Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA) provides an overview of recent advances in gene therapy.
As Dr. Marks states, we are in a very interesting time for gene therapy development. More specifically, Dr. Marks noted that the FDA learned a tremendous amount during the Covid-19 pandemic about clinical trial design, patient safety, drug development, etc. The organization hopes to use that knowledge to both fix some of the issues with gene therapy clinical trials. Some issues that existed before the pandemic and augmenting those with what has been learned during the pandemic.
Regarding the safety of gene therapy, Dr. Marks noted that there were some safety concerns in the past clinical trials but the scientific community has found the vectors that appear to alleviate those safety concerns. Dr. Marks said the new vectors are reasonably safe and appear to achieve some remarkable results, such as the approved gene therapy for spinal muscular atrophy.
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