Course

Target Identification for Gene Therapy

Self-paced
0.75 credits

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Full course description

The steps and requirements for developing gene therapies for Huntington’s Disease (HD) will be discussed. Current HD therapies being studied and those already approved will be covered.

Learner Objectives:

a.     Describe the mechanism of action for a gene therapy.  

b.     Understand the need for different modalities of treatments for genetic disorders.

c.      Identify the necessary components to making a gene therapy “drug”.

d.     Explain the specifications considered for a gene therapy’s target product profile.

e.     Examine the causes and treatments for Huntington’s Disease

f.       Explore how pharmacodynamic biomarkers measure the effectiveness of gene therapies for Huntington’s Disease

Course Topics

1.     FDA Description of Gene Therapy

2.     Modalities of Treatment: Small Molecule vs. Gene Therapy

3.     Elements of a Gene Therapy

4.     Target Product Profile: Specifications for Safety and Efficacy

5.     Huntington’s Disease

6.     Huntington-Lowering Therapies

7.     Development of Pharmacodynamic Biomarkers

8.     Huntington’s Disease Gene Therapy Portfolio

Course Speaker:

Dr. Robert Pacifici is the Chief Scientific Officer of CHDI Foundation, a private, not-for-profit research organization that works with an international network of scientists to accelerate therapeutics development for Huntington’s disease. Previously he was the Site Director and Chief Scientific Officer at the Research Triangle Park Laboratories of Eli Lilly and Company. There he oversaw the company's global screening and quantitative-biology efforts. Prior to joining Lilly, Pacifici was Vice President of Discovery Technologies at Xencor, a privately held biotechnology company that applied rational design principles to the development of protein therapeutics. At Amgen for nearly ten years, Pacifici held positions of increasing responsibilities including leadership for their automation, high throughput screening, and information technologies groups.

He received a BS in Biochemistry from the University of Massachusetts, Amherst, and a PhD in Biochemistry from the University of Southern California.

Outside of CHDI, Robert currently participates in several external boards and advisory committees including:  An adjunct appointment at the University of Southern California's (USC) Department of Molecular Pharmacology and Toxicology; USC Board of Supervisors of the International Center for Regulatory Science;  Council member for National Institutes of Health/National Institute of Neurological Disorders and Stroke (NIH/NINDS) from 2011 till 2014; Panel of Experts for National Center for Advancing Translational Sciences (NCATS) at NIH, in the NIH Center for Translational Therapeutics (NCTT). This division is home to two programs that seek to advance promising therapeutic agents through late-stage preclinical development: Therapeutics for Rare and Neglected Diseases (TRND) and Bridging Interventional Development Gaps (BrIDGs); Chair, Working Group for the NINDS Anticonvulsant Screening Program; Member, Spinal Muscular Atrophy Foundation Scientific Advisory Board; Member, DART Rx, Scientific Advisory Board.

Robert has previously served on: Chair, NIH/NINDS Spinal Muscular Atrophy Project's Scientific Steering Committee; Advisor, Marigold Foundation for Myotonic Dystrophy; Advisor, Cooperative International Neuromuscular Group (CINRG) the clinical research arm of the Duchenne Muscular Dystrophy Research Center (DMDRC) and the Center for Genetic Medicine Research at the Children’s National Medical Center (CNMC); Member, Science Advisory Board for Edison Pharmaceuticals; Member, TREAT ALS Steering Committee; Member, Pathogenesis of Facioscapulohumeral Muscular Dystrophy advisory board. 

He also serves in the non-scientific capacity of Board Member with the Asia America Symphony Association. Robert, his wife Eunjoo, and his two children Sarina and Noah, live in Palos Verdes Estates, California. Robert is an avid amateur road-cyclist and a classic BMW enthusiast. 

Learner Level: Intermediate/Advanced

Audience:

This symposium is designed for Clinical Research Coordinators, Clinical Research Nurses, Project Managers, Monitors, Regulatory, Medical and Clinical Affairs Professionals, or junior faculty with three or more years of experience seeking to further develop clinical research skills and principles. Clinical research professionals should have responsibilities that include clinical trial start up planning, mentoring and complex issues involving site management, study management, and sponsor / CRO challenges.

Learner Disclosures:

  • Accreditation Status: Emory Nursing Professional Development Center (ENPDC) is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s (ANCC) Commission on Accreditation.
  • Financial Relationships: ENPDC has evaluated everyone who has the ability to control content of this activity (planning committee members, subject matter experts, presenters) and found that no relevant financial relationships exist.
  • Awarding Contact Hours: To obtain contact hours participants must
    • Review the entire activity
    • Pass the quiz with 100% of questions answered correctly
    • Complete the evaluation at the end
    • Certificates will be distributed at the end of the evaluation.
  • Enduring Materials Dates:
    • Start Date: 05/01/2024
    • Expiration Date: 05/01/2027

Need Help with Registration? Please contact us at ene@emory.edu or 404-727-9208.

Program Information:

This is the fifth course in a five (5) course program, Innovations in Regenerative Medicine Products.

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