These facilities can be used to produce commercial scale quantities of AAV
but could also be used to manufacture other biologics, including cell therapeutics, recombinant proteins and monoclonal antibodies.
patent #6,936,466, titled "Transcriptionally Activated AAV
Inverted Terminal Repeats (ITRs) for Use with Recombinant AAV
Vectors" covers the use of small regulatory control elements in conjunction with sequences within the AAV
genome to drive gene expression.
Cell Genesys is currently evaluating liver-directed AAV
gene therapy in animal studies of both types of hemophilia -- hemophilia A and B -- which result from genetic deficiencies in blood clotting factors VIII and IX, respectively.
Importantly, this data helps us to further understand the potential of this lead product opportunity, in particular as we seek additional product opportunities in therapeutic areas of interest that are complementary to our program in inflammatory arthritis and as we pursue ways to further leverage our investment in AAV
manufacturing and scale up, through strategic collaborations.
Our commercial activities are built on a solid foundation of AAV
biology, and we are pleased to share the advances that we and our collaborators have made in this area with the gene therapy community.
This study provides new information on the relationship between the number of AAV
vectors introduced into a given cell and the cellular localization of those particles.
These presentations underscore the potential therapeutic utility of AAV
GeneThera's PURIVAX(TM), is a multi-resin anion exchange chromatography system that dramatically improves biological purity and viral titer of recombinant Adenovirus and AAV
Our presence speaks to the breadth of our scientific expertise and capabilities in drug development using AAV
, which are integral to our current and future product development opportunities and partnerships.
The Company is also pursuing opportunities to further leverage its investment in AAV
manufacturing and scale up, through additional product collaborations, or through strategic relationships with contract manufacturers.
The patent describes AAV
vectors that utilize a small, human DNA sequence, P1.
patent #6,995,006 is titled, "Methods for generating high titer helper-free preparation of released recombinant AAV