Dr Shane Hegarty is Chief Scientific Officer, Co-Founder and IP Co-Inventor at AXONIS Therapeutics, a Boston-based biotechnology company. He has 15+ years of experience leading cutting-edge, award-winning neuro-protection, -modulation and -regeneration research for neurological disorders. In recent years, Shane has completed a number of competitive biotech entrepreneurial programs, such as Creative Destruction Labs and Harvard I-Labs. As a Research Fellow in Professor Zhigang He’s lab in Boston Children’s Hospital and Harvard Medical School, Dr. Hegarty led in vivo phenotypic screens, such as a genome-wide AAV-CRISPR screen for neuro-protection and -regeneration in a CNS injury model. Before leaving a tenure-track position in Ireland to pursue cutting-edge research in the US, he was a Faculty Member in Neuroscience Department of University College Cork, and received the prestigious Neuroscience Ireland Investigator Award in 2016. During his academic career, Dr. Hegarty was awarded several highly-competitive scholarships and fellowships to support independent research, including from the Irish Research Council, National University of Ireland and William Randolph Hearst Foundation. A Royal Irish Academy grant supported post-doctoral training in the Erasmus MC, Netherlands and KU Leuven, Belgium. A highly productive researcher with 30+ research papers (22 as first/senior author), >1,400 citations, several patents, h-index = 18, and a serial grant awardee. Dr. Hegarty has acted as an Editor for Frontiers and The Biochemist, and expert reviewer for a number of esteemed journals and international funding agencies. An experienced entrepreneur, innovator, researcher, team builder and teacher, who graduated top of his Neuroscience BSc class.
With the tremendous progress in recent years of genetic screening and more sophisticated clinical diagnosis and biomarker approaches, broader populations of patients who share common pathologies and/or molecular etiologies are being stratified into subpopulations of orphan and rare diseases. This has enabled precision medicine approaches that, for example, can selectively target the underlying genetic driver of a disease. Moreover, in the past, a popular drug development strategy has been to test novel therapies in a subpopulation before expanding into the larger disease population. This expert panel will offer diverse perspectives on indication selection considerations and challenges for novel orphan drugs that have broad therapeutic potential for several rare disease patient subpopulations, such as clinical development strategies, regulatory landscape, patient accessibility and the impact of the IRA on commercial models.