Juan Tan, PhD
Associate Director II
Alice Zhang, PhD
Director, Strategy Support
Lin Liu, PhD
Vice President, Molecular Biology and Gene Therapy
Originally Aired: February 27, 2024
Time: 8:00 am PT, 11:00 am ET, 17:00 CET
Advances in the understanding of rare disease biology, coupled with innovative technology and therapeutic platforms, has led to advances in the research and development of drugs for rare diseases. Strategies such as enzyme replacement, cell therapy, and oligonucleotide therapeutics create a diverse toolbox for tailored interventions, leading to an increase in drug approvals. Despite this progress, many rare diseases still lack useful treatments and the need for new approaches remains high.
In this GEN webinar, our expert speakers will discuss important trends in rare disease therapeutics and highlight ways of accelerating the drug discovery and development process. The webinar will provide a comprehensive and stimulating perspective for the many research teams committed to increasing drug accessibility for people living with rare diseases.
A live Q&A session followed the presentation, offering a chance to pose questions to our expert panelists.
Produced with support from: