Gene editing Following the discovery that CRISPR technology completed its first regular delivery to the human body as a drug, Intellia Therapeutics CEO John Leonard said he hoped to provide gene therapy to patients “very soon.”
“These approaches will undergo standard clinical trials where any drug or gene therapy is studied, so we are in the early stages of this phase,” Leonard said on Acesparks’s “Close Call” on Thursday afternoon.
He said the company expects medical technology to undergo standardized testing in the next few years, “but our hope is that it will be available to patients very soon.”
CRISPR, or short palindromic recurrences with regular intervals, effectively cuts off genomes and DNA to treat genetic diseases.
The latest development, a test result between Intellia and Regeneron Biotechnology, cured a rare disease after it was sent as an IV infusion. Previously, other applications of CRISPR technology were limited to ex vivo therapy or the removal of cells from the body for genetic manipulation in the laboratory and then reintroduced into the body.
“What excites us about this is that we have been able to completely inactivate this gene and have made great strides in the clinical impact of the patient, and therefore in the field of gene editing,” Leonard said.
Consequences of heart disease, diabetes and extensive disease
CRISPR has extensive applications, and Leonard points out that a lot of work is being done to target some of the most common diseases and causes of death, such as heart disease and diabetes.
“The challenge is to get into the specific genes that cause disease, so we started in the liver, where there are a lot of problems with the genes that cause the disease, and we’ve been shown that we can achieve this very, very successfully.” Leonard said. “Then there’s the other tissues we’re looking for, especially the bone marrow, where a long list of blood-borne diseases can be considered.”
Leonard points out that for CRISPR, it is the fight against diseases caused by a monogenic or specific gene that allows this type of gene editing therapy to be successfully performed. Other diseases that are polygenic, such as cancer or autoimmune diseases, “will be harder to deal with,” he added.
The researcher is monitoring the CRISPR / Cas9 process using a stereomicroscope at the Max Delbrueck-Molecular Medical Center.
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The new treatment is still in its infancy and it is not yet priced, but as it develops, Leonard said it is “very valuable to patients and can save resources for the health care system as a whole.”
“Indeed, it depends on some of the advantages of being a single-applied therapy,” Leonard said. “Over time, we think this will be very, very convenient in the economy of this industry as a whole.”
Jennifer Doudna, who won the 2020 Nobel Prize in Chemistry for her work on editing CRISPR genes and is one of the founders of Intellia, recently The value of the Acesparks Evolve Global Summit is that it’s a huge challenge, and treatment for CRISPR’s early-successful case of sickle cell anemia could still cost $ 2 million.
“It’s definitely not a price point that exists for most people who can benefit from this,” he said. Innovations in the delivery of CRISPR may help lower costs, but Dudna said the medical field needs to figure out “how to increase molecular production to reduce costs.”
He describes the evolution of technology to Acesparks in the 10 years since his first work was published, from clinical trials showing its effectiveness in treating diseases: “I think one of the fastest roles is technology from fundamental, elementary science to real application. “
“This is because the technology comes at a time when there is a huge demand for genome editing and a lot of information about genomes,” Dudna said.