The group's principal activity is to develop pharmaceutical products for serious and chronic hematological and neurological diseases. The group has developed proprietary dna-based drug delivery technologies including gene delivery platform based on adeno-associated virus (aav) vectors. The dna drug-based delivery technologies are used to develop products designed to treat hematological and neurological diseases that are difficult to treat using conventional pharmaceutical drugs. The group's advanced product development programs are designed to utilize aav vector technology to deliver dna into the cells of patients in order to generate the expression of therapeutic proteins to treat hemophilia and Parkinson's disease.
