Overview  

While the gene therapy field is not new, the regulations and paradigm for how to approach and navigate gene therapy development is novel and evolving. In January of 2020, the FDA finalized six gene therapy specific guidance documents, with more anticipated in the near future. As a regulatory professional trying to establish oneself in the gene therapy space, it is essential to have a fundamental understanding of the FDA’s framework. 

Learning objectives          

• Identify US regulatory pathways available for gene therapy products 
• Discuss key considerations for the development of gene therapy products
• Describe the current state of gene therapy regulations in the US
• Recognize the major challenges and potential benefits of gene therapy development
  
   

Target Audience

• Junior regulatory professionals from industry, academia and government. 
  
  

Speakers

Daniela Drago 
Daniela Drago, Ph.D., RAC is Senior Director Regulatory Sciences at Biogen. Before joining Biogen, she was an Associate Professor at George Washington University's School of Medicine and Health Sciences. During her career, she has held senior positions in global regulatory and medical affairs at Roche, Vifor Pharma, and Bausch & Lomb. Daniela has significant experience in the development of small molecules, biologics, gene therapies, and combination products in rare and common disease areas, including oncology, ophthalmology, and neuroscience. In recognition of her achievements, Daniela received several awards, including the TOPRA Award for Regulatory Excellence and the RAPS Community Leadership Award. Currently, she serves on the Regulatory Affairs Certification's Board of Directors, the Executive Committee of the American Course in Drug Development and Regulatory Science, and the Regulatory Affairs Committee of the American Society of Gene and Cell Therapy. She is also the former Chair of the RAPS DC/Baltimore Chapter and a member of the Advisory Council to the TOPRA Board. Daniela received her Ph.D. in chemistry from the Swiss Federal Institute of Technology (ETH) in Zurich. 

Kiley Keelin 
Kiley Keelin, MA is a Director of Regulatory Sciences at Biogen. Prior to joining Biogen, she served as a Global Regulatory Strategy Lead at Spark Therapeutics, where she developed and led global regulatory strategies for the central nervous system and the ophthalmology portfolios. Most notably, Kiley’s responsibilities included Luxturna, the first FDA-approved AAV (in vivo) gene therapy, a treatment for patients with a hereditary retinal disease caused by biallelic RPE65 gene mutations. During her 12 year career, she has been an integral part of growing regulatory teams in several startups.  Her extensive experience includes developing and executing regulatory strategies for biologics, including vaccines, immunotherapies, and gene therapies across various disease areas, including ophthalmology, neuroscience, and oncology.  She has led the development and maintenance of a wide range of regulatory applications, including INDs, CTAs, CTNs, MAAs and BLAs. Kiley received her BA from Johns Hopkins University and her MS from the Drexel University College of Medicine. 

CPD: This course offers 1 CPD hours for successful completion. To learn why CPD is important visit our Lifelong Learning page. 

How do I register?

Simply log in or create an account and register below. 

Pricing

Standard

• Member: £70 + VAT
• Non-Member: £95 + VAT

Discounted Places

A limited number of discounted places are available at the rates below for those:

• in low income countries as defined by the World Bank
• working for agencies, charities or NGOs. 

Please email meetings@topra.org for a discount code before making your booking.

Discounted fees:

• Member:  £35 + VAT*
• Non-member: £47.50 + VAT*

* VAT, if applicable, is charged at a rate of 20%.

Terms and conditions

By purchasing this webinar, you are agreeing to the terms and conditions.