Accelerating Therapeutic DiscoveryTM

News From The Alpha-1 Project

20

MIAMI, FL and BOSTON, MA, April 19, 2013 – The Alpha-1 Project (TAP) today announced a $150,000 commission to Darrell Kotton, MD, to expand development of induced pluripotent stem cell (iPSC) lines created from tissue donated by patients with Alpha-1 Antitrypsin Deficiency (Alpha-1). Kotton is Professor of Medicine and Co-director of both The Alpha-1 Center and the Center for Regenerative Medicine at Boston University School of Medicine and Boston Medical Center.

“We are happy to announce The Alpha-1 Project’s investment in providing tools for researchers and industry,” said Jean-Marc Quach, Executive Director of TAP. “This commission signals our intent to direct research and resources aimed at speeding the development of new therapies for Alpha-1. I must emphasize that additional funds will need to be raised from the community if Dr. Kotton is to meet the goal of completing 20 stem cell lines over the next three years.”

Kotton plans to make the Alpha-1 iPSC lines available to all researchers interested in studying stem cell technology and possible therapies for Alpha-1.

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26

MIAMI, FL and GAINESVILLE, FL (March 26, 2013) - The Alpha-1 Project (TAP) today announced its first investment in a potential new treatment for Alpha-1 Antitrypsin Deficiency (Alpha-1), the most common known genetic risk factor for emphysema. TAP is partially funding further trials of a gene therapy treatment being developed by Applied Genetic Technologies Corporation (AGTC).

Alpha-1 is a genetic disorder characterized by low levels of or absent alpha-1 antitrypsin protein in the blood. Alpha-1 patients are at risk for severe lung disease as adults and liver disease at any age. Up to 3% of all people diagnosed with COPD may have undetected Alpha-1. The condition can lead to severe disability and reduced life expectancy.

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13

MIAMI -- Jean-Marc D. Quach, a longtime leader in healthcare management and marketing, has been named executive director of The Alpha-1 Project, a subsidiary of the Alpha-1 Foundation.

Quach, of Englewood Cliffs, NJ, is not a newcomer to the world of Alpha-1 Antitrypsin Deficiency. From 1999-2004, he was vice president and general manager of Express Scripts Specialty Distribution Services, where he worked with AlphaNet and Bayer to create a "direct distribution" model for Prolastin®, then the only Alpha-1 augmentation treatment available. The business model is still used for orphan and other high-risk or shortage-prone drug products.

“Jean-Marc, with his record of leadership and innovation, is the perfect match for the Foundation’s initiative to speed the development of new therapies for Alpha-1,” said John W. Walsh, Foundation president and CEO.

The Alpha-1 Project (TAP) is a for-profit, wholly owned subsidiary of the not-for-profit Alpha-1 Foundation. “TAP is a venture philanthropy company,” said Marcia F. Ritchie, vice president and chief operating officer of both TAP and the Alpha-1 Foundation. “The goal is to accelerate the discovery, development and commercialization of treatments and drug therapies for those with Alpha-1,” Ritchie said.

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