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Prenatal Somatic Cell Gene Therapies: Charting a Path Forward for Clinical Applications

As gene therapy gains momentum in treating single gene disorders in pediatric and adult patients, it is an apt time to discuss clinical applications to treat fetuses with severe or fatal genetic diseases. Preclinical data underscore the possible advantages of gene therapy or gene editing before birth: fetal exposure to a missing protein may result in tolerance; treating before formation of the blood-brain barrier may improve neurologic function; and early treatment may improve survival for many progressive diseases, some of which are fatal before birth. There are also risks and other considerations that are particular to prenatal application. We are convening a group of scientists, clinicians, ethicists, patient advocates, and regulatory professionals to assess the risks and benefits of prenatal somatic cell gene therapy (PSCGT) in humans. Discussion will focus on therapies that target somatic cells of fetuses with severe, early onset disorders.

The objective of the virtual workshop is to consider the various scientific, clinical, ethical, and regulatory aspects of prenatal somatic cell gene therapy for fetuses with severe, early onset genetic diseases and to articulate a prudent roadmap for first-in-human clinical applications.

The workshop will:

  •     Summarize the current state of fetal therapy, including the path to IND for current prenatal medical therapies (stem cell and enzyme replacement therapy).
  •     Consider the available preclinical data and scientific rationale for prenatal gene therapy in various animal models.
  •     Review several gene therapy and gene editing strategies currently in clinical trials for postnatal indications, using spinal muscular atrophy as an example.
  •     Examine principles of fetal and maternal monitoring, informed consent, and patient perspectives in designing a clinical trial.

This virtual collaborative workshop is co-sponsored by the UCSF-Stanford CERSI, the UCSF Center for Maternal-Fetal Precision Medicine, and the U.S. Food and Drug Administration.

Event Details

  • Val Dougherty

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