Ana Mingorance | Chief Development Officer
Loulou Foundation
Ana Mingorance, PhD, is the Chief Development Officer of the Loulou Foundation, and is focused on accelerating the development of therapeutics for CDKL5 deficiency. Ana is a specialist in genetic epilepsy syndromes and orphan drug development, and is a strong advocate for patient involvement in drug discovery and development.
Appearances:
Day 1- World Orphan Drug Congress USA 2025 @ 14:50
Panel: Indication selection considerations for orphan drugs with broad therapeutic potential
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.
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