I/II trial results for their groundbreaking gene therapy, AMT-130. AMT-130 is a one-time administered gene therapy that's designed to inhibit the production of the mutant huntingtin protein, a key driver of Huntington's Disease. The results? A significant decrease in disease progression over a 24-month trial period. This is not just a win for UniQure but a beacon of hope for all affected by this devastating condition. At Greenstone Biosciences, this news is particularly exciting as we are aligned in our commitment to advancing the understanding and treatment of rare diseases. Our dedication to this mission is reflected in our extensive biobank of human induced pluripotent stem cells which include a variety of lines related to neurological diseases such as Huntington’s Disease, Alzheimer’s Disease, and Parkinson’s Disease amongst others (https://lnkd.in/gJBxJmEK). These serve as a vital resource for discovering new genetic targets and paving the way for future gene therapies such as AMT-130. Join us in celebrating this incredible achievement and stay tuned for more updates as we continue to push the boundaries of science and medicine. 🔗 Learn more about this breakthrough here: (https://lnkd.in/gEjK5AeY). #HuntingtonsDisease #GeneTherapy #Innovation #GreenstoneBiosciences #RareDiseaseResearch #BiotechBreakthrough

Just announced: The first gene therapies for patients with sickle cell disease (12 years and older) have just been approved by the FDA. These products offer new options and hope for patients living with SCD, a disease which has been historically neglected and underfunded. Casgevy is also the first FDA-approved therapy which utilizes CRISPR/Cas9, underscoring the power and potential of gene editing in the treatment and cure of disease. #cellandgenetherapy #sicklecelldisease #crisprcas9 https://lnkd.in/epKKU9j5

🌟 Embarking on a Journey of Discovery in Gene Therapies In recent years, the resurgence in gene therapies has caught attention, providing hope for transforming cancer and rare disease treatments. Grateful for the chance to connect with influential minds in Cell and Gene Therapy (CGT), such as Dr. Rahul Purwar, Dr. Albeena Nisar, and Dr. Lakshmikanth Gandikota. Their insights have not only enriched my understanding of CGT's progress but have also highlighted the critical needs in this evolving field. 🚀💬 Insightful discussions with them clearly showed how CGT is changing the way we will take care of cancer in the future. The ability to tailor treatments to individual genetics is no longer a distant dream but a tangible reality. 💡 Being part of an organization contributing significantly to CGT advancements is a source of immense pride. Witnessing firsthand the dedication to providing seamless solutions from discovery to commercialization, I'm inspired by the Thermo Fisher Scientific tools and technologies propelling CGT into uncharted territories. https://lnkd.in/dmbsvrXe 🚀 Reflections from Recent Dialogues: Precision Tailoring: CGT introduces a new era of precision, providing treatments unique to each patient's genetic makeup. *Immunotherapy does wonders: CGT orchestrates therapeutic vaccines, presenting a targeted and less invasive approach by harnessing the body's immune system. *Genetic Editing Frontiers: The breakthroughs with CRISPR and gene-editing tools are not just rewriting possibilities; they are rewriting the future of treating genetic disorders. 💭 Join the Conversation: Inspired by recent dialogues, I'd love to hear your thoughts and experiences in the comments. Together, let's shape the future of cancer care! 🌍💙 Amy Butler; Amit Chopra, Kim Baltier, Julie Planchet, Hong Hwei Ng, Pablo Ramos Luque, Geoffrey Poon #CGTInsights #ThermoFisherImpact #FutureOfCancerCare #RevolutionizingCancerTreatment

It is so exciting to see cell and gene therapies getting through the research phase to become accessible to all patients in need. Here is to 2024 being another great year for these life saving medicines! Check out this article on Biospace to learn more about the 7 FDA approvals granted to cell and gene therapies in 2023. #CellTherapy #GeneTherapy #MedicalResearch #PatientCare #TranslationalMedicine #BenchtoBedside

Unlike single gene approaches, explore how Ocugen’s gene therapies for eye diseases target master genes rather than individual genetic mutations #AdFeature with Ocugen https://lnkd.in/efR6dgSz

New Blog: Navigating the Complex World of Primary Immunodeficiencies (PIDs) This Primary Immunodeficiencies week, Caroline Beltran explores the intricate landscape of PIDs—from diagnosis difficulties to pioneering treatments such as gene therapy. Uncover the advances that are reshaping how we manage these complex immune disorders. 👉 Read here: https://lnkd.in/ewgfvj4A Stay tuned for part 2 of this blog later this week. #PrimaryImmunodeficiencies #GeneTherapy #LifeSciences #HealthcareInnovation #SynexaLifeSciences

Gaurav Shah, MD, CEO, and Kinnari Patel, PharmD, MBA, President and COO, recently spoke with Forbes about how Rocket is revolutionizing gene therapy to help change the trajectory of inherited, rare cardiovascular diseases, including #DanonDisease, plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) and BAG3-associated dilated cardiomyopathy (BAG3-DCM). Rocket’s cardiovascular #GeneTherapy programs have the potential to impact more than 100,000 patients in the U.S. and Europe. Our focus remains on finding potentially curative treatments for patients facing life and death, who don’t have time on their side, and we will continue our efforts to fulfill our mission to develop gene therapies for these devastating rare diseases. Read the article here: https://lnkd.in/gN7mSNxd

This excellent review by Wang et. al. describes adeno-associated virus as a delivery vector for gene therapy of human diseases. Quoting from the abstract: "Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for treating various diseases. However, as rAAV is being more widely used as a therapy, the increased demand has created challenges for the existing manufacturing methods. Seven rAAV-based gene therapy products have received regulatory approval, but there continue to be concerns about safely using high-dose viral therapies in humans, including immune responses and adverse effects such as genotoxicity, hepatotoxicity, thrombotic microangiopathy, and neurotoxicity. In this review, we explore AAV biology with an emphasis on current vector engineering strategies and manufacturing technologies. We discuss how rAAVs are being employed in ongoing clinical trials for ocular, neurological, metabolic, hematological, neuromuscular, and cardiovascular diseases as well as cancers. We outline immune responses triggered by rAAV, address associated side effects, and discuss strategies to mitigate these reactions. We hope that discussing recent advancements and current challenges in the field will be a helpful guide for researchers and clinicians navigating the ever-evolving landscape of rAAV-based gene therapy."