PDA Letter Article

The Future of Cell and Gene Therapies is Here

by Austin Caudle, Metabolon, Inc.

After decades of research and some ill-fated setbacks, R&D and manufacturing pipelines around the globe are filled with gene and cell therapies, opening up the long-anticipated promise of these powerful technologies, making patient-centered therapies and precision medicine a reality. Many technical hurdles, such as the challenges around how to incorporate DNA into a gene, have been reduced through a new generation of vectors, and stem cell companies are increasingly excited about therapies that are proving safe and efficacious in treating currently untreatable diseases. Initiatives to develop novel therapeutic options for chronic, rare and inherited diseases along with the growing demand for regenerative treatment options are the most significant drivers.


Regulators are also exploring the bases of these treatment options. On July 12, the U.S. FDA recommended that Novartis’ CAR-T therapy for treating advanced leukemia in children and adults, CTL019, be considered for approval. This customized therapy involves harvesting a patient’s white blood cells and “reprogramming” them to attack tumors. A final decision from the Agency is expected in November.

The Agency is also in the early stages of developing regulations for the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) gene editing technique (1). This experimental procedure removes targeted DNA sequences, offering the potential to modify genes in patients that lead to congenital disease, cancers and other ailments.

In light of these advancements and the challenges of manufacturing complex and individualized treatments, PDA will host the 2017 PDA Cell and Gene Therapy Conference in San Diego this December. Experts in medicine, regulation, quality, and facilities and engineering will converge at this event to facilitate discussion on current topics such as the application of big data, management of unique regulatory requirements and considerations for scaling of processes and processing spaces. The conference opens with presentations addressing the current state of gene and cell therapy and each subsequent session tackles a critical component, interspersed with speakers from FDA, medical institutions, pharmaceutical and biotechnology companies, and concludes with a session looking at next-generation approaches.

As part of the final conference plenary session, Jennifer Bond, PhD, Project Leader for the Clinical & Translational Science Institute (CTSI) at Duke University, will present on the development of a gene therapy with adeno-associated virus (AAV) vectors. Duke University researchers have developed a gene therapy they hope could enhance or replace the only FDA-approved treatment currently available to patients. This therapy uses a modified virus to deliver a gene to the liver where it produced acid alpha-glucosidase (GAA) an enzyme missing in people with Pompe disease. Approval has been received from FDA to launch a Phase I clinical run in humans. The emerging gene therapy is the latest development from a team of scientists at Duke who have been working for three decades to study the causes and potential treatments for glycogen-storage diseases, and specifically Pompe. To complement Bond, Robert Kesterson, PhD, Professor of Genetics at the University of Alabama at Birmingham School of Medicine, will speak on “CRISPRs & Gene Editing: Laboratory Use and Clinical Future.”

Please join us for this exciting event to hear these and other influential speakers as gene and cell therapy takes center stage for two days in sunny Southern California.


  1. Brennan, Z. “Regulating CRISPR: FDA and Industry Offer Perspective.” Regulatory Focus (June 21, 2017)