Original title: The first in-person gene editing technology is beginning to show efficacy, creating a new situation in the treatment of genetic diseases
After years of research and development, gene editing technology is gradually moving from the laboratory to the clinic. On Saturday, the world‘s first clinical data of the CRISPR gene editing technology in an individual was announced, preliminarily showing the safety and effectiveness of the technology. The results of this study were published in the “New England Journal of Medicine”, marking a milestone in the treatment of genetic diseases.
This research was jointly developed by the American biotechnology company Intellia Therapeutics Company and Regeneron Company. CRISPR in vivo gene editing therapy NTLA-2001 is used in the treatment of hereditary transthyretin amyloidosis (ATTR) disease. The study showed a positive effect. A one-time injection can effectively reduce the level of transthyretin (TTR) in the serum, with an average decrease of 87% in TTR.
Researchers believe that the decrease in serum TTR levels is a known biomarker of ATTR amyloidosis, indicating that patients benefit. The gradual accumulation of TTR protein in tissues may cause neurological diseases and heart diseases. This latest clinical data also shows the potential efficacy of gene editing technology in the treatment of other genetic diseases.
Intellia’s Chief Medical Officer, Dr. David Lebwohl, said: “This is the first time that gene editing has been proven effective for humans. For patients with amyloidosis, this gene editing therapy may be a good one. But the future applications are far more than that. It opens up a whole new field of treatment for patients.”
Intellia was co-founded by Professor Jennifer Doudna, winner of the 2020 Nobel Prize in Chemistry. Other American gene editing technology startup companies include Editas Medicine, CRISPR Therapeutics, Beam Therapeutics, etc.
Lebvo was a member of the Kymriah CAR-T cell immunotherapy developed by Novartis. He believes that the in vivo CRISPR/cas9 gene editing technology will take precision therapy one step further. By precisely editing targeted cells in the patient’s body, it will open the door for the use of this platform to treat other genetic diseases.
He said that Intellia chose to start with multiple neurological diseases and hope to enter more disease fields including cardiomyopathy in the future. There are as many as tens of millions of people suffering from severe cardiomyopathy worldwide.
Brad Loncar, founder of the biological fund company Loncar Investments, told CBN reporters: “The publication of the first clinical data of in-person gene editing therapy is a milestone event, which opens up unlimited opportunities for future genetic disease treatments. imagination.”
Longka told the CBN reporter that there are already some gene therapies on the market, such as Onpattro, a multiple neurological disease therapy for patients with ATTR amyloidosis, but these therapies must be injected every three weeks. “If a one-time treatment is born, the market prospects will be great.” He said.
The RNA therapy Onpattro was developed by the biotechnology company Alnylam, and its sales revenue in 2020 will exceed 300 million U.S. dollars. Other RNA therapies for the treatment of polyneuropathy include Pfizer’s approved cardiomyopathy therapy Vyndaqel, and biotechnology company Lonis’ RNA therapy Tegsedi. Longka believes that if Intellia and Regeneron’s new therapy can finally prove effective, it will be disruptive to the market.
“Intellia hopes to provide treatments for all forms of genetic diseases. We will have a dialogue with regulatory agencies in the near future to understand how to promote drug research and development.” Lebvo said, “Gene editing technology is being used more and more. People accept.”
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