Applied Genetic Technologies Corporation Announces Initiation of Phase 1 Clinical Study on Safety of AGTC-0106.GAINESVILLE, Fla. -- Applied Genetic Technologies Corporation (AGTC AGTC Advanced Airport Ground Traffic Control System ), a private clinical-stage biotechnology company, announced today the initiation of a Phase 1 clinical study to test the safety of AGTC-0106, its investigational gene therapy product for patients with Alpha-1 Antitrypsin deficiency alpha-1 antitrypsin deficiency An inherited condition–frequency, ±1:10,000, characterized by low or absent production of alpha-1 antitrypsin, an enzyme which is critical to tissue remodeling Clinical The PiZZ phenotype is characterized by early-onset ("Alpha-1"). Alpha-1 is a form of congenital emphysema emphysema (ĕmfĭsē`mə), pathological or physiological enlargement or overdistention of the air sacs of the lungs. A major cause of pulmonary insufficiency in chronic cigarette smokers, emphysema is a progressive disease that commonly that causes degradation of a patient's lung tissue due to inadequate production of alpha-1-antitrypsin (AAT Alpha-1-antitrypsin (AAT) A blood component that breaks down infection-fighting enzymes such as elastase. Mentioned in: Chronic Obstructive Lung Disease ). Current treatment requires weekly intravenous infusions of a human blood derived product containing AAT. AGTC-0106 is designed to replace those infusions by providing patients with a correct version of the AAT gene in order to restore natural levels of the AAT protein. John Walsh, the President and CEO (1) (Chief Executive Officer) The highest individual in command of an organization. Typically the president of the company, the CEO reports to the Chairman of the Board. of the Alpha-1 Foundation, who himself suffers from the disease, said, "We are extremely excited about the progress being made on the development of this potential life-saving therapy for Alpha-1. Individuals with Alpha-1 have great hope that AGTC's clinical study will result in more efficacious treatment and improve the quality of life for those of us suffering from Alpha-1 related lung disease lung disease Pulmonary disease Pulmonology Any condition causing or indicating impaired lung function Types of LD Obstructive lung disease–↓ in air flow caused by a narrowing or blockage of airways–eg, asthma, emphysema, chronic bronchitis; ." AGTC-0106 is a gene therapy product which uses a modified version of a safe, naturally-occurring virus to deliver the AAT gene to patients. The virus, adeno-associated virus adeno-associated virus a replication-defective, single-stranded DNA virus classifed in the genus Dependovirus of the family Parvoviridae. They depend on help provided by coinfection with adenoviruses for their replication. Not known to cause disease. , has never been associated with illness in humans and more than 50% of the population is exposed normally to this virus. Twelve patients are expected to participate in the clinical study to test the safety of AGTC-0106 with a secondary goal of determining the minimum dose required to achieve effective levels of AAT in the blood. "We are delighted to take this next step in our development of this important new product," said Sue Washer, CEO of AGTC. "We appreciate the support of the Alpha-1 community and the Alpha-1 Foundation. We all look forward to a treatment which will provide patients with the safe and reliable levels of AAT to protect their lungs." About AGTC: AGTC is developing novel therapeutics for patients with unmet medical needs utilizing the non-pathogenic adeno-associated virus. AGTC's investors include Interwest Partners (Menlo Park, CA), Intersouth Partners (Durham, NC), MedImmune Ventures (Gaithersburg, MD), and Skyline Ventures (Palo Alto, CA). AGTC is collaborating with Genzyme to develop, manufacture and market a gene therapy product to treat Wet Age-Related Macular Degeneration Age-related macular degeneration (ARMD) Degeneration of the macula (the central part of the retina where the rods and cones are most dense) that leads to loss of central vision in people over 60. . The Company is located in Gainesville, Florida in the University of Florida's Sid Martin Biotechnology Development Incubator. |
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