Pharmaceutical, medical and veterinary preparations for prevention, amelioration and treatment of disorders, namely blood disorders, lysosomal storage disorders, metabolic disorders, namely diabetes, Hurler syndrome, Niemann-Pick disease, Tay-Sachs disease, Gaucher disease, Fabry disease, Krabbe disease, Phenylketonuria, Friedreich ataxia, Zellweger syndrome, Adrenoleukodystrophy, Wilson disease and Hemochromatosis, inflammatory disorders, namely C3 glomerulopathy, IgA nephropathy, lupus nephritis, systemic lupus erythematosus, membranous nephropathy, membranoproliferative glomerulonephritis, paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, autoimmune haemolytic anemia, ANCA-associated vasculitis, peritonitis, age-related macular degeneration, diabetic retinopathy, dense deposit disease, autoimmune arthritis, rheumatoid arthritis, atherosclerosis, chronic cardiovascular disease, Alzheimer's disease, systemic vasculitis, Guillain-Barre syndrome, and Henoch-Schonlein purpura and immune system disorders, autoimmune disorders, hepatological disorders, peripheral nervous system disorders and central nervous system disorders, namely diseases of the brain, diseases of the spinal cord, Gaucher disease type 2, Gaucher disease type 3, neurodegenerative diseases namely dementia, Parkinson's disease, Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS), ocular and ophthalmological disorders, muscular disorders, musculoskeletal disorders, bone disorders, cardiovascular and vascular disorders, endocrine disorders, gastrointestinal disorders, dermatological disorders and diseases namely, dermatitis, eczema and psoriasis, acne, rosacea, ichthyosis, vitiligo, urticaria and alopecia areata, respiratory disorders, urological disorders, cancers and infectious diseases; pharmaceutical, medical and clinical preparations for therapeutic and prophylactic purposes in connection with blood disorders, lysosomal storage disorders, metabolic disorders, namely diabetes, Hurler syndrome, Niemann-Pick disease, Tay-Sachs disease, Gaucher disease, Fabry disease, Krabbe disease, Phenylketonuria, Friedreich ataxia, Zellweger syndrome, Adrenoleukodystrophy, Wilson disease and Hemochromatosis, inflammatory disorders, namely C3 glomerulopathy, IgA nephropathy, lupus nephritis, systemic lupus erythematosus, membranous nephropathy, membranoproliferative glomerulonephritis, paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, autoimmune haemolytic anemia, ANCA-associated vasculitis, peritonitis, agerelated macular degeneration, diabetic retinopathy, dense deposit disease, autoimmune arthritis, rheumatoid arthritis, atherosclerosis, chronic cardiovascular disease, Alzheimer's disease, systemic vasculitis, Guillain- Barre syndrome, and Henoch-Schonlein purpura and immune system disorders, autoimmune disorders, hepatological disorders, peripheral nervous system disorders and central nervous system disorders, namely diseases of the brain, diseases of the spinal cord, Gaucher disease type 2, Gaucher disease type 3, neurodegenerative diseases namely dementia, Parkinson's disease, Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS), ocular and ophthalmological disorders, muscular disorders, musculoskeletal disorders, bone disorders, cardiovascular and vascular disorders, endocrine disorders, gastrointestinal disorders, dermatological disorders and diseases namely, dermatitis, eczema and psoriasis, acne, rosacea, ichthyosis, vitiligo, urticaria and alopecia areata, respiratory disorders, urological disorders, cancers and infectious diseases; pharmaceutical, medical and clinical preparations for use in treatment, amelioration and prophylaxis of genetic and inherited and non-infectious diseases and disorders, namely blood disorders, lysosomal storage disorders, metabolic disorders, namely diabetes, Hurler syndrome, Niemann-Pick disease, Tay-Sachs disease, Gaucher disease, Fabry disease, Krabbe disease, Phenylketonuria, Friedreich ataxia, Zellweger syndrome, Adrenoleukodystrophy, Wilson disease and Hemochromatosis, inflammatory disorders, namely C3 glomerulopathy, IgA nephropathy, lupus nephritis, systemic lupus erythematosus, membranous nephropathy, membranoproliferative glomerulonephritis, paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, autoimmune haemolytic anemia, ANCA-associated vasculitis, peritonitis, age-related macular degeneration, diabetic retinopathy, dense deposit disease, autoimmune arthritis, rheumatoid arthritis, atherosclerosis, chronic cardiovascular disease, Alzheimer's disease, systemic vasculitis, Guillain-Barre syndrome, and Henoch-Schonlein purpura and immune system disorders, autoimmune disorders, hepatological disorders, peripheral nervous system disorders and central nervous system disorders, namely diseases of the brain, diseases of the spinal cord, Gaucher disease type 2, Gaucher disease type 3, neurodegenerative diseases namely dementia, Parkinson's disease, Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS), ocular and ophthalmological disorders, muscular disorders, musculoskeletal disorders, bone disorders, cardiovascular and vascular disorders, endocrine disorders, gastrointestinal disorders, dermatological disorders and diseases namely, dermatitis, eczema and psoriasis, acne, rosacea, ichthyosis, vitiligo, urticaria and alopecia areata, respiratory disorders, urological disorders and cancers; pharmaceutical, medical and clinical preparations for treatment, amelioration and prophylaxis of chronic and systemic disease, namely blood disorders, lysosomal storage disorders, metabolic disorders, namely diabetes, Hurler syndrome, Niemann-Pick disease, Tay-Sachs disease, Gaucher disease, Fabry disease, Krabbe disease, Phenylketonuria, Friedreich ataxia, Zellweger syndrome, Adrenoleukodystrophy, Wilson disease and Hemochromatosis, inflammatory disorders, namely C3 glomerulopathy, IgA nephropathy, lupus nephritis, systemic lupus erythematosus, membranous nephropathy, membranoproliferative glomerulonephritis, paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, autoimmune haemolytic anemia, ANCA-associated vasculitis, peritonitis, age-related macular degeneration, diabetic retinopathy, dense deposit disease, autoimmune arthritis, rheumatoid arthritis, atherosclerosis, chronic cardiovascular disease, Alzheimer's disease, systemic vasculitis, Guillain-Barre syndrome, and Henoch-Schonlein purpura and immune system disorders, autoimmune disorders, hepatological disorders, peripheral nervous system disorders and central nervous system disorders, namely diseases of the brain, diseases of the spinal cord, Gaucher disease type 2, Gaucher disease type 3, neurodegenerative diseases namely dementia, Parkinson's disease, Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS), ocular and ophthalmological disorders, muscular disorders, musculoskeletal disorders, bone disorders, cardiovascular and vascular disorders, endocrine disorders, gastrointestinal disorders, dermatological disorders and diseases namely, dermatitis, eczema and psoriasis, acne, rosacea, ichthyosis, vitiligo, urticaria and alopecia areata, respiratory disorders, urological disorders and cancers; pharmaceutical, medical and clinical preparations for treatment, amelioration and prophylaxis of blood disorders, lysosomal storage disorders, metabolic disorders, namely diabetes, Hurler syndrome, Niemann-Pick disease, Tay-Sachs disease, Gaucher disease, Fabry disease, Krabbe disease, Phenylketonuria, Friedreich ataxia, Zellweger syndrome, Adrenoleukodystrophy, Wilson disease and Hemochromatosis, inflammatory disorders, namely C3 glomerulopathy, IgA nephropathy, lupus nephritis, systemic lupus erythematosus, membranous nephropathy, membranoproliferative glomerulonephritis, paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, autoimmune haemolytic anemia, ANCA-associated vasculitis, peritonitis, age-related macular degeneration, diabetic retinopathy, dense deposit disease, autoimmune arthritis, rheumatoid arthritis, atherosclerosis, chronic cardiovascular disease, Alzheimer's disease, systemic vasculitis, Guillain-Barre syndrome, and Henoch-Schonlein purpura and immune system disorders, hepatological disorders, peripheral nervous system disorders and central nervous system disorders, namely diseases of the brain, diseases of the spinal cord, Gaucher disease type 2, Gaucher disease type 3, neurodegenerative diseases namely dementia, Parkinson's disease, Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS); pharmaceutical, medical and clinical preparations for use in gene therapy; pharmaceutical, medical and clinical preparations for liver-targeted viral gene therapy; genetic medicines, namely chemicals, compounds and compositions used to cure, halt and prevent genetic-based disease and illness and to ease symptoms and to help in the diagnosis of geneticbased diseases and illnesses, by modifying gene expression to correct and compensate for an abnormal phenotype, namely for use for prevention, amelioration and treatment of disorders, namely blood disorders, lysosomal storage disorders, metabolic disorders, namely diabetes, Hurler syndrome, Niemann-Pick disease, Tay-Sachs disease, Gaucher disease, Fabry disease, Krabbe disease, Phenylketonuria, Friedreich ataxia, Zellweger syndrome, Adrenoleukodystrophy, Wilson disease and Hemochromatosis, inflammatory disorders, namely C3 glomerulopathy, IgA nephropathy, lupus nephritis, systemic lupus erythematosus, membranous nephropathy, membranoproliferative glomerulonephritis, paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, autoimmune haemolytic anemia, ANCA-associated vasculitis, peritonitis, agerelated macular degeneration, diabetic retinopathy, dense deposit disease, autoimmune arthritis, rheumatoid arthritis, atherosclerosis, chronic cardiovascular disease, Alzheimer's disease, systemic vasculitis, Guillain- Barre syndrome, and Henoch-Schonlein purpura and immune system disorders, autoimmune disorders, hepatological disorders, peripheral nervous system disorders and central nervous system disorders, namely diseases of the brain, diseases of the spinal cord, Gaucher disease type 2, Gaucher disease type 3, neurodegenerative diseases namely dementia, Parkinson's disease, Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS) , ocular and ophthalmological disorders, muscular disorders, musculoskeletal disorders, bone disorders, cardiovascular and vascular disorders, endocrine disorders, gastrointestinal disorders, dermatological disorders and diseases namely, dermatitis, eczema and psoriasis, acne, rosacea, ichthyosis, vitiligo, urticaria and alopecia areata, respiratory disorders, urological disorders, cancers and infectious disease; gene therapy preparations; preparations for therapeutic delivery of nucleic acids; viral and nucleic acid vector preparations, namely gene-based biological preparations and medical reagents for use in gene delivery, gene transfer, gene regulation and gene modulation for therapeutic, prophylactic and medical purposes; adeno-associated viral gene therapy preparations.

Pharmaceutical manufacturing and processing to the order and specification of others; biotechnological and biopharmaceutical manufacturing and processing to the order and specification of others; viral vector production to the order and specification of others; gene therapy manufacturing to the order and specification of others; pharmaceutical formulation to the order and specification of others.