According to a Breakthrough attempt where the CRISPR gene editing technology has completed its first systematic administration as medicine to a human body, Intellia therapeutics CEO John Leonard said he hoped gene therapy could be made available to patients “very, very soon”.
He added that the company expects medical devices to undergo standard testing in the next few years, “but we hope it will be available to patients very, very soon.”
CRISPR, or clustered, regularly arranged short palindromic repeats, cuts genomes and cuts DNA effectively to treat genetic diseases.
The latest development, the result of a study between Intellia and a biotech company Regeneron, treats a rare disease after it has been given as an IV infusion. Previously, other uses of CRISPR technology have been limited to ex vivo therapy, or where cells are removed from the body for genetic manipulation in a laboratory and then returned to the body.
“What’s special about it is that we were able to completely inactivate this gene and see this in the clinical impact of the patient, which is a huge step forward in gene editing,” said Leonard.
CRISPR has wide uses, and Leonard said a lot of work is being done to tackle some of the most common diseases and causes of death, such as heart disease and diabetes.
“The challenge is getting into those specific genes that cause disease, so we started in the liver, an area where there are a lot of problems with disease-causing genes, and we have been shown to be very, very successful at doing this reachable.” said Leonard. “After that, there are other tissues that we are tracking, especially the bone marrow, where a long list of blood-borne diseases can be treated.”
A key for CRISPR is targeting diseases that are monogenic or caused by a specific gene, which enables this type of gene-editing therapy to be successful, Leonard said. Other polygenic diseases like cancer or autoimmune diseases are “more difficult to fight,” he added.
A researcher observes the CRISPR / Cas9 process through a stereo microscope at the Max Delbrück Center for Molecular Medicine.
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The new treatment is still in its early stages and no price has been set, but as it develops Leonard believes it will be “very valuable to patients and likely resource-efficient for the health system as a whole”.
“It really comes down to some of the benefits of a one-time dose, which is literally a one-time therapy,” said Leonard. “We anticipate that over time this will generally be very, very beneficial to the economics of this entire area.”
Jennifer Doudna, who was awarded the 2020 Nobel Prize in Chemistry for her work on CRISPR gene editing and is the co-founder of Intellia, recently said the CNBC Evolve Global Summit these costs pose a significant challenge, and in the case of sickle cell anemia, in which CRISPR was successful early on, Treatment can still be $ 2 million.
“That is clearly not a price point that makes this available to most of the people who can benefit from it,” she said. Innovations in the delivery of CRISPR can help cut costs, but Doudna also said medicine needs to figure out how “to scale molecule production so that we can cut costs”.
She told CNBC that as the technology evolved from the publication of her early work to clinical studies showing it was effective in treating disease in less than 10 years, “one of the fastest advances in technology from the basic , initial science to a specific application. “
“This is mainly because the technology comes at a time when there is a huge demand for genome editing and a great deal of knowledge about genomes,” said Doudna.