About Gene Therapy
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. A number of gene therapies are now approved by the FDA for the treatment of serious and life-threatening diseases, like spinal muscular atrophy, beta thalassemia and cerebral adrenoleukodystrophy.
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.
HOW ARE GENE THERAPIES DIFFERENT FROM TRADITIONAL MEDICINES?
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 understanding of the long-term benefits and safety profiles of gene therapies, 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 involve 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.
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
inherited retinal disease, and
There are many more diseases that could one day be treated with gene therapy.
WHAT IS GENE EDITING?
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.