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The money will fill gaps in gene editing and help find ways to better disseminate knowledge and tools.
The National Institutes of Health has launched a new funding program aimed at shoring up research gaps around therapeutic genome editing in humans.
NIH’s Somatic Cell Genome Editing Program will spend some $190 million over 6 years to better hash out the processes and tools needed to edit the human genome, and also to create resources to share the resulting knowledge with the broader scientific community. Genome editing has already been the focus of much attention and much research, thanks largely to publicity around the breakthrough CRISPR/Cas9 technology, but this step stands to further cement its ascent.
In announcing the new funding pool, NIH Director Francis S. Collins, MD, PhD, said genome editing is “revolutionizing biomedical research.” The NIH’s goal with the funding, he added, is to “dramatically accelerate” the time it takes to translate these scientific breakthroughs to real-world clinics.
The technology is seen as particularly promising in the world of rare diseases. Many rare diseases can be triggered by changes to a person’s DNA. By editing those patients’ DNA, the hope is that scientists can essentially correct the conditions. This would be helpful since rare diseases often are considered a low priority by drug developers, and even when therapies are developed, they are often extremely expensive.
The first attempt on United States soil to treat a patient using a gene-editing therapy within the body was attempted in November on a 44-year-old patient with Hunter syndrome.
Meanwhile, CRISPR Therapeutics, a company based on the gene-editing technology, filed an application in December for its first gene-editing clinical trial, which will focus on treating patients with β-thalassemia and sickle cell disease.
The NIH noted that the new funding will focus on somatic cells, which don’t reproduce. Thus, changes to the DNA of these cells won’t be passed down to future generations. They will, however, prevent or cure diseases, researchers said.
Rachel Britt, PhD, a health specialist and communications director for NIH’s Common Fund, which will fund the somatic cell genome editing program, said the money is earmarked to plug specific gaps in the research.
“The NIH identified several genome editing research areas in need of investment, including optimized genome editors, specifically targeted delivery systems, and more predictive animal models and studies,” she told Healthcare Analytics News™.
Britt said the research funded by NIH can help out the broader scientific community, rather than mere individual research efforts.
”Although research in each of these fields has been very active, efforts have focused on the needs of individual studies and not on overall procedures for improving and testing the safety and efficacy of genome editing approaches,” she said.
Britt outlined 5 key components of the program: creating better animal models for assessing genome editing in vivo, developing tools and assays to detect adverse consequences from gene editing in human cells; finding new technologies to deliver genome editing machinery into tissues and cells in vivo; and launching a “Dissemination and Coordinating Center” to make findings and data funded by the program available to the wider scientific public.
A number of specific funding opportunities have already been announced. They are listed on NIH’s Common Fund website.