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Tuesday, June 25 • 2:00pm - 3:15pm
#263: Drug Development for Ocular Disease, New Therapies, Regulations, and Patient Perspectives

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-614-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session will highlight recent advances and review challenges and opportunities in rare disease ocular therapies. New therapeutic approaches have emerged including promising gene and cell-based therapies in retinal diseases. Relevant case studies and special considerations will be discussed. We will explore the regulatory and scientific issues with experts to provide insights from the FDA and different sponsors.

Learning Objectives

Identify recent advances in in ocular drug development therapies; Discuss the existing regulatory framework; Describe challenges and opportunities in ocular rare disease drug development highlighting what has been done and what might be done in the future.


Nita Ichhpurani, PMP


Gene Therapy for Inherited Retinal Diseases
Melissa Shiao Hui Liew, MD

FDA/CBER Perspective on the Development of Gene Therapy Products for Retinal Disorders
Wei Liang, PhD

Changing What it Means to be Blind: We're All in This Together
Kristin Smedley

avatar for Wei Liang

Wei Liang

Pharmacologist, OTAT, CBER, FDA
Dr. Liang is a Pharmacology/Toxicology Reviewer in the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). She joined OTAT in 2006. Her primary focus is the review of preclinical testing... Read More →
avatar for Nita Ichhpurani

Nita Ichhpurani

Consultant, Phase One Forward, Canada
At Pharmacia (now Pfizer), Nita was a discovery Chemist and then PM in Clinical Pharmacology in oncology and CNS research. At MDS PS, she managed Global Central lab teams and later become the Latin American Lab Manager. At MDS PS and Celerion, she managed drug development consulting... Read More →

Kristin Smedley

President, Curing Retinal Blindness Foundation
Kristin Smedley is President and Co-Founder of the Curing Retinal Blindness Foundation, the only patient organization in the world for her two sons’ blindness, CRB1 LCA/RP. Kristin has led the CRBF to raise over $1.3 Million, introduced the first ever legislation in the United States... Read More →
avatar for Melissa Liew

Melissa Liew

Global Head of Translational Medicine-Opthamology, Novartis Institutes of Biomedical Research
Melissa is Vice President and Global Head of Translational Medicine-Ophthalmology, at Novartis Institutes of Biomedical Research, based in Cambridge Massachusetts. In her role, she is working to translate cutting edge basic science discoveries into transformative novel medicines for... Read More →

Tuesday June 25, 2019 2:00pm - 3:15pm PDT
Room 6D San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  06: PreClin Dev-EarlyPhaseCR, Session |   09: Regulatory, Session