About Us
The Department of Genetic Medicine at Weill Cornell leads a dynamic and innovative translational research program, advancing diverse fields such as Genetic Therapy and Personalized Medicine.
The Department of Genetic Medicine at Weill Cornell leads a dynamic and innovative translational research program, advancing diverse fields such as Genetic Therapy and Personalized Medicine.
Our translational research program aims to leverage our expertise in genetic therapies and personalized medicine to develop clinical solutions that target the molecular causes of human diseases.
The Department of Genetic Medicine advances treatments and diagnostics through diverse clinical trials, including drug testing and research to better understand diseases.
The Belfer Gene Therapy Core Facility (BGTCF) is a cutting-edge genetic medicine research facility.
The Department of Genetic Medicine at Weill Cornell leads a dynamic and innovative translational research program, advancing diverse fields such as Genetic Therapy and Personalized Medicine.
Our translational research program aims to leverage our expertise in genetic therapies and personalized medicine to develop clinical solutions that target the molecular causes of human diseases.
The Department of Genetic Medicine advances treatments and diagnostics through diverse clinical trials, including drug testing and research to better understand diseases.
The Belfer Gene Therapy Core Facility (BGTCF) is a cutting-edge genetic medicine research facility.
Phase I Randomized, Double-blind, Placebo Control Study for an Anti-cocaine Vaccine
Cocaine addiction is a major social and medical problem, however there is currently no effective therapy. The purpose of this clinical trial is to assess the safety and preliminary efficacy of a vaccine for individuals with cocaine dependence. The vaccine is designed to evoke an immune system response and prevent cocaine from reaching the brain. Because addiction is a chronic relapsing illness, characterized by cycles of drug use and abstinence, a vaccine against cocaine could be a lifetime therapy for the addiction. This trial is a phase I, double-blind, placebo-controlled study. All inquiries are confidential.
Criteria: Adults with cocaine dependence
Study duration: 32 weeks
Age: 21 to 69 years old
Compensation: up to $2400
Additional information can be found on clinicaltrials.gov
Friedreich’s Ataxia (FA) is a genetic disease that causes nervous system damage, which leads to impaired muscle coordination over time, and is also associated with cardiac disease, specifically cardiomyopathy. While the neurological manifestations of FA are debilitating, the cardiac disease is the major cause of mortality and responsible for 60 to 65% of deaths in those with FA. We currently have two studies focused on cardiac disease in those with FA.
Gene Therapy for the Cardiomyopathy of Friedreich’s Ataxia
FA is caused by a mutation in the gene that codes for the protein frataxin (FXN). Frataxin is critical for proper energy production within cells, however, those with FA produce reduced levels of this protein. Without enough FXN, there is not enough energy for the heart to function normally.
In this study, we use an investigational gene therapy that involves a single, intravenous administration of a modified adeno-associated virus, that delivers the normal FXN gene to the heart. It is hoped the study drug will help produce the normal amount of frataxin, and thus return the function of the heart cells and limit the effects of the cardiac disease. The goal of this phase I study is to assess the safety and initial estimates of efficacy of the gene therapy in treating FA-associated heart disease.
Criteria: definitive diagnosis of FA -- detailed eligibility to be discussed with the study team
Study duration: 5 years
Age: 18-50 years old
Compensation: you will receive the reimbursement of your travel expenses, accommodations and meals associated with participating in the trial
Additional information can be found on clinicaltrials.gov and jcto.well.cornell.edu
Characterization of the Cardiac Phenotype of Friedreich’s Ataxia
The purpose of this study is to compare different tests and procedures and to evaluate their usefulness in assessing the cardiac manifestations of Friedreich’s Ataxia. It is currently unknown which of these parameters provides the best evaluation of both the manifestation and progression of FA-associated heart disease and this study aims to fill that gap, in addition to gaining general knowledge about FA. This is an observational/non-interventional study.
Criteria: adults or adolescents with Friedreich’s Ataxia; or adult and adolescent healthy volunteers
Study duration: 3 years
Age: 12-50 years old
Compensation: you will receive the reimbursement of your travel expenses, accommodations and meals associated with participating in the trial
More information can be found on clinicaltrials.gov
Collection of Airway, Blood and/or Urine Specimens for Research Studies
Team up with us in our fight against lung disease! This study aims to collect vital cell tissue and fluid from both healthy individuals and those affected by lung disease. By participating, you’ll be directly contributing to groundbreaking laboratory research that aims to further our understanding of the causes, progression, and treatment of lung disease. As a volunteer, you will undergo procedures like bronchoscopy (inserting a scope into the lungs), alongside other assessments such as chest x-rays and breathing tests – all crucial for advancing the study of lung disease. Your involvement is not only invaluable to us, but also to the millions affected worldwide by lung ailments. Take this opportunity to make an impact and help shape the future of medicine!
Many of our past and current projects include the study of the following conditions:
Do you have a lung condition not listed above? Please reach out to discuss how you may still participate!
No lung disease? No problem! The participation of healthy volunteers is imperative to our research efforts. Contact us for more information on how you can contribute to this worthy cause.
Criteria: adults with lung disease; or healthy adult volunteers
Study duration: 2-3 visits
Ages: over 18 years old
Compensation: monetary compensation available per procedure – inquire with study team for details
More information can be found on clinicaltrials.gov and jcto.weill.cornell.edu