Hello, I'm Agnes.
Agnes Rafalko, PhD, is a biotech founder and translational science leader with over 15 years of experience advancing first-in-class therapies from early discovery through IND-enabling development and clinical readiness, with a strong focus on rare and pediatric diseases. She has built and led multidisciplinary teams across discovery, preclinical development, bioanalytics, CMC, and translational strategy, and is known for translating complex, high-uncertainty biology into credible, development-ready programs. Agnes is the founder and former CEO and CSO of Glycomine, a venture-backed rare-disease biotechnology company developing small-molecule therapies for Congenital Disorders of Glycosylation. At Glycomine, she led scientific and technical strategy end-to-end, raised $45 million in venture financing, oversaw IND-enabling development, and supported global regulatory interactions and early clinical planning. She later served as Director of R&D at MiNA Therapeutics, where she led translational programs for lipid-nanoparticle–formulated RNA therapeutics across oncology and rare disease. In this role, she developed a systematic decision framework to evaluate thousands of rare genetic diseases for suitability with novel RNA modalities, enabling focused, capital-efficient portfolio prioritization despite limited precedent. Earlier in her career at BioMarin, Agnes established the company’s first mass spectrometry bioanalytical capability and developed a critical bioanalytical method that resolved a major manufacturing bottleneck, lifted a production hold, enabled study progression, and delivered multi-million-dollar cost savings—an experience that cemented her long-standing commitment to rare disease drug development. Across roles, Agnes has worked extensively with academic investigators, CROs, patient advocacy groups, and funding organizations. She has helped initiate natural history studies essential for rare disease clinical development and has led non-dilutive funding strategies spanning SBIR/STTR and patient-advocacy–led foundation programs. She is deeply motivated by building therapies that meaningfully improve the lives of patients and families affected by serious rare diseases.