Orphan Indications

Hope for rare disease patients.

A rare or orphan disease is any disease that affects a small percentage of the population. Most of these are genetic, and are present throughout the person's entire life, even if symptoms do not immediately appear.

In the U.S., the Orphan Drug Act of 1983 – which was written to encourage research into rare diseases – defines an orphan disease as any disease or condition that affects less than 200,000 persons in the United States, or about 1 in 1,500 people. In Europe, rare diseases are defined as life-threatening or chronically debilitating diseases which are of such low prevalence that special combined efforts are needed to address them, generally meaning fewer than 1 in 2,000 people.

AGTC is currently developing products for three orphan diseases – Alpha-1 Antitrypsin Deficiency (Alpha 1), X-linked Retinoschisis (XLRS) and Achromatopsia (ACHM).  The U.S. Food and Drug Administration and the European Medicines Agency have granted orphan disease designation to AGTC for each three of the diseases, which means these regulatory agencies will provide a variety of special types of advice and assistance to AGTC as we develop our gene therapy products for these diseases.

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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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