Spotlight on Cell and Gene Therapy

1. Cell therapies repair or replace damaged cells or tissue and involve the injection of intact, living cells into a patient. These cells are either derived from the patient (autologous cell therapy) or a donor (allogeneic cell therapy). Cell therapies can be categorized as those utilizing hematopoietic/stem cell approaches or immune cell approaches. 

2. Gene therapy involves introducing, removing, or changing genetic material to alter how a protein or group of proteins is produced in a cell, to alleviate or cure a disease. Gene therapies show great promise with the advancement of genome editing techniques. Currently the FDA has approved gene therapies for an eye disorder called Leber congenital amaurosis and a muscle disorder called spinal muscular atrophy. Gene therapy is typically performed in vivo but there are ex vivo approaches as well. One has the option to choose between viral and non-viral gene therapy. In vivo gene therapy using adeno-associated viruses (AAV) is one of the most common applications.

3. Cell and gene therapies are in some applications combined using ex vivo genetically modified cells as therapeutic agent (cell-based gene therapy) such as ex vivo chimeric antigen receptor (CAR)-T cell therapy. Cell and gene therapy technologies can be autologous or allogeneic. Autologous transplantation is patient specific, whereas allogeneic transplantation extracts cells from a universal donor before cell expansion and delivery into multiple patients.

Each approach has advantages and challenges.

Cell and gene therapies are transforming how patients with cancer and genetic diseases are treated. Although the therapeutic opportunities for patients are promising, the stakes for drug developers are high. Companies wishing to scale up the manufacture of cell and gene therapy from translational research to the clinical trials phase must address complex challenges. For example, scaling up production under GMP conditions or dealing with regulatory bodies. Additionally, smaller operations may experience further inefficiencies and costs owing to paperwork-heavy compliance processes.

Traditionally GMP solutions for cell and gene therapy are not implemented until later in the process, however the use of research grade materials in the transitional phase may lead to invalidated experiments, new filings being needed, and more back and forth with regulatory agencies to defend decisions made earlier on, right at the finish line. Collaborating with a strong and experienced partner can help you succeed on this path.