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AGTC Receives FDA Orphan Drug Status for Alpha One Antitrypsin Deficiency Treatment
Gainesville, Florida
January 24, 2003
Applied Genetic Technologies Corporation, AGTC, a drug research company developing novel human therapeutics, announces that U.S. regulators have granted its treatment for Alpha One Antitrypsin Deficiency (A1AD) “orphan-drug” status.
Orphan-drug status, aimed at encouraging research into rare illnesses, grants seven years of market exclusivity to a product after market launch, allows the firm to apply for research funding and tax credits, and waives the normal application fees for new product licensure.
A1AD is a hereditary defect that causes early on-set emphysema in adults. Patients experience significant loss of daily function and shortened life spans. A1AD is the most common potentially lethal hereditary disease of American and Northern European adults affecting approximately one in 2500 white Caucasians. There are currently 82,000 symptomatic patients in the US and Europe and this number is expected to grow at 4% per year.
AGTC’s treatment for A1AD utilizes its Adeno-Associated Virus technology to deliver the normal human gene to patients so their body can produce the missing protein. It is expected that all patients would be treated only once stabilizing deterioration of lung function in those chronically affected. For newly diagnosed patients, treatment would presumably lead to prevention of lung abnormalities and symptom-free, normal life spans.
Extensive pre-clinical work has been completed on the Company’s treatment for A1AD and AGTC is currently raising a Series B venture investment of $12 million in order to fund Human Clinical Trials and scale-up its production process to support market launch.
In September 2001 AGTC received Series A venture funding from PrimBio Tech, SAS, of Paris, France. The $6 million investment funded the Company’s work to complete pre-clinical toxicology studies, initiate Phase I Human Clinical Trials, and complete the initial development of its proprietary high-efficiency production method.
Contact:
Susan B. Washer, CEO
386-462-2204 or swasher@biotech.ufl.org
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