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An Anonymous Coward writes:

After intense discussions regarding the ethical, legal, and social implications of this technology, conversations were initiated at the NIH that led to the establishment of the Recombinant DNA Advisory Committee (RAC) in 1974. The RAC’s mission was to advise the NIH director on research that used emerging technologies involving manipulation of nucleic acids — a mission that was eventually expanded to encompass the review and discussion of protocols for gene therapy in humans. In 1990, the FDA oversaw the first U.S. human gene-therapy trial, which involved pediatric patients with adenosine deaminase deficiency and was conducted at the NIH Clinical Center in Bethesda, Maryland.

Although no major safety concerns were initially reported, over the course of the 1990s it became evident that many questions regarding the safety and efficacy of gene therapy remained unanswered. These unknowns were brought into sharp focus in 1999 when Jesse Gelsinger died of a massive immune response during a safety trial of gene therapy for ornithine transcarbamylase deficiency.1 This tragic death led to closer scrutiny of the field, including a greater focus on open dialogue and increased regulatory oversight.

[...]

In changes proposed on August 17, 2018, in the Federal Register, the NIH and the FDA seek to reduce the duplicative oversight burden by further limiting the role of the NIH and RAC in assessing gene-therapy protocols and reviewing their safety information. Specifically, these proposals will eliminate RAC review and reporting requirements to the NIH for human gene-therapy protocols. They will also revise the responsibilities of institutional Biosafety Committees, which have local oversight for this research, making their review of human gene-therapy protocols consistent with review of other research subject to the NIH Guidelines. Such streamlining will also appropriately place the focus of the NIH Guidelines squarely back on laboratory biosafety.

https://www.nejm.org/doi/full/10.1056/NEJMp1810628 [nejm.org]

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