Mar 09, 2018
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Duties: Orphan diseases represent a collection of disorders that afflict fewer than 200,000 individuals for any single disease type, yet there are more than 7,000 distinct orphan diseases. The Orphan Disease Center (ODC) in the Perelman School of Medicine was founded in 2011 to promote and fund research in rare diseases globally, beyond Penn, making a difference for these under-served patient populations. A unique aspect of ODC is that it is a 'Center without walls' which sources and contributes to developing transformative therapies through the use of platform technologies and of research strategies that can be deployed across multiple rare disease programs. The main feature is a pilot grant program which provides $8M annually to over 35 global institutions.

We are currently seeking a new Director of Therapeutic Development to over see the Program of Excellence focused on Lysosomal Storage Disorders.

Reporting to the Executive Director of the Orphan Disease Center (ODC) and supporting a Program of Excellence, your goal will be to provide international leadership in establishing research agenda in rare disease that are a high priority of the ODC. You will be developing strong partnerships with key stakeholders, including patient advocacy groups, partnering academic institutions and key opinion leaders in relevant diseases by organizing international scientific symposiums related to diseases of interest to the program of excellence, partnering with ODC staff to organize these events. You will have the primary responsibility for creating and executing a strategic plan, to establish clinical development for novel therapeutics. You will seek advisement from all relevant stakeholders, including patient advocacy groups, ODC leadership, and external advisors in initial development of this plan. In executing this plan, you will undoubtedly be required to have extensive interactions with biopharmaceutical industry partners, knowledgeable on the development of novel therapeutics and will require you to travel to biomedical conferences. You will establish working groups within each disease to address research and clinical practice gaps, and focus on filling those gaps to further the development of effective treatments.

You will be critical in the success of the program of excellence, providing careful oversight of the research conducted by grant recipients, assuring progress and progression of science towards a biopharmaceutical partnership. It will be essential that you have experience in biopharm to assure these critical links occur between the grant recipients and corporate partners.

Joining the ODC offers you with unique opportunity to contribute to revolutionizing the practice of medicine for these under-served patient groups.

Qualifications: * PhD with a background in Lysosomal Storage Disorders and 1 to 5 years of drug development experience required; MD preferred
* Experience writing clinical development plans and clinical trials required
* Experience with patient registries/natural history studies preferred
* Preferred experience in rare diseases
* Self starter but also a strong desire to contribute and work collaboratively to achieve goals
* Success in roles requiring execution of multiple tasks while responding to multiple priorities
* Highly functioning, detail-oriented, and analytical candidate who can think strategically
* Excellent oral/written communication skills required

Reference Number: 40-28026

Salary Grade: 030

Employment Type: Exempt

Org: OD-Center for Orphan Disease Research and Therapy

Special Requirements:

Job Family: B-Executive/Managerial Administration

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