Gene therapy is a cutting-edge approach to treating genetic conditions and other diseases by introducing a healthy version of a specific gene. This method holds immense potential in revolutionizing medical treatment by addressing underlying genetic causes. Gene editing, a related technique, involves making precise changes to genes to correct mutations or introduce beneficial variations. Both […]
Category: gene therapy
Advancements in Gene Therapy for Skin Diseases
Recent breakthroughs in gene therapy have shown promising results in the treatment of inflammatory skin diseases such as psoriasis. While mild cases can be managed with traditional medications, severe symptoms often require systemic drugs with potential side effects. Gene therapy offers a targeted approach by modifying the patient’s genes to address the root cause of […]
Promising Data from First Human Stem Cell Therapy Trial
Exciting advancements have been made in the field of stem cell therapy with promising data emerging from the first human trial. This progress offers hope for a new approach to Alzheimer’s care, potentially aiding in the healing process of the brain rather than just slowing the disease progression. The Phase 1 study is focused on […]
Customizable Lipid Nanoparticle Development for mRNA Delivery in Gene Therapy
Exciting advancements in gene therapy research now offer customizable lipid nanoparticle development services for efficient mRNA delivery systems. These lipid nanoparticles are tailored to support your specific mRNA research needs, providing a one-stop solution for enhancing gene therapy applications. With a focus on innovation and customization, inquire today to revolutionize your gene therapy experiments with […]
Challenges in Gene Therapy for DMD
The year 2024 marked a significant milestone in the field of gene therapy for Duchenne muscular dystrophy (DMD) with the FDA approval of delandistrogene moxeparvovec. This AAV-based gene therapy, developed by Sarepta Therapeutics, delivers a synthetic micro-dystrophin transgene and has shown promise in slowing the progression of the disease. However, despite this progress, challenges remain […]
Gene Therapy’s Regulatory Challenges for Rare Diseases
In a recent update, Rocket Pharmaceuticals Inc. revealed the latest developments regarding RP-A501, their gene therapy designed for treating Danon disease. This rare X-linked dominant genetic disorder is characterized by a clinical triad of cardiomyopathy, skeletal myopathy, and intellectual disability. Despite promising advancements in gene therapy for rare diseases, regulatory uncertainties loom over Rocket Pharma’s […]
Trump Tariffs Threaten Rare Disease Cell and Gene Therapy
The biotech industry is sounding the alarm as Trump’s tariffs put rare disease cell and gene therapy at risk. With over 10,000 rare diseases still lacking FDA-approved therapies, companies like BioMarin are urging the preservation of incentives to sustain innovation. BioMarin, known for gene therapies like Roctavian for hemophilia A, is facing financial challenges despite […]
AAV Gene Therapy Vectors for Hemophilia
Recent research explores the theoretical risk of hepatocellular carcinoma (HCC) following adeno-associated virus (AAV) gene therapy in hemophilia patients. While data from clinical trials are reassuring, concerns remain about long-term oncogenic potential. A single case of HCC post AAV infusion in a patient with prior HCV infection raised questions, but further analysis suggested AAV integration […]
Gene Therapy Vectors for Hemophilia: Efficacy and Safety
Recent studies on gene therapy vectors for hemophilia have highlighted the varying effectiveness and risks associated with different AAV isotypes and gene constructs. Dosages ranging from 2 × 10^11 vector genome [vg]/kg to 6 × 10^13 vg/kg have been tested, showing significant reduction in bleeds for both hemophilia A and hemophilia B patients. Phase-1 trials […]
