Rejuvenate Bio’s Post

🔬 Unlocking Disease Mechanisms with Gene Knockout and Knock-In Models 🧬 In our most recent article we explore the powerful applications of gene knockout and knock-in models in understanding and treating various diseases. Key Highlights: -Innovative Tools: Discover the latest in gene editing technologies, including CRISPR/Cas9, TALENs, and ZFNs. -Understanding Diseases: How these models help decode gene functions and disease mechanisms. -Therapeutic Development: The role of gene editing in developing cutting-edge treatments like CAR T-cell therapy. -Challenges and Future Directions: Insights into the limitations and potential of these models in disease research. These technologies are advancing our knowledge of diseases like cancer, cardiovascular conditions, and neurodegenerative disorders and paving the way for novel therapeutic strategies. 🔗Read the article here: https://lnkd.in/eyJSthUv #GeneEditing #CRISPR #BiomedicalResearch #DiseaseMechanisms #TherapeuticDevelopment #GeneticResearch #Biotechnology #HealthInnovation #PrecisionMedicine

🔬✨ Transforming #Healthcare with Cell & Gene Therapies! ✨🔬 Pharma R&D is pushing boundaries with cell and gene therapies, offering new hope for genetic disorders like cancer and autoimmune diseases. These innovative treatments use living cells and #DNA/#RNA to replace faulty genes or cells, changing lives. 🌿🧬 🔍 How do Cell & Gene Therapies (#CGT) work, and what makes them so effective? 🌟 Our article explores the crucial role of #AncillaryMaterials (AMs) in these groundbreaking treatments. 🚀 Technologies like #CRISPR, CAR-T, and #AI-driven personalised medicine are pushing the boundaries of medical science, promising more precise and effective treatments. Learn more about these revolutionary therapies in Đurđa Zdravković's and Dr. Hürter's article: https://lnkd.in/epNZJj5h #innovation #cellandgene

Researchers have developed a gene therapy approach using an advanced prime editing system to correct the F508del mutation, which causes cystic fibrosis. This technique allows precise editing of DNA to repair the mutation in patient-derived lung cells. The restored function of the edited cells matched current CF drug therapies, indicating the potential of gene therapy as a one-time treatment. However, the challenge remains in effectively delivering the gene-editing system to patient cells.

📢 New report just released: Strengthening Pathways for Cell and Gene Therapies: Current State and Future Scenarios. Cell and gene therapies can provide transformative benefits to patients who are often left with no other treatment options. Recent years have seen record funding, clinical research, and new launches in this sector. Despite this increasing activity, there are significant barriers to the future success and sustainability of these treatment modalities. This report provides a foundation for meaningful discussions about the future of the sector and the role cell and gene therapies will play in healthcare. Download the report to read about: 🧬the current state of these therapies across the product lifecycle, beginning with pre-commercial activities such as funding and clinical research. 🧬the hurdles to commercialization, including infrastructure needs, reimbursement, and patient access. 🧬potential future scenarios across eight indicators that can be monitored to understand the evolution of the sector through 2035 and beyond. https://bit.ly/49TlrL4 #CellTherapy #GeneTherapy #LifeSciences #Healthcare

🌟 Unlocking the Future of Healthcare: Insights from Cell and Gene Therapies 🌟 1) Revolutionizing Treatment: Cell and gene therapies hold immense promise for patients with limited options. As of the end of 2023, 76 cell and gene therapies have been launched globally, more than double the number of therapies that had been launched by 2013. 🚀 2) Navigating Challenges: The treatment journey for patients who may be eligible for treatment with a cell and gene therapy is complex, and numerous barriers exist throughout the process. 💡 3) Looking Ahead: Significant uncertainty still exists about the future trajectory and sustainability of the cell and gene therapy sector, with a range of future scenarios possible across eight key indicators which will define the future through 2035 and beyond 🌐🔍

The first project in this program to advance gene therapies for very rare conditions will focus on CHAT-mutated congenital myasthenic syndrome.

I'm thrilled to share news that Charles River Laboratories has just announced an exciting partnership with Navega Therapeutics. 🧬🤝 The collaboration, emerging from Charles River's Cell and Gene Therapy Accelerator Program, promises to harness the power of adeno-associated virus 9 (AAV9) in creating a pioneering gene therapy - NT-Z001. This is not just another treatment; it's a non-opioid alternative that's desperately needed in the current medical field. 💉 What's truly groundbreaking is how NT-Z001 targets a specific gain-of-function mutation. By focusing on the Nav 1.7 gene, it could alleviate inherited erythromelalgia, small fiber neuropathy, and other severe pain disorders. This alliance signifies a leap forward in making this gene therapy candidate available for phase 1 clinical trials, with Charles River providing pivotal contract development, manufacturing capabilities, and expert advisory services. 💡 The implications go far beyond rare diseases - there's potential for broader pain conditions, like neuropathic and inflammatory pain, to be addressed as well. Kudos to both teams for leading the charge toward a future where pain management can be effective without the risks opioids present. This is the power of precision medicine. 👏 #GeneTherapy #ChronicPain #PersonalizedMedicine #CharlesRiver

With the advent of cell and gene therapies, enabled by CRISPR and next-generation gene editing technologies, we are seeing therapies emerge that have the potential to address and correct the genetic causes of disease, offering a possibility for permanent treatment

🔬 Unveiling Advancements in Gene & Cell Therapy at BSGCT 2024! 🧬 Dr. Ronit Mazor, a distinguished Principal Investigator at the U.S. Food and Drug Administration's Office of Gene Therapy, will be presenting at The Boston Society Gene & Cell Therapy 2024 Conference. In her insightful presentation titled 'Can the Immunogenicity of AAV Vectors be Mitigated?', Dr. Mazor tackles a critical aspect of adeno-associated virus (AAV)-mediated gene therapy. Despite proven clinical safety, immunogenicity remains a challenge. Dr. Mazor's innovative approach involves silencing the most immunodominant T cell epitope in AAV9 through rational design chimerism. This breakthrough ensures that the chimeric vectors maintain their functions and potency, while effectively avoiding any cellular immune responses. About Dr. Ronit Mazor: With an impressive background, Dr. Mazor leads a dedicated research group at the FDA, focusing on understanding cellular and humoral immune responses to gene therapy vectors. Her responsibilities extend to critical contributions in CMC reviews for BLA and IND submissions of biological products, particularly in the realm of viral vector gene therapies. Before joining the FDA, Dr. Mazor made significant contributions as a senior scientist at Medimmune/AstraZeneca, specializing in antibody discovery and protein engineering with a focus on predicting and mitigating immunogenicity of therapeutic proteins. This conference session promises a deep dive into the latest advancements in gene therapy, guided by Dr. Mazor's expertise. Secure your participation today to be part of this enlightening discussion shaping the future of gene and cell therapy. For more details, please visit us at: https://lnkd.in/dKe_9Rsg #GeneCellTherapy #BSGCT2024 #Immunogenicity #GeneTherapy #CellTherapy #FDA #Innovation #Biotechnology #Healthcare #Conference #Research #ScientificAdvancements Stephanie Pasas Farmer, PhD, Yan G. Ni, Anshul Gupta, Steven Louie, Jochem Gokemeijer

First ever CRISPR gene therapies for sickle cell patients are underway by 2 companies.... 💉 Vertex’s Casgevy: Their gene therapy, developed with CRISPR Therapeutics, targets sickle cell disease by editing the faulty hemoglobin gene. Patients have begun receiving infusions after a slow rollout. #GeneTherapy #CRISPR #SickleCell 🧬 Bluebird bio’s Lyfgenia: Lyfgenia, approved for sickle cell disease, works by replacing patients’ defective hemoglobin-producing cells with genetically corrected ones. Initial treatments have also commenced. #GeneTherapy #SickleCell #BluebirdBio If this works, then this could be a huge milestone for gene therapies. Source: https://lnkd.in/eKBYbcHG