Recent advancements in drug development have highlighted the importance of understanding the Cytochrome P450 (CYP) enzyme system encoded by the P450 gene family. This system plays a crucial role in drug metabolism, with certain drugs being metabolized by multiple CYP isozymes. Variability in the expression and activity of these enzymes among individuals, species, and ethnic […]
Category: gene
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 […]
Personalized CRISPR Gene Editing in Clinical Trials
In a groundbreaking development in the field of gene editing, personalized CRISPR therapy is now being tested in clinical trials. This marks a significant advancement in the CRISPR revolution, as it involves specific gene editing tailored to individual patients. Previous FDA-approved CRISPR therapies targeted blood diseases like sickle cell anemia and thalassemia by removing bone […]
HLA Gene Editing in RheumaGen for Autoimmune Disorders
A breakthrough clinical trial in gene editing by RheumaGen is revolutionizing the treatment of autoimmune disorders. RG0401 targets specific HLA alleles to encode molecules found in individuals resistant to rheumatoid arthritis. By extracting a patient’s hematopoietic stem cells (HSCs) and introducing engineered HLA alleles ex vivo, the therapy aims to create cells with modified HLA […]
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 […]
Optimizing Stem Cell Differentiation Protocols for Macular Degeneration Treatment
In a groundbreaking study, researchers utilized single-cell RNA sequencing to analyze the gene expression profiles of individual human embryonic stem cells as they underwent a 60-day differentiation protocol. This innovative approach not only enabled the mapping of transient states during the cells’ transformation into retinal pigment cells but also led to the optimization of the […]
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 […]
