Reporter |
Editor |Xie Xin
On June 27, biotechnology companies Intellia Therapeutics and Regeneron jointly announced that the investigational in vivo CRISPR gene-editing therapy NTLA-2001 achieved positive results in a Phase 1 clinical trial, demonstrating both safety and efficacy.
These are also the first globally published clinical trial results for an in vivo CRISPR gene-editing therapy. The trial results were simultaneously published in the leading international medical journal *The New England Journal of Medicine*.
NTLA-2001 therapy is an in vivo gene editing treatment that utilizes lipid nanoparticle (LNP) delivery vectors to deliver mRNA sequences encoding sgRNA targeting the pathogenic TTR gene and an optimized SpCas9 protein to the liver.
In the study, NTLA-2001 was used to treat a severe rare genetic disease called transthyretin amyloidosis (ATTR), which is characterized by the progressive accumulation of misfolded transthyretin (TTR) protein in tissues, primarily in the nerves and heart.
Clinical data show that among the six treated patients, three received a dose of 0.1 mg/kg and three received a dose of 0.3 mg/kg. Twenty-eight days after treatment, plasma TTR protein levels in patients receiving the two different doses decreased by an average of 52% and 87%, respectively. No serious adverse events were observed.
John Leonard, CEO of Intellia Therapeutics, stated that the results of this clinical trial indicate that NTLA-2001 may halt the progression of transthyretin amyloidosis (ATTR) disease and reverse its devastating complications with a single injection. This clinical trial also addresses the challenge of delivering CRISPR to the liver, opening the door for the treatment of other genetic diseases. He further stated that the company will rapidly advance this therapy while expanding its existing R&D pipeline.
The Chinese public may be unfamiliar with Intellia Therapeutics, a company founded by Jennifer Doudna, the 2020 Nobel Laureate in Chemistry, who was awarded the Nobel Prize for her outstanding contributions to CRISPR-Cas9 gene-editing technology.
CRISPR-Cas9, also known as the "genetic scissors" technology, is currently the most promising gene-editing technology. CRISPR stands for "Clustered Regularly Interspaced Short Palindromic Repeats," a mechanism used by bacteria to defend against viral invasion. Compared to previous technologies, CRISPR-Cas9 offers advantages such as low cost, ease of use, and high efficiency. It can even alter the code of life—DNA—within a matter of weeks, making gene editing and modification "routine," and has thus swept through the entire biological sciences community.
Using these technologies, researchers can alter the DNA of animals, plants, and microorganisms with extreme precision. This technology has revolutionized the life sciences, creating new opportunities in plant breeding, holding promise for innovative cancer therapies, and potentially turning the dream of curing genetic diseases into reality.
When presenting the 2020 Nobel Prize in Chemistry to Jennifer Doudna and Emmanuelle Charpentier, Claes Gustafsson, Chairman of the Nobel Committee for Chemistry, stated that genetic tools possess immense power and impact us all. They have not only revolutionized basic science, but also yielded new crops, and will lead to breakthrough novel medical therapies.
And now, as CRISPR technology takes a major step forward in clinical trials, the "breakthrough novel medical therapies" anticipated by Claes Gustafsson may no longer be far off.