What is gene therapy for animals?
Our new generation of therapies works by using a vector delivered by a simple intramuscular injection. A vector is the shell from a benign virus enclosing genetic material (DNA) which enters the cells in the pet patient and transmits the ability to produce a therapeutic protein.
The genetic material is specific for each disease and will direct the patient’s body to produce a therapeutic protein which is expected to treat the targeted chronic disease. Vectors are often made from viruses that have been stripped of their disease-causing features. Scout Bio’s technology partner, University of Pennsylvania’s Gene Therapy Program, has been a leader in the development of gene therapy vector technologies for nearly three decades. It has been at the forefront of this area of human medicine and now provides Scout Bio with world-leading capabilities in adeno-associated viral (AAV) vector design, optimization and early clinical-scale manufacturing for animal health.
Adeno-associated viruses are a very common family of viruses that do not cause disease. AAVs have been specifically engineered to ensure they are stable and cannot replicate by themselves, making them highly suitable vectors. They do not alter the patient’s ‘native’ genome but instead introduce the instruction for the build of the therapeutic protein as a separate DNA structure outside of the patient’s cell’s chromosomes. AAV vectors are the choice for many gene therapy applications, including the first FDA-approved human gene therapies for genetic diseases.
The laboratory, or pre-clinical, research focuses on isolating the gene which is responsible for instructing a cell to make the desired therapeutic protein and then working to optimize the incorporation of that protein into the viral vector. Once the final formulation has been created and tested for safety, then pet patients suffering from the chronic condition being studied can receive an injection of the AAV vector which contains the therapeutic gene. The target cells within the pet will then begin to produce the therapeutic protein for the pet within the pet’s own body.
Our 17 year old kitty, was sadly diagnosed with fairly progressed kidney disease. Miraculously I ran into this Research Study for Cats with Kidney Disease. I pursued seeing if Sushi could be accepted into the program. He met their criteria and was accepted. This truly was a miracle as far as I am concerned, as the treatment he received renewed his life. His kidney values improved, and thus his quality of life. Being part of the study gave us more time to have Sushi part of our life. The Doctor and staff at VCA were all attentive and caring. We are very much grateful for all the treatment and care. I would highly recommend this study or treatment to anyone pursuing help for their cat with kidney disease. With no hope in sight, it truly turned his condition into something more workable and hopefully. Thank you to everyone involved in Sushi’s care. We are grateful.
I wanted to send a heartfelt thank you for allowing him in the study for CDK anemia. We are so happy to participate and I joke with him that he’s “a man of science”.
Hoping you are able to help many others with the results of your study. We are additionally so grateful for the additional support you have shown us for Ryan’s health. Wanted to share some love back and make sure all those involved in your company heard how grateful we truly are!
The day I found your study was like a light in a dark tunnel. Our two littermate kitties, Misty and Mischief, have both participated in Scout Bio’s study. Our girl Misty was really sick. You gave us some very valuable time with her. Misty’s brother has recently joined the study too. He was able to join at an earlier point in his kidney disease and is doing really well. Words can’t express how truly grateful I really am.