Belfer Gene Therapy Core Facility

To provide Weill Cornell Medicine, Cornell – Ithaca, and researchers from beyond the Cornell community with the infrastructure to carry out basic, translational, and clinical research utilizing gene transfer for therapeutics and vaccines. 

The Belfer Gene Therapy Core Facility (BGTCF) provides an enabling function at Weill Cornell Medicine in two significant ways: 

1. The BGTCF trains users from research laboratories in the methods of viral vector construction and characterization. This allows the visiting trainees to bring the learned expertise to their home laboratory, thereby facilitating research progress that aligns with their labs’ overall goals and objectives. In effect, this mechanism casts a net of expertise larger than the BGTCF facility itself, propagating gene therapy applications towards their therapeutic goals.  

2. The Good Manufacturing Practices (GMP) component of the BGTCF provides a pathway to clinical investigations for researchers with translational programs in which commercial options would be prohibitively expensive. As a result, numerous safety/toxicology studies and clinical trials have been conducted with viral vectors produced by the BGTCF. These include adenovirus vectors, adeno-associated vectors, and cell lines produced for academic investigators, as well as biotechnology companies, non-profit foundations, and the National Institutes of Health (NIH). The BGTCF has produced over 100 GMP drug candidates for safety/toxicology studies and clinical trials. Viral vectors produced by the BGTCF have been used in 28 clinical trials in the US and Europe to date.  

The BGTCF facility provides the capacity to: 

• Synthesize new gene therapy vectors and vaccines based on adenovirus and adeno-associated virus replication-deficient platforms 
• Validate the function of vectors 
• Perform pre-clinical testing in proof-of-concept models 
• Conduct toxicology, safety, and distribution studies in support of regulatory filings 
• Produce GMP-grade vectors for clinical trials 

The BGTCF staff work closely with faculty in the Department of Genetic Medicine and other Weill Cornell Medical College departments to support research in the areas of gene therapies and vaccines, in addition to providing contract services to outside academic and corporate groups needing the specific expertise and capabilities of the BGTCF.  

The BGTCF enables researchers and supports the education of Cornell laboratories for the methods and strategies of gene therapy product construction and production. The BGTCF has facilitated, and currently supports, ongoing initiatives for translation to clinical trial for members of the local, national, and international research community including: 

• Weill Cornell Medicine
  • Dept. of Ophthalmology 
  • Dept. of Pathology & Laboratory Science 
  • Dept. of Cardiothoracic Surgery 
  • Citigroup Imaging Facility 
  • Dept. of Neurosurgery 
  • Dept. of Pediatrics 
• Memorial Sloan Kettering Cancer Center 
• Hospital for Special Surgery 
• Rockefeller University 
• University of Massachusetts Medical School 
• University of Pittsburgh Medical School 
• UCLA-Cedars Sinai Hospital 
• National Institutes of Health 
• Numerous biotech companies and non-profit foundations
  • Telethon Institute of Genetics and Medicine (TIGEM), Italy 
  • University of Paris, France 
  • Lexeo Therapeutics 
  • Adverum Biotechnologies