Gene Therapy 101

Gene therapies are changing the future of human health as we know it. After more than 20 years of careful study, gene therapies are now making their way through the FDA approval process and to patients who need them. 

What does gene therapy mean?

Human genes are made up of our DNA, which influences the way our bodies function on the most basic level. Our genetic makeup can change due to mutations or variants in our genes. This can be hereditary (inherited from a parent) or caused by aging or environmental factors such as chemicals or radiation.

Gene therapy works by adding, removing, or changing DNA in patients. Typically, new genetic material is delivered into a patient’s cells by what’s known as a vector. Vectors typically take the form of a virus because viruses are successful at infecting our cells. These viruses are modified so they can't cause disease when used in people.

Gene therapies target the cause of the disease directly to create a potentially long-lasting change in the body.

While the scientific and medical communities continue to advance our understand of the long-term benefits and safety profiles of gene therapies, it is clear that these transformative therapies are fundamentally different from existing treatment options. There are many new challenges and unknowns involved with bringing gene therapies to patients. For example, traditional clinical trial and regulatory review frameworks may not apply. When it comes to manufacturing gene therapies, the large-scale, repeatable processes involved with traditional medicine research and development must be much more highly customized and involves smaller populations. And novel reimbursement pathways must be considered to help ensure the value of gene therapies is fully realized, which will ideally facilitate patient access.

How are gene therapies different from traditional medicines?

What diseases does gene therapy treat? 

There are currently hundreds of gene therapies in development  for the treatment of genetic diseases. Some of the initial diseases that are being studied include sickle cell disease, spinal muscular atrophy, Duchenne muscular dystrophy, hemophilia, AADC Deficiency, Friedreich's Ataxia, inherited retinal disease and beta thalassemia.

 

There are many more diseases that could one day be treated with gene therapy.

Gene editing is the manipulation of the genetic material with the aim to correct a genetic disorder.

 

Gene therapy differs in that it works to transplant normal genes into cells in place of missing or defective ones.

What is gene editing? How is this different?

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The Institute for Gene Therapies advocates for a modernized regulatory and reimbursement framework that encourages the development of transformative gene therapies and promotes patient access. 

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