Pharmaceutical preparations for treatment of rare genetic diseases; pharmaceutical preparations primarily comprising cells for treatment of rare diseases; pharmaceutical preparations and therapeutic agents for use in gene therapy for treatment of rare diseases; pharmaceutical preparations for gene therapy for the treatment of genetic diseases or disorders; pharmaceutical preparations for use in the treatment of gene therapy treatable diseases.

Pharmaceutical preparations for treatment of rare genetic diseases; pharmaceutical preparations primarily comprising cells for treatment of rare diseases; pharmaceutical preparations and therapeutic agents for use in gene therapy for treatment of rare diseases; pharmaceutical preparations for gene therapy for the treatment of genetic diseases or disorders; pharmaceutical preparations for use in the treatment of gene therapy treatable diseases.