New treatments, new hope
At AGTC, we use gene therapy to develop long-lasting treatments for patients with genetic disorders.
Gene therapy replaces broken genes with normal functional genes, allowing a patient’s own body to produce proteins to treat their illness. A single treatment provides long-lasting benefit – sometimes even for a lifetime - leading to a better quality of life for patients worldwide.
The innovative delivery method AGTC uses is the non-toxic adeno-associated virus (AAV), a safe virus that delivers healthy copies of the gene, replacing defective copies.
AAV is an ideal delivery method because:
- It is safe, having never been shown to cause disease.
- It is effective and provides long-lasting benefit.
- Its production is fully scalable and does not require animal-derived products.
- It has been approved for use in human clinical trials by U.S. and European regulatory agencies.