Gene Therapy

 

Gene therapy holds promise for treating countless diseases and disorders including cancer, cystic fibrosis, heart disease, diabetes, hemophilia, AIDS, and many more. Through use of a vector (viral or non-viral), a new copy of a gene or relevant nucleotide sequence is inserted into a patient’s cells ex vivo or in vivo to either compensate or correct for the deleted or defective version that is causing the disease, or enhance an existing version. The emergence of powerful new technologies such as the gene-editing tool CRISPR served as a breakthrough for the biotech industry and elicited resurgence in the field, which is now being met by impressive research demand.

 

Currently, gene therapy is only usually available as part of a clinical trial, and product approvals using these technologies are just on the horizon with numerous technical challenges (and opportunities) associated with them. Preclinical and clinical research is ongoing  to better understand how and when gene therapy will be effective in a patient. Particular needs include: 1) improvements in vector safety; 2) regulation of gene expression; 3) enhanced cell transduction capability; 4) vector packaging; and 5) reference materials for viral tools, but the manufacturing, and measurement and analytical techniques to support these processes are lacking.

 

The absence of a common set of standards in gene therapy programs has proven to be a significant challenge  for regulators, industry leaders, and academic researchers, but there is no mistaking the utility, and, therefore, need, for standards (and related technologies) in these areas. Mike Kelly (Biogen) and Dr. Maritza McIntyre (Advanced Therapies Partners) Co-chair our effort to elucidate the specific needs for characterization and control in the gene therapy field and subsequently pioneer projects to bring us one step closer to establishing consensus standards.

 

Gene Therapy Chairs

Dr. Mike Kelly, Biogen

Dr. Maritza McIntyre, Advanced Therapies Partners