ULTRAGENYX GENE THERAPY

Chemical and biochemical products for use in gene delivery technology, namely, nucleic acids, including coding and non-coding deoxyribonucleic acids, plasmids, vectors, promotors, enhancers and other regulatory sequences, all for in-vivo laboratory use in gene therapy

Pharmaceutical preparations for the treatment of liver, hepatological, cardiovascular, central nervous system, inflammatory, autoimmune, musculoskeletal, ophthalmologic, respiratory, and hematologic diseases and disorders

Biomedical and scientific research in the fields of genome editing, gene editing, genome engineering, gene modulation, genetic diseases, gene therapy, and cell therapy; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; conducting clinical trials for others in the field of gene therapy design and delivery; biomedical research in the field of gene therapy design and delivery; gene therapy design and delivery development of biochemical assays

Providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of genetic diseases and disorders; providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of liver, hepatological, cardiovascular, central nervous system, endocrine, inflammatory, metabolic, autoimmune, musculoskeletal, neurological, ophthalmologic, respiratory, and hematologic diseases and disorders