GeneQuine develops drugs that are based on gene therapy. The concept of gene therapy is to introduce genetic material into the patient's own cells in the body. The cells produce then a therapeutic protein according to the template that the introduced genetic material (DNA) provides. Therefore, the patient's body itself produces the therapeutic agent. One of the major advantages of this approach is that the therapeutic protein can be produced over a long period of time as opposed to conventional drugs, which have to be administered frequently because they are metabolized by the body. Gene therapy has proven to be safe and efficacious in a number of diseases including inherited disorders such as lipoprotein lipase disorder, hemophilia and leber's congenital amaurosis as well as cancer.
GeneQuine's product candidates are next generation gene transfer vehicles. These gene therapy vectors are highly efficient in entering joint cells and are able to confer multi-year gene expression. An excellent safety profile of these vectors has been demonstrated in several species.
GeneQuine is focused on the development of gene therapy agents for the treatment of osteoarthritis. Osteoarthritis is a degenerative joint disorder characterized by cartilage loss and inflammation. Patients affected by osteoarthritis experience joint pain as well as swelling and stiffness of the joints leading to limited joint functionality and impaired mobility. Osteoarthritis is the most common joint disorder with approximately 80 million people in the EU and the USA being currently affected. Moreover, osteoarthritis is also a major health problem in companion animals such as horses and dogs with approximately 1 million and 18 million patients respectively.
GQ-201 is GeneQuine's gene therapy-based lead product for the treatment of osteoarthritis in horses. The product candidates GQ-202 and GQ-203 are the respective analogues for osteoarthritis in dogs and humans. GeneQuine is also developing the gene therapy product candidates GQ-301 and GQ-303 for musculoskeletal diseases in horses and humans. Compared to the 200 series, these product candidates express a protein with a different pharmacological profile and mechanism of action.
The development statuses of GeneQuine's product candidates are shown below:
|Experimental Studies||Proof-of-Concept in Target Species||Toxicology, Pharmacokinetics, Pharmacodynamics||Clinical Field Studies||Market Authorization|
|Preclinical Studies||Clinical Phase I||Clinical Phase II||Clinical Phase III||Market Authorization|
GeneQuine is currently developing a robust and scalable GMP manufacturing process for its product candidates. Since GeneQuine's drug candidates are all based on the same gene therapy vector type, the GMP manufacturing process is being developed as a platform process applicable to all of the current and furture pipeline products.