Senior Group Leader - Rare Genetic Eye Diseases and Therapeutic Innovation

## Responsibilities - Lead an internationally visible research program focused on inherited eye diseases, combining disease mechanisms, human modelling, and therapeutic development. - Develop a program that bridges basic discovery and translational application, with the potential to advance precision ophthalmology for rare genetic diseases. - Integrate CRISPR-based genome engineering, functional genomics, and single-cell or multi-omic profiling to investigate disease mechanisms and therapeutic response. - Develop innovative therapies including RNA therapeutics, antisense oligonucleotides, readthrough approaches for nonsense mutations, gene editing, cell-based therapies, and mutation-independent or pathway-based therapeutic strategies. - Conduct preclinical target validation using disease models, organoids, and animal systems. - Engage in biomarker discovery, patient stratification, and translational frameworks supporting therapeutic readiness. - Build a major program in ocular genetics and translational ophthalmology within a world-class rare disease institute. - Collaborate with Necker Hospital and national and European rare disease networks. ## Requirements - PhD, MD, or MD/PhD in a relevant field. - Recognized expertise in inherited eye diseases, ocular development, and translational ophthalmology, supported by an outstanding record of peer-reviewed publications. - Strong experience in human stem cell biology, including the derivation and use of patient-specific iPSC models and advanced ocular organoid systems. - Demonstrated ability to develop human disease models relevant to retinal, corneal, iris, or other ocular disorders, and to use these systems for mechanistic and translational studies. - Expertise in developmental and regenerative approaches to ocular biology, including tissue specification, degeneration, fibrosis, or cellular plasticity. - Experience in integrating CRISPR-based genome engineering, functional genomics, and single-cell or multi-omic profiling to investigate disease mechanisms and therapeutic response. - Ability to bridge rare disease genetics, disease modelling, and therapeutic innovation, including RNA-based therapies, readthrough approaches for nonsense mutations, gene modulation strategies, or cell-based therapies. - Strong track record of scientific leadership, competitive funding, and successful multidisciplinary collaborations involving basic, translational, and clinical research. - Excellent communication, mentoring, and team-building skills, with the capacity to structure a visible and ambitious research program within the institute. ## Nice to Have - Expertise in human stem cell models, ocular organoids, rare disease genetics, and/or therapeutic development for inherited eye disorders. ## Benefits - Senior-level group leader position. - Internationally competitive package. - Access to cutting-edge technological platforms. - Dynamic and collaborative scientific environment. - Strong clinical integration and translational opportunities in rare genetic diseases.