Nucleic acid sequences and chemical reagents for medical purposes; pharmaceutical preparations for the treatment of genetic diseases and disorders; genetic medicines for the treatment of diseases and disorders with known genetic cause or component and disease states resulting from gene expression or genetic sequence; nucleic acid therapeutic agents for the treatment of Huntington's disease, Duchenne muscular dystrophy, and alpha-1 antitrypsin deficiency; pharmaceutical preparations for the prevention, diagnosis, and treatment of metabolic, cardiometabolic, endocrine, musculoskeletal, cardiovascular, cardiopulmonary, renal, genitourinary, oncological, hepatological, ophthalmic, respiratory, neurological, neurodegenerative, gastrointestinal, hormonal, inflammatory, dermatological, psychiatric, autoimmune and immune system related diseases and disorders.;
Research and development in the fields of pharmaceuticals, genetics, therapeutics, and drug formulation and delivery; scientific research; scientific research in the fields of pharmaceuticals, medicine, and nucleic acid therapeutics; research, development, and testing services in the field of nucleic acid therapeutics for medical and scientific applications; medical research; laboratory research services relating to pharmaceuticals.;