A5060321767- Virus-based gene therapy research
- Lysosomal Storage Disorders Research
- Viral Infectious Diseases and Gene Expression in Insects
- CAR-T cell therapy research
- Biosimilars and Bioanalytical Methods
- Nuclear Receptors and Signaling
- Monoclonal and Polyclonal Antibodies Research
- Neurological disorders and treatments
- Botulinum Toxin and Related Neurological Disorders
- Pancreatic function and diabetes
- CRISPR and Genetic Engineering
- Musculoskeletal pain and rehabilitation
- Intraocular Surgery and Lenses
- Glaucoma and retinal disorders
- Cancer Treatment and Pharmacology
- 3D Printing in Biomedical Research
- Advanced Biosensing Techniques and Applications
- Phosphodiesterase function and regulation
- Animal Genetics and Reproduction
- Diabetes Treatment and Management
- Diabetes and associated disorders
- RNA Interference and Gene Delivery
- Biochemical and Molecular Research
- Healthcare and Venom Research
- Cell Image Analysis Techniques
Sangamo BioSciences (United States)
2019-2025
XOMA (United States)
2009-2015
Zinc-finger nuclease (ZFN)-based in vivo genome editing is a novel treatment that can potentially provide lifelong protein replacement with single intravenous administration. Three first-in-human open-label ascending single-dose phase 1/2 studies were performed parallel (starting November 2017) primarily to assess safety and tolerability of ZFN therapy mucopolysaccharidosis I (MPS I) (n = 3), MPS II 9), hemophilia B 1). Treatment was well tolerated no serious treatment-related adverse...
Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the alpha-galactosidase A (GLA) gene, which encodes exogalactosyl hydrolase, (α-Gal A). Deficient α-Gal activity results progressive, systemic accumulation of its substrates, globotriaosylceramide (Gb3) and globotriaosylsphingosine (Lyso-Gb3), leading to renal, cardiac, and/or cerebrovascular early demise. The current standard treatment for enzyme replacement therapy, necessitates lifelong biweekly infusions...
Abstract Patients with hemophilia A require exogenous factor VIII (FVIII) or nonfactor hemostatic agents to prevent spontaneous bleeding events. Adeno-associated virus (AAV) vector–based gene therapy is under clinical investigation enable endogenous FVIII production. Giroctocogene fitelparvovec a recombinant AAV serotype 6 vector containing the coding sequence for B-domain–deleted human F8 gene. In ongoing phase 1/2, dose-ranging Alta study, 4 sequential cohorts of male participants severe...
The 16th Workshop on Recent Issues in Bioanalysis (16th WRIB) took place Atlanta, GA, USA September 26–30, 2022. Over 1000 professionals representing pharma/biotech companies, CROs, and multiple regulatory agencies convened to actively discuss the most current topics of interest bioanalysis. WRIB included 3 Main Workshops 7 Specialized that together spanned 1 week order allow exhaustive thorough coverage all major issues bioanalysis, biomarkers, immunogenicity, gene therapy, cell therapy...
The 18th Workshop on Recent Issues in Bioanalysis (18th WRIB) took place San Antonio, TX, USA May 6-10, 2024. Over 1100 professionals representing pharma/biotech companies, CROs, and multiple regulatory agencies convened to actively discuss the most current topics of interest bioanalysis. WRIB included 3 Main Workshops 7 Specialized that together spanned 1 week allow an exhaustive thorough coverage all major issues bioanalysis biomarkers, immunogenicity, gene therapy, cell therapy vaccines....
Abstract Recombinant adeno-associated virus (AAV) vectors are the leading platform for gene delivery a variety of clinical applications. Patients with preexisting antibodies to AAV currently excluded from most therapy trials avoid vector neutralization and ensure response therapy. Anti-AAV neutralizing (NAbs) typically assessed by in vitro cell-based transduction inhibition (TI) assays. However, relevance determined enrollment cutoff inherent variability assay present challenges use as an...
XMetA is a fully human, allosteric monoclonal antibody that binds the insulin receptor with high affinity and mimics glucoregulatory, but not mitogenic, actions of insulin. Here we evaluated efficacy both single repeat s.c. administrations in reducing hyperglycemia obese cynomolgus monkeys naturally developed type 2 diabetes, model shares many features human diabetes. The data show administration at dose levels ranging from 1.5 to 10 mg/kg markedly reduced fasting hyperglycemia, peak effect...
A clear scientific and operational need exists for harmonized bioanalytical immunogenicity study reporting to facilitate communication of findings expedient review by industry health authorities. To address these key gaps provide a report structure documenting results, this cross-industry group was formed establish recommendations develop submission template agency filings. Provided here are clinical anti-drug antibody (ADA) assay results using ligand-binding technologies. This publication...
Pre-existing antibodies to viral capsids may have a negative impact on the efficacy and safety of adeno-associated virus (AAV)-based gene therapies. Total antibody (TAb) and/or cell-based transduction inhibition (TI) assays been used exclude seropositive individuals in clinical studies. Published AAV seroprevalence patient enrollment criteria regarding status lack comparability between assay formats, hindering direct cross-study comparison. To identify critical factors impacting TI detection...
Abstract Immunogenicity testing and characterization is an important part of understanding the immune response to administration a protein therapeutic. Neutralizing antibody (NAb) assays are used characterize positive anti-drug (ADA) response. Harmonization reporting NAb assay performance results enables efficient communication expedient review by industry health authorities. Herein, cross-industry group experts have harmonized recommendations provided bioanalytical report (BAR) submission...