PackGene Biotech, Inc.

🔬 PackGene at University of Maryland, Baltimore – Let’s Talk Science! We're excited to be on campus for the Life Science Exhibit at UMB! Come meet Diana Eng and discover how our AAV, lentivirus, mRNA, and plasmid platforms are transforming research and therapeutic development. 📍 SMC Campus Center, 2nd Floor 🗓 Tuesday, April 15, 2025 🕚 11:00 AM – 1:00 PM EST 📌 621 West Lombard Street, Baltimore, MD Grab some giveaways, ask questions, and let’s talk about how we can help accelerate your work! #UMBBiotech #GeneTherapy #AAV #mRNA #Lentivirus #PlasmidProduction #ResearchSolutions

This past month marked Colorectal Cancer Awareness Month. We spotlighted the evolving role of CAR-T therapy in colorectal cancer, where promising clinical trials and cutting-edge genetic engineering strategies are offering renewed hope in tackling metastatic CRC. #CARTtherapy #CRC #Oncology #LVV #Packgene

🚀 GeneVentiv Therapeutics has acquired a global license for GENV-002, a groundbreaking gene editing therapy developed in Dr. Dwight Koeberl's lab at Duke University, designed to treat all forms of Pompe disease. GENV-002 (AAV9.GAA and AAV9.Cas9-gRNA) is a pioneering gene editing therapy using liver depot strategy, enabling stable GAA transgene expression from early infancy—a critical advancement beyond current gene therapies. This innovative approach offers a potential cure for 120,000+ individuals, overcoming limitations of current treatments. 🌟 #CRISPR #GeneTherapy #RareDiseases #Pompedisease #AAV #Packgene https://lnkd.in/eNQ5xmr6

Sarepta Therapeutics and Roche shared that an independent data monitoring committee has reviewed Elevidys, an AAVrh74-based gene therapy delivering a micro-dystrophin gene to treat Duchenne muscular dystrophy (DMD). The DMC confirmed a favorable benefit-risk profile, allowing dosing to continue in three clinical trials (Phase 1 Study 104, Phase 2 ENVOL, Phase 3 ENVISION) without changes. 🧬💉 Despite a prior pause in EU trials following a patient’s death from acute liver failure (ALF) in the U.S., linked partly to a CMV infection, Sarepta is working with the EMA to resume and expects no major delays. Elevidys, dosed at 1.33 x 10¹⁴ vg/kg via IV infusion, uses the MHCK7 promoter for muscle-specific expression. Patient safety remains the priority! #GeneTherapy #DMD #Clinicaltrials #AAV #Packgene https://lnkd.in/e9X4sFPr

🚨 Clinical Update from Opus Genetics! 👁️ Opus Genetics shared encouraging 1-month data from the first pediatric patient treated with its investigational gene therapy OPGx-LCA5 for LCA5-related inherited retinal disease (IRD): ✅ Improved visual function reported ✅ No treatment-related adverse events ✅ Second pediatric patient dosed; full cohort enrollment expected in Q2 2025 ✅ Initial data from all three expected in Q3 2025 🧑⚕️ The therapy uses AAV8 to deliver a functional LCA5 gene directly to the retina—offering hope for patients with no approved treatments today. 🧠 An FDA Type D meeting also helped clarify the design for a potential pivotal trial in 2026, bringing this therapy one step closer to patients. #GeneTherapy #Ophthalmology #RareDisease #RetinalDegeneration #oculardisorders #AAV #Packgene https://lnkd.in/e-zzTpsz

Research in mice with Huntington's disease showed that using BDNF and Noggin can help the brain make new neurons 🧠. These new neurons can connect to and fix damaged motor circuits 💪, suggesting a possible treatment strategy. AAV4 vectors were used to deliver to BDNF and Noggin to the brain for a long-lasting effect 🧬. The study showed that these newly generated neurons connect with the complex networks in the brain responsible for motor control, replacing the function of the neurons lost in Huntington's, and even contributing to improved motor behavior in the mice. This approach, which encourages the brain's own repair mechanisms, could potentially be combined with other cell replacement therapies ✨. #HuntingtonsDisease #BDNF #Noggin #Neuroregeneration #Neurorepair #RegenerativeMedicine #GeneTherapy #AAV #Packgene https://lnkd.in/eFq5CAyS

🌟Significant progress in allogeneic CAR T therapy for autoimmune diseases. Allogene Therapeutics' ALLO-329, targeting CD19 and CD70, has been granted three FDA Fast Track Designations for SLE, IIM, and SSc. The company is advancing towards a Phase 1 trial with innovative lymphodepletion strategies. #CART #Autoimmunity #celltherapy #AllogeneTherapeutics #ClinicalTrials #LVV #Packgene https://lnkd.in/dBhmPNVM

🌟Intellia Therapeutics has reached a crucial stage in the development of nexiguran ziclumeran (nex-z), a one-time CRISPR gene therapy for familial amyloid polyneuropathy (FAP), with the dosing of the first patient in its Phase 3 MAGNITUDE-2 trial. 💡 This global study follows promising Phase 1 results demonstrating significant reduction of the TTR protein. The potential for a single-dose treatment to halt or reverse FAP progression is significant. #GeneEditing #CRISPR #FAP #ATTR #Intellia #LNP #Packgene https://lnkd.in/dtmmjybp

Recent Gene Therapy research from Dariusz C. Górecki, Ph.D., and colleagues emphasizes the critical role of the immune response to re-expressed dystrophin in developing permanent Duchenne Muscular Dystrophy (DMD) treatments. Gene therapy, while promising, faces a major challenge: 💡The immunogenicity of both viral vectors and the newly expressed dystrophin protein. 💡The immunogenicity of re-expressed dystrophin is a particularly pressing and complex issue that has received insufficient attention. 💡Therapeutically expressed dystrophin can trigger an immune response because the protein contains new antigenic epitopes not present during immune system development. The article discusses factors involved in these immune responses and their impact on treatment outcomes: 💡Key unresolved questions include the mechanisms of immunity induction, the impact on therapeutic efficacy, and the role of patient-specific risk factors (e.g., revertant fibers, inflammation, pre-existing T cells). 💡Assessing a patient's immune status before therapy could be an important step in mitigating anti-dystrophin responses. 💡This assessment might warrant the development of new testing and therapeutic approaches. Addressing the unanswered questions surrounding these immune mechanisms 🔓 is essential for refining gene therapy (i.e., AAV) approaches and ultimately creating more effective and well-tolerated treatments for DMD, offering a more promising future for those affected. #DMDresearch #GeneTherapy #NeuromuscularDisorders #Immunogenicity #AAV #Packgene https://lnkd.in/dfGj7j4u