Genetics Clinical Trials Office

The Department of Genetics and Genomic Sciences and the Icahn Genomics Institute (IGI) at the Icahn School of Medicine at Mount Sinai is paving the way for the future of genomic research and medicine at the largest and most diverse health care system in New York City.

The Department's Division of Medical Genetics, a NORD Center of Excellence for Rare Diseases in patient care and clinical research, has a substantial history of translational work and conducting clinical trials for patients with rare genetic disorders, and the IGI serves as the DNA and RNA therapies hub for Mount Sinai. 

We have a longstanding history of translational work and conducting innovative clinical trials for patients with rare genetic disorders. These include early stage first in human studies to late stage pivotal clinical trials. These clinical trials have led to approval of new rare disease treatments for patients by the Food and Drug Administration (FDA) and European Medicines Agency (EMA).

We are experienced in performing industry, National Institutes of Health (NIH), and investigator-initiated studies. Our studies include groundbreaking gene therapies, small interfering RNAs, messenger RNA therapies, enzyme-based therapies, synthetic biotic and small molecules, and many more. We are committed to advancing genetic medicine by increasing access to novel therapeutics to patients with rare genetic disorders.

Expertise in Gene Therapies

Our team has been at the forefront of conducting gene therapy trials including first-in-human phase I trials. These have used novel AAV liver-targeted technology to treat rare diseases such as Phenylketonuria, Fabry Disease, Ornithine Transcarbamylase Deficiency with others on the way. Some therapies included preclinical work done at Mount Sinai, such as a novel gene editing approach developed to treat Fabry Disease, illustrating our success with bench-to-bedside research, and our commitment to treating patients with rare and genetic diseases.

Study Services

We have executed all types of clinical research studies. Whether your study is observational, a first-in-human trial, or a phase III pivotal trial, our team will help get you through start-up to closeout efficiently.

Our services include:

  • Completing confidential disclosure agreements
  • Conducting feasibility assessments
  • Protocol design
  • Coordinating site qualification and site initiation visits
  • Negotiating clinical trial agreements and study budgets
  • Providing research staff to successfully execute the study
  • Conducting regulatory submissions
  • Training study personnel
  • Promoting recruitment and retention of study subjects
  • Resolving operations or regulatory issues
  • Facilitating monitoring and audits, including FDA audits

Visit the intranet for information on internal investigator-initiated studies and contact the CTO Director at the Department of Genetics and Genomic Science to initiate a new study. The Office of Research Services outlines the details for industry initiated clinical research at the Icahn School of Medicine.