Gene Therapy

GeneQuine develops drugs that are based on gene therapy. The concept of gene therapy is to introduce genetic material into the patient's cells in the body. The cells then produce 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 or eliminated 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.

Osteoarthritis

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.

Gene Therapy for Osteoarthritis

Sufficiently high drug concentrations and long-term bioavailability within the joint are two of the major challenges for efficient treatment of osteoarthritis. Both attributes are requisite for sustained efficacy and a disease-modifying effect.

Medicinal products currently used to treat osteoarthritis as well the investigational drug candidates used in clinical studies are typically chemical or biological substances administered either systemically or by a direct injection into the joint. However, if administered systemically, only small portions of the administered drug reach the joint space. When injected directly into the joint, most active compounds are rapidly cleared from the joint by physiological mechanisms resulting in short dwell times in the joint. Frequently repeated joint injections, however, are not feasible in the long-term treatment of a chronic joint diseases such as osteoarthritis.

A single joint injection of a gene therapy drug is able to establish local long-term production of a therapeutic protein by the joint cells. This way, optimal drug concentrations and long-term bioavailability within the affected joint can be achieved.