September 2013

Orphan Disease Center Seeks to Expedite Orphan Disease Treatments

Orphan_Disease_Center

Orphan diseases represent a collection of disorders that afflict less than 200,000 individuals for any single disease type, yet there are more than 7,000 distinct orphan diseases. In the aggregate, over 25 million people in the United States suffer substantial morbidity and mortality from orphan diseases. Despite this huge number, research in most disease types has lagged far behind other major areas due to a combination of technological and funding limitations.

Recognizing these challenges, the Penn Center for Orphan Disease Research and Therapy (CODRT) was formed with the mission of facilitating and expediting the development of novel therapies for orphan diseases. The Center will achieve this mission through promoting development of novel therapeutic strategies and translating these into the clinic, building on partnerships among investigators, academic institutions, industry, and federal and private funding agencies.

The Penn Center for Orphan Disease Research and Therapy invites faculty members at Penn and CHOP who conduct research in rare diseases to join the Center. The CORDT will provide resources and funding opportunities to Center members with demonstrated expertise in rare disease research and/or therapy development. Trainees interested in rare disease research can join as Associate members. Center membership will provide:

  • Connections between researchers and clinicians at Penn/CHOP and other intuitions who are working on orphan diseases
  • Access to drug repurposing screens as well as small molecule screens
  • Sources of funding for research on orphan diseases
  • Access to small and large pharmaceutical companies to partner in the development of new therapeutics for orphan diseases
  • Access to animal models of orphan diseases

For membership information, please click here.

Research Funding and Events

The Center for Orphan Disease Research and Therapy has also announced a Request for Applications to support research on the development of improved therapies for patients with syndromes due to MPS I including Hurler, Hurler-Scheie, and Scheie. Particular emphasis will be placed on treatments that improve aspects of the disease that are not adequately treated by enzyme replacement therapy such as pathology in the CNS, skeletal system, eye, and heart, among others.

To learn more about research funding opportunities through the CORDT, see the Center site.

The Center is also set to host a number of upcoming events. On Thursday, November 21, the Center will hold its first annual retreat, “Charting Paths Forward in Rare Diseases.” The day will start with an overview of the center followed by presentations on Center partners and opportunities in rare disease research, drug development, and biomarker discovery.

Guest speakers will include John F. Crowley, JD, MBA, chairman and CEO of Amicus Therapeutics; Larry Gold, PhD, chairman, founder, and CEO of SomaLogic, Inc.; Stephen C. Groft, PharmD, director of the NIH Office of Rare Diseases; John C. McKew, PhD, chief of the  Therapeutics Development Branch at the NIH Center for Translational Therapeutics; Bill Leinweber, president and CEO of the National Disease Research Interchange; and Dave C Schultz, PhD, facility director of the Wistar Institute’s Molecular Screening Facility.

And in the Spring of 2014, the Center will sponsor two events to focus awareness on orphan diseases and research. On Friday, May 2, 2014 we will hold the First Annual Symposium on Orphan Diseases followed by a Cycling Event for Rare Diseases on Saturday May 3. Beginning and ending on the University of Pennsylvania campus, the cycling event is designed to raise money and awareness for international rare disease research.

For information and registration, please click here.

To read more about the Penn Center for Orphan Disease Research and Therapy, please visit the Center site.

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