Gene Therapy Toward More Control?

Last week, a study in Nature Communications by the lab of Dr. Ming Guo at the University of California, Los Angeles described a transgene-based approach to delete lethal mitochondrial DNA via genetic modifications which include expression of PINK1/parkin genes and other activity that promotes mitophagy. The implications of these results have significance for a number of severe diseases such as Parkinson’s and, more broadly, aging.

Such studies demonstrate the significant advancements that genetic engineering has made and the revolutionary potential of gene therapy. And with promising data coming out of clinical trials, coupled with novel gene editing approaches such as CRISPR/CAS-9, there is growing confidence in gene therapy as a viable therapeutic option in the future.

However, the area is still quite new and there are at least as many questions as there are answers. From a regulatory perspective, there is little that has been done to regulate gene editing. Currently, the UK is the only country that regulates mitochondrial replacement. While the FDA, in 2016, attempted to ignite discussion on the technology, further discussions were halted.

However, ongoing conversations are occurring. Regulatory authorities in the US are debating how to regulate these genetic modification technologies. The Human Gene Editing Initiative between the National Academies of Sciences and the National Academies of Medicine will release, in 2017, a Consensus Study on Human Gene Editing, the culmination of multiple years of works and public hearings, a consensus study on gene editing technologies which will cover not just the scientific merits of such technologies, but also the clinical, ethical, legal, and social implications of their use.

Interest in deeper regulation is also felt in the White House, with the President’s Council of Advisors on Science and Technology (PCAST) calling on the creation of a new entity tasked with developing a national biodefense strategy within six months, triggered by their argument that synthetic DNA, gene therapy, and genome-editing technologies like CRISPR open up security issues due to their ability to be misused.

It will be interesting to see how these initiatives shape up with the rapidly expanding scientific landscape.

Akron at World Stem Cell Summit Roundtable

Akron will participate at the Cell Therapy Manufacturing Roundtable at the upcoming World Stem Cell Summit, to take place December 6-9, 2016 in West Palm Beach, Florida. The roundtable will take place 1:30PM – 2:30PM on December 9th. Please let us know if you plan to attend and want to schedule a meeting.