bluebird bio’s Post

🔬📖 If you follow me, you know I love to write, maybe too much sometimes. Sorry about that! I am a self-appointed storyteller, but I know every picture tells its own story. Exhibit A below. 🖼️🔬 🙌 Still, I feel I need to add how encouraged I am by the excitement around #viralvector #genetherapies. While only a handful of in-vivo viral vector gene therapies are currently on the market, as of early 2024, a growing number of gene therapy assets are in #clinicaltrials, and obviously, a far greater number are in #preclinical development. 🧬I happen to work for a science-driven company that is uniquely positioned to offer unparalleled access to our team of experts, like Eduard Ayuso, vincent zuliani, Goutham Kumar Ganjam, Florian Leseigneur, Marco Wachowius, and Martin Rhiel. This affords all of those early-phase, pre-clinical gene therapy pioneers a definitively tailored, collaborative experience. From viral construct design to fully integrated, time-saving #scaleup strategies aimed at accelerating the pathway to commercialization, #DINAMIQS is helping our clients clear the hurdles along their asset journey. #CDMO #GeneTherapy #ManufacturingExcellence #ProvenResults #ProcessDevelopment #VectorDesign #Analytics #Upstream #DownStream #Innovation Credit: BioKnow; @andrewpannu

We are almost at the end of #ASGCT2024! There have been many great talks and posters all week, but some of the best have been saved for last. Make sure to stop by today's posters to see some amazing research, including from the FDA! Yes, you read that correctly. At the FDA, we don't just regulate cell and gene therapies. We also have world-class scientists conducting cutting-edge research in the field. For a great example, I highly recommend checking out Sojin Bing's poster (#1559) from Ronit Mazor's lab at CBER. Using rational design, they were able to reduce the #immunogenicity of #AAV9 in mice. This work is incredibly important for addressing the potential side effects of lifesaving #AAV-mediated gene therapies! For more details about their work, go to https://lnkd.in/eUDKmVdg #GeneTherapy #technology #innovation

Our annual publication, Transfer to Transform (T2T) shares the story of technology commercialization at Nationwide Children's Hospital — a story of bold thinkers, exciting new technologies and job-producing startups! We are delighted to share our 2024 T2T, including a featured article, "Innovative Approaches to Patient Care." Tuberous sclerosis complex (TSC) is a devastating genetic disease that affects nearly 1 in every 5,500 newborns and approximately 2 million people worldwide. The most severe form of TSC, Type 2, is caused by mutations in the TSC2 gene. Mark E. Hester, PhD, and colleagues have developed and are now testing a gene replacement therapy for TSC Type 2. The gene therapy uses adeno-associated viruses (AAVs) to deliver a functional version of the gene to affected cells. The researchers have begun preliminary testing and in vitro efficacy studies have shown robust proof-of-concept for further advancement. For more on this article and to view and download the 2024 T2T, click here: https://bit.ly/49pkkla #InnovateWithUs #TechTransferTeam #GeneTherapy #LifeAtNCH

🚀 Unlock the future of gene therapies with our latest webinar in collaboration with Bio-Rad Laboratories! 🌟 💻 Discover more on next-generation, non-replicative HSV-1 vectors for sustainable and more precise gene therapies Gain insights into the current challenges with vectors such as: ⚡Delivery – delivering the gene to the correct cells. Viral vectors can successfully target some key tissues (liver, heart, and muscle), but many tissues remain unreachable ⚡Manufacturing – scalable manufacturing continues to be a challenge across almost all vectors ⚡Potency and durable effect ⚡Limited vector load 🌐💡Don't miss out! Sign up now to explore the cutting-edge advancements in gene therapy: https://bit.ly/47T0RIG #GeneTherapyWebinar #InnovationInScience #BioRadCollaboration

A novel, low-cost, implementable Cell & Gene Therapy solution that needs your support in finding potential partners. Stephan Kadauke, MD, PhD and his team are on a mission to increase accessibility to lifechanging cell and gene therapies at less than 10% of currently available commercial options. Their innovative therapy works to reduce cost barriers, increase accessibility by creating local solutions, and also streamlines the logistics for delivering the care. In other words, they are doing the innovative work to address healthcare inequalities that can be lost in current healthcare systems. Their work can only exist with a community effort. "It would be immensely helpful to connect with others engaged in similar initiatives or those looking to expand access to Cell & Gene Therapies in underrepresented regions." If this sounds like you or someone you know--- please please please reach out, share, and help spread the word so that Stephan & his team can help make this a reality for more patients.

🔬 Researchers at the University of Zurich just made a huge leap: they've found a new way to transport large genes. A discovery that has the potential to be a game-changer for #GeneTherapy. 🧬 The REVeRT method, spearheaded by Prof. Elvir Becirovic and his team, is at the core of this breakthrough. Traditional gene therapy techniques have limitations, especially when handling larger genes. REVeRT not only navigates this hurdle, but it also does so with a heightened level of efficiency and minimal side effects. ❓ So, why is this significant? The real innovation lies in the flexibility this method brings to gene therapy. With REVeRT, large genes can be divided into two fragments at various points, offering a precise approach to treating a broad range of conditions – from common blood disorders to diseases associated with aging. 📍 This goes to show that the #GreaterZurichArea is a hub for cutting-edge research, particularly in #LifeSciences and #BioTech. By building an ecosystem that encourages this level of innovation, we're setting the stage for such groundbreaking discoveries that have global implications. 🔍 For a deeper dive into this revolutionary research, check out our full article here: https://okt.to/YKiRIH. And if you're curious about the flourishing life sciences scene in the Greater Zurich Area, head over to our website: https://okt.to/Yho2cL.

𝗡𝗲𝘄 𝗖𝗥𝗜𝗦𝗣𝗥 𝗣𝗹𝗮𝘁𝗳𝗼𝗿𝗺 𝗳𝗼𝗿 𝗣𝗿𝗲𝗰𝗶𝘀𝗶𝗼𝗻 𝗠𝗲𝗱𝗶𝗰𝗶𝗻𝗲 𝘃𝗶𝗮 𝗘𝘅𝗼𝘀𝗼𝗺𝗲𝘀 🧬 Enhanced Delivery: New "safeEXO-Cas" platform improves CRISPR/Cas9 delivery using exosomes. #CRISPR #GeneEditing 🎯 Targeted Treatment: Customizable exosomes enable precise gene editing, reducing off-target effects. #PrecisionMedicine #Genetics 🧪 Cancer Focus: Platform shows promise in targeting lung cancer cells, enhancing therapeutic potential. #CancerResearch #Therapies 🔬 Interdisciplinary Effort: Collaboration across fields highlights the importance of integrated research. #Interdisciplinary #Research 📈 Broader Impact: Potential to revolutionise gene therapy, offering new hope for treating genetic disorders. #HealthInnovation #GeneticDisorders 🧬 Data Integration: Bioinformatics support such expertise by Independent Data Lab will be crucial for analysing CRISPR/Cas9 efficiency and precision in gene editing. #Bioinformatics #CRO Check it out here: https://lnkd.in/egYd3rQK

Porton Advanced │ Cell & Gene Therapy CDMO/CRO and BioMap Form Exciting Partnership!   Big news from Porton Advanced! We're thrilled to announce a strategic collaboration with BioMap that marks a significant leap forward in the world of genetic medicine and artificial intelligence.   We take pride in contributing our game-changing Adeno-Associated Virus (AAV) vector technology and robust research data to the forefront of this exciting partnership. Together with BioMap, we'll dive into the development of next-gen AAV assembly efficiency models and distribution models. Our goal is to unleash the potential of large-scale life science models to expedite AAV vector design, while broadening the utilization of AI models within the gene therapy landscape.   Porton Advanced and Biomap are united in our mission to bring about positive impact to patients globally. Stay tuned for more updates! #PortonAdvanced #BioMap #GeneTherapy #AI #AAV #AdenoAssociatedVirus #VectorTechnology #ArtificialIntelligence #Partnership

Have you signed up for our upcoming webinar with Bio-Rad Laboratories? Now's the time! Key takeaways: ✅ Discover the rise of HSV-1 Vectors: learn why these vectors are poised to transform gene therapy. ✅ Explore the unique attributes of HSV-1 vectors, ideal for neurological applications and beyond. ✅ Learn how customisation of HSV-1 vectors can unlock a multitude of advantages, from expanding capacity to enabling sustained expression. ✅ Engage with our experts as they share their outlook on the future of cell and gene therapy.