Orange County Bio’s Post

🔬 Exciting News in Gene Therapy! 🔬 bluebird bio has reached a pivotal milestone with the successful completion of the first commercial cell collection for LYFGENIA™ (lovotibeglogene autotemcel), their innovative gene therapy for sickle cell disease. This marks a significant step forward following FDA approval in December 2023. #LYFGENIA™, designed to treat patients over 12 with a history of vaso-occlusive events, utilizes a one-time ex-vivo lentiviral vector gene therapy. The therapy aims to reduce disease symptoms by introducing a functional β-globin gene into the patient's own hematopoietic stem cells. This process promises to produce adult hemoglobin with anti-sickling properties, potentially minimizing the frequency of painful sickle cell crises. The first cell collection was conducted at Children's National Hospital in Washington, DC, part of a network of over 60 Qualified Treatment Centers. This network comprises centers recognized for their expertise in transplant, cell and gene therapy, and sickle cell disease. David Jacobsohn, MD, MBA, Chief of the Division of Blood and Marrow Transplantation at Children’s National, expressed his pride in leading the commercial application of this groundbreaking treatment, highlighting its promise as a "groundbreaking treatment alternative" and a "beacon of hope" for patients and families affected by sickle cell disease. As bluebird bio continues their long-term safety and efficacy follow-up study (LTF-307), the healthcare community watches eagerly, hopeful for the positive impact LYFGENIA™ might have on those it aims to help. Please visit the following link for further details: https://lnkd.in/gTkD7S7E #Healthcare #Pharmacy #GeneTherapy #SickleCellDisease #bluebirdbio #Biotech

🔹 In an unprecedented breakthrough, scientists at the Children's Medical Research Institute have successfully conducted the first gene therapy trials on a complete human liver, marking a milestone in the quest for effective treatments against severe inherited diseases. Using a normothermic liver perfusion system, this pioneering study opens up new possibilities for directly assessing adeno-associated virus-based therapies in the target organ, potentially overcoming current limitations of preclinical models and accelerating the development of more efficient and safer gene therapies. This innovative approach not only promises to improve the precision of therapeutic dosing and the identification of side effects but also to reduce reliance on animal trials, bringing us one step closer to real solutions for patients facing diseases with limited treatment options. Follow the link below to learn more 👉🏼 https://lnkd.in/dznmPyAW #techtitute #medicine #genetherapy #perfusion

Gene editing targeting lungs shows promise in preclinical study A preclinical study by ReCode Therapeutics shows promise for a lung-targeted gene-editing therapy designed to correct the CFTR gene mutation in cystic fibrosis (CF) patients. Using Selective Organ Targeting (SORT) lipid nanoparticles (LNPs), the therapy effectively targeted lung stem cells in mice through intravenous infusion. This method demonstrated significant gene correction, with potential for long-lasting treatments for CF and other genetic disorders. ReCode's lead candidate, RCT2100, which delivers healthy CFTR mRNA, is currently in Phase 1 trials. Further safety studies are needed before moving to clinical applications. To read more: https://lnkd.in/gDXH6DjT #CFA #CFAustralia #CysticFibrosisAustralia #News #ReCodeTherapeutics

We're proud to share an exciting milestone reached by Zhongmou Therapeutics Technology (Wuhan) Co., Ltd. in the fight against inherited retinal diseases. Their latest clinical data reveal significant advancements in optogenetic gene therapy, specifically targeting Retinitis Pigmentosa (RP). This therapy, known as ZM-02, has demonstrated notable improvements in patient vision, offering hope to millions worldwide suffering from this degenerative condition. A universal solution to a previously incurable challenge, ZM-02 stands as a testament to the power of innovation and dedication in healthcare. Congratulations to the team at Zhongmou Medical for their groundbreaking work in ophthalmology. A pivotal component of this success is the collaboration with PackGene Biotech, Inc., who provided the essential Adeno-Associated Virus (AAV) vectors used in the therapy. This partnership exemplifies how collaboration and innovation can lead to groundbreaking advancements in medical science. Learn more: https://lnkd.in/gVvdF6-M #GeneTherapy #RetinitisPigmentosa #Optogenetics #AAV

Scientists at the Children's Medical Research Institute have made a groundbreaking advancement in gene therapy by testing AAV-based therapies in whole human livers using an ex situ normothermic perfusion system. This novel method allows for the direct evaluation of gene therapies within the human liver, overcoming the challenges posed by traditional preclinical models. Key findings include the superior performance of AAV-SYD12 and AAV-LK03 variants under non-neutralizing conditions and the identification of challenges related to neutralizing antibodies. This research opens new possibilities for developing effective liver-directed gene therapies, marking a significant step forward in personalized medicine. https://lnkd.in/g4YHcvxd

Cell & Gene therapy is a revolutionary field that has transformed the landscape of disease treatment and now sponsors are exploring alternative ways to study cell and gene therapies.

1st Gene therapy for Metachromatic Leukodystrophy.

#molecularbiology #primerdesign The first prime editor will soon enter the clinical phase #Chronicgranulomatousdisease (CGD: Chronic Granulomatous Disease) is a rare hereditary disorder that weakens the body's #immune system. A genetic defect in the NADPH Oxidase enzyme gene causes xenobiotic cells to not function properly in dealing with various infections, and this condition causes patients to be exposed to serious infections. 👉Pioneering PM359 gene therapy provided by Prime Medicine Company is promising news in this field. This method is the first gene editing in humans to target this debilitating disease. This innovative treatment uses a new method called "Prime editing" to precisely correct the genetic mutation of the gene responsible for CGD in the patient's own hematopoietic stem cells (HSCs). These stem cells, which are responsible for the production of blood cells, are modified outside the body (extracorporeal) and then injected into the patient. In this way, this method has the ability to provide a permanent solution and cure for this disease. 👉Receiving #Food and #Drug Organization (FDA) approval for a phase I/II clinical trial for PM359 is an important step in evaluating its safety and effectiveness in patients with CGD. The design of the trial was carried out with high accuracy and included the registration of participants in three separate groups. Initially, adults with CGD with stable disease status will enter the trial to assess the safety of this treatment. If PM359 shows positive results in this initial group, the trial will be expanded in the next phase to groups including adolescents, children and even patients with active infections or severe inflammation. This step-by-step approach, while ensuring patient safety, helps to gradually evaluate the effectiveness of PM359 in a wider population of patients with CGD. 👉The development of PM359 shows a revolutionary progress in the field of gene editing and is promising for the treatment of patients with CGD. This treatment offers a potential therapeutic approach, unlike current treatment methods that mainly focus on controlling infections. Beyond CGD, Prime Medicine is actively developing gene editing treatments for various other diseases. The success of PM359 could pave the way for a new era of gene-based therapies and provide revolutionary treatment options for many untreatable genetic diseases. Key phrase: Prime editing: A new type of gene editing method that, unlike the CRISPER/Cas9 method, enables the rewriting of short sections of DNA without the need to create double-strand breaks. More information👇 https://lnkd.in/eC23n58f

Excited to share a new publication in the Journal of Molecular Therapy which highlights the eShunt®️ Platform as a CNS gene therapy delivery method. Thanks to the teams at UMass Chan Medical School and Tufts Medical Center for your work on this study. "We're very excited about these promising preclinical results, which suggest that the eShunt System delivery approach could provide biopharmaceutical and biotechnology organizations with a minimally invasive method for delivering gene therapies directly to the cortex and brain." #bbb #CNS #drugdelivery #neurovascular #endovascular https://lnkd.in/eeKCGPm7

Gene therapy is revolutionizing modern medicine, but finding a safe and effective delivery vehicle remains a critical challenge. While liposomes offer significant promise over viral-based delivery methods, they struggle with limited delivery efficiency across various cell types. A key obstacle is our incomplete understanding of the cellular structures necessary for liposome uptake, particularly in migratory versus epithelial cells. In our latest research, we've discovered the cellular structures required for efficient liposome-based gene delivery using human pluripotent stem cells. This advancement could pave the way for more effective and safer gene therapy applications in the future. #GeneTherapy #BiomedicalResearch #StemCells #Liposomes #MedicalInnovation #NIGMS #NIH #NEI Basic and Translational Research, Dept of Ophthalmology, IUSM https://lnkd.in/gvCfnAwM