GMP Facility

Good Manufacturing Practice (GMP) Production Facility at Nationwide Children’s Hospital

The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital is home to a current Good Manufacturing Practice (cGMP) Production Facility that operates according to FDA cGMP guidelines to ensure the safety of manufactured biologic products. In 2009, NCH established this Good Manufacturing Practice (cGMP) Production Facility, also known as the Clinical Manufacturing Facility (CMF), after successful completion of a Facility Master Validation Plan.

The CMF specializes in the production of recombinant Adeno-Associated viral vectors (rAAV) for use by faculty at Nationwide Children's Hospital, The Ohio State University Medical Center and external collaborators. The primary focus of the CMF is to provide researchers with the highest quality gene transfer vectors to support early phase clinical trials. The CMF consists of a 1,200-sq ft. clean room suite with ISO Class 5/7/8 spaces. The biological drug substances are manufactured according to the FDA Guidance for Industry cGMP for Phase I Investigational Drugs, to ensure product safety, identity, purity, and strength.


Mission
The mission of the CMF is to supply high-quality, clinical-grade viral vectors that can be tested as investigative new drugs in patients with incurable genetic diseases.  The CMF specializes in the production of recombinant Adeno-Associated viral vectors (rAAV) for use by faculty at Nationwide Children’s Hospital, The Ohio State University Medical Center and external academic collaborators.  The CMF allows the Center for Gene Therapy to prioritize products for clinical trials and dramatically reduce costs for vector production.  


Vectors Being Produced
Through this facility, viral vectors are being produced for treatment of several muscular dystrophies including:

  • Duchenne and Becker
  • Limb girdle
  • Spinal muscular atrophy
  • Inclusion body myositis

Additional Viral-Based Therapies
Additional viral-based therapies are being developed to target:

  • Congenital blindness
  • Myotonic dystrophy
  • Facioscapulohumeral muscular dystrophy (FSHD)
  • Lysosomal storage diseases
  • Peripheral neuropathies
  • Pancreatic and liver cancers

Contact the GMP Facility

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000