Genetics Clinical Trials Office

The Genetics Clinical Trials Office at the Department of Genetics and Genomic Sciences has a longstanding history of translational work and conducting innovative clinical trials for patients with rare genetic disorders. Supported by the Division of Medical Genetics and Genomics—a National Organization for Rare Disorders (NORD) Rare Disease Center of Excellence—and the Icahn Genomics Institute, which serves as Mount Sinai's hub for DNA and RNA-based therapies, our office brings together deep clinical expertise and robust research infrastructure to advance genetic medicine. To date, we have supported more than 50 industry-, NIH-, and investigator-initiated studies, ranging from early-stage first-in-human trials to late-stage pivotal clinical trials—several of which have led to approval of new rare disease treatments by the FDA and European Medicines Agency.

Our studies span a broad range of therapeutic approaches, including gene therapies, small interfering RNA therapies, messenger RNA therapies, enzyme-based therapies, synthetic biotic therapies, and small molecules. A particular area of strength is gene therapy, where our team has been at the forefront of conducting first-in-human phase I trials using novel adeno-associated virus liver-targeted technology to treat rare diseases including Phenylketonuria, Fabry Disease, and Ornithine Transcarbamylase Deficiency. Some of these therapies originated from preclinical work conducted at Mount Sinai, illustrating our commitment to bench-to-bedside research and to expanding access to novel therapeutics for patients with rare and genetic diseases.

Study Services and Site Qualifications

The Genetics Clinical Trials Office is equipped to execute all types of clinical research studies—from observational studies and first-in-human trials to phase III pivotal trials—guiding sponsors and investigators efficiently from startup to closeout. Our team provides a full range of study services including feasibility assessments, protocol design, site qualification and initiation, clinical trial agreement and budget negotiation, regulatory submissions, study personnel training, subject recruitment and retention, and facilitation of monitoring and audits including FDA audits. Our principal investigators are expert clinicians and experienced researchers committed to the rights, safety, and overall well-being of study participants, and our clinical and research teams are recognized as early and high recruiters for clinical trials.

Site selection is critical to the success of any clinical trial, and our dedicated Clinical Trials Office is structured to meet each study's unique needs—identifying and selecting the best personnel and leveraging the full resources of the Icahn School of Medicine at Mount Sinai to simplify the qualification and training process. Our study staff includes multiple experienced clinical research coordinators trained in adverse event tracking and reporting, case report form completion, and multiple electronic data capture systems. Financial services include budget development and negotiation, conflict of interest review, grants and contracts facilitation, and timely invoicing and account reconciliation.

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