Agenda Details

The advent of recently translated technologies, including antisense oligonucleotides and CRISPR-mediated gene editing, has resulted in a new era of precision medicine and unprecedented drug development for individual patients. There are also gene therapies achieving first-time market approval for indications, such as spinal muscular atrophy (SMA) and adrenoleukodystrophy (ALD), which can maximize availability of these therapies for patients. In this interactive workshop, you will learn from both scientific and patient advocate perspectives, along with participating in round table discussions on the topics covered. We will explore the spectrum of accessibility for gene therapies covering: 1) precision medicine advances, 2) expanded access approaches, 3) conventional clinical trial design, 4) and market approved therapies.