PAST
For over 20 years scientists and researchers have been investigating ways gene therapy can both treat or prevent genetic diseases; attempting to treat the disease at the molecular level by correcting mistakes at the gene level.
The 80’s kicked off the Human Genome Project. Completed in it’s entirety in 2003 the project identified and mapped all genes from the human genome. With this, researchers are more easily able to identify target genes.
By the early 90’s the first gene therapy was being delivered to newborn patients with ADA deficiency (adenosine deaminase deficiency, ADA-SCID) a rare metabolic disease.
PRESENT
Fast forward to 2012, as Europe approved the first ever therapy (Glybera, uniQure) for an ultra-rare inherited disorder lipoprotein lipase deficiency (LPLD). Followed then by GSK in 2016, receiving approval for the first gene therapy for a small population of children with ADA-SCID.
FUTURE
With current gene therapy advancements and multiple ongoing trials, the focus has now shifted to manipulating particular sequences within the gene to determine their function or to alter their effect.
One of these manipulations, CRISPR (clustered regularly interspersed short palindromic repeats that characterize the genomic loci involved) has been making headlines as well as huge waves in the industry.
The Food and Drug Law Institute provided some insightful information in this month’s issue of Update Magazine with their feature article titled “Ready or Not, CRISPR and Gene Editing Have Arrived and Are Here to Stay.”
The article not only provides a general understanding about gene editing and CRISPR, it also discusses potential applications and regulatory challenges.
With key players like Editas, CRISPR, and Intellia in developments, and Abeona, Precision and Caribou on the rise, 2016 and 2017 look to be promising years for even more gene editing advancements.
As for the regulatory bodies;
As FDLI mentions, the FDA will focus on intended use and like all potential new therapies; safety and efficacy. They (including the OCTGT and OOPD) will continue to offer more and more understanding and insight into these therapies. And by the looks of things, will continue to incentivize by granting Orphan Drug Designations, Fast Tracks, Breakthrough Therapy Designations, and additional and early support with pre-preIND meetings.
