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.

Our technology

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.

Contact Us

Alachua – Headquarters
14193 NW 119th Terrace, Suite #10
Alachua, Florida 32615
One Kendall Square, 1st Floor, Suite 14102
Cambridge, MA 02139

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