- In an editorial in the New England Journal of Medicine and in a notice in the Federal Register, Francis Collins, director of the National Institutes of Health, and Scott Gottlieb, commissioner of the Food and Drug Administration, laid out new efforts to push forward advances in the fast-expanding field of gene therapy.
- The proposed changes aim to reduce duplication between the NIH, the FDA and the Recombinant DNA Advisory Committee, including removing the requirements for the RAC to review and report on gene therapy protocols, and revising the responsibilities of institutional biosafety committees.
- This changes the role of the RAC back to its original function, as an adviser to the NIH director "on the scientific, safety, and ethical issues associated with emerging biotechnology."
The Food and Drug Administration and the National Institutes of Health have been watching the development of gene therapy closely over the past few decades.
As the therapies evolved, so have federal frameworks, such as the Genetic Modification Clinical Research Information System (GeMCRIS), the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules, the 2017 revisions to the Common Rule (the 1981 rule of ethics regarding participation in biomedical and behavioral research), and the new 2018 FDA guidance aiming to create a modern, comprehensive framework for the development, review and approval of gene therapies.
The evolution of guidance and regulations has led to overlap between the NIH, FDA and research oversight bodies, and the aim of the changes is to streamline and rationalize the process to meet the new needs of what is becoming an established field.
"We at the NIH and the FDA look forward to working together with all our stakeholders to implement these changes. We share common goals: advancing science and human health and accelerating the availability of safe and effective gene therapy, along with the many promising new products that future biotechnologies may bring," Francis Collins, director of the NIH, and Scott Gottlieb, commissioner of the FDA, wrote in the New England Journal of Medicine editorial.
It's been a long route from the first gene therapy patient, a four-year-old girl called Ashanthi with adenosine deaminase (ADA) deficiency, also known as severe combined immunodeficiency or bubble boy disease, who was treated with white blood cells containing the corrected gene in 1990.
Progress is now picking up speed, following last December's historic FDA approval of Spark Therapeutics' Luxturna (voretigene neparvovec-rzyl) for a rare form of hereditary blindness.
On the manufacturing side, Lonza opened the doors to its new 300,000-square-foot plant near Houston, Texas, in April 2018. The company claims it is the world's largest site dedicated to production of cell and gene therapies.
Over in R&D, Novartis has picked up the gene therapy-focused biotech AveXis, bringing on board a gene therapy platform and a late-stage candidate for spinal muscular atrophy (SMA). And earlier this month, Orchard Therapeutics raised $150 million in a Series C funding round to advance its gene therapy pipeline.