Our Science
What is gene therapy?
Many rare genetic diseases are caused by a mutation in a single gene that prevents the proper production and function of a specific protein.
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Gene therapy is a method to treat these diseases by addressing the genetic cause. In most cases, this means delivering a healthy copy of the mutated gene to the patient's cells. Delivering these genes requires a carrier known as a vector which are often modified and engineered viruses. Once the DNA encoding the healthy gene enters the cell it can go on to be transcribed and then translated into the required protein.
Our Gene Therapy Strategy
At Gencorrex, we use leading gene therapy technology that has a proven track record in the clinic. Our focus is in making the most clinically translatable therapeutics and de-risking their development through the use of well-known materials and targeted delivery.
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AAV9 : A Proven Gene Delivery Vehicle
Our gene therapies employ the use of the AAV9 capsid to deliver genes to patient cells. AAV9 has unique properties such as the ability to cross the blood-brain barrier and a higher level of transduction in brain cells making it an ideal candidate for our target diseases. AAV9 has been approved clinically and is well-characterized in both animals and humans making it the ideal vector for translating our therapeutics to the clinic
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Targeted Gene Delivery : Intrathecal Administration
We want to ensure that our gene therapies have the best chance of success following there delivery. We use intrathecal administration to increase the amount of gene therapy vector going to the brain allowing for targeted delivery of the therapeutic transgene. We test various intrathecal routes in pre-clinical models to determine the best option for each gene therapy, ensuring that patients will get the most benefit from our treatments.
Translatability : Robust Pre-Clinical Assessments
At the heart of Gencorrex's platform is a dedication to translatable pre-clinical research by an advanced team of scientists. We design all of our pre-clinical investigations with human translation in mind. This means that we try to simulate the age, delivery method, immunosuppression protocols and evaluation outcomes as accurately as possible in our models. We also ensure that the immune profile and toxicity of each gene therapy is evaluated at every step of development before we take our therapeutics to the clinic.
