Welcome to our new website! If this is the first time you are logging in on the new site, you will need to reset your password. Please contact us at firstname.lastname@example.org if you need assistance.
Your membership opens the door to free learning resources on demand. Check out the Member Knowledge Center for free webcasts, publications and online courses.
This comprehensive resource covers product change evaluation, postmarket surveillance, audit/inspection compliance, and various other laws and regulations pertaining to maintaining a product on the market.
Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
Regulatory News | 14 November 2017 | By Michael Mezher
At a Senate Health, Education, Labor and Pensions (HELP) hearing in Washington, D.C. on Tuesday, experts explained that the current regulatory framework for gene editing in the US is appropriate and cautioned that an overly strict approach could drive research to other countries.
Specifically, the hearing focused on CRISPR/Cas9, which is a gene editing tool that scientists can use to quickly and accurately edit DNA in a variety of applications.
While there are currently no clinical trials involving CRISPR in human patients in the US or Europe, Matthew Porteus, an associate professor at Stanford University, said he expects such trials to launch within the next 12 to 18 months.
"The current regulatory structure with the FDA, the Recombinant DNA Advisory Committee (RAC) and [an institutional review board] IRB is completely adequate in handling the assessment of the science and ethics of doing genome editing of somatic cells," Porteus said.
Katrine Bosley, CEO of Editas Medicine, said she supports the current regulatory framework for gene editing involving NIH's RAC and FDA.
"FDA has the appropriate authority and they're exercising it well and thoughtfully, so I don't see any need for any change in legislation," she said.
Bosley offered some praise to FDA for keeping on top of the rapidly developing field.
"Our experience has been very much that [FDA is] at the leading edge of understanding the science. It is a fast moving field and they're keeping pace with it," she said.
Bosley also said her company is preparing an investigational new drug application (IND) for its CRISPR-based treatment for Leber's congenital amaurosis type 10, a rare genetic disorder that causes blindness, which it hopes to file by mid-2018.
Jeffrey Kahn, director of the Johns Hopkins Berman Institute of Bioethics, called the current regulatory framework for gene editing "robust," but cautioned the committee on several ethical challenges that regulators will face as the use of these technologies moves forward.
First, Kahn said that regulators will need to find a way to manage "indication creep" for gene therapies, as once those technologies reach the market it may be difficult to ensure they are used in the intended manner.
Kahn also said gene editing that would introduce heritable changes raise serious ethical concerns. "We will need very strict oversight if that's ever to go forward," he said, pointing to recommendations from the National Academies of Science as a model for future guidelines.
But Kahn cautioned that overly strict regulation or prohibitions, as such Canada's criminal ban on germline gene editing, could push scientific research to other countries with little or no oversight.
"A much smarter approach to policy is strict control to allow careful, responsible science to go forward in ways that are controlled and within our borders," Kahn said.
Tags: Gene Editing, Gene Therapy, CRISPR, CRISPR-Cas9, Editas