A CRISPR-Cas9 gene editing technology that has shown promise in clearing HIV from mice is headed into human testing.
Excision BioTherapeutics will usher the CRISPR-based therapy EBT-101 into clinical trials after the FDA cleared an investigational new drug application, according to the company’s press release.
EBT-101 is under development as a potential virus-clearing treatment for patients with HIV—or, put in the company’s words, “a potential functional cure for chronic HIV.”
We don’t like to throw the word “cure” around here. But Excision thinks the therapy could replace standard-of-care retroviral therapy, which keeps HIV from replicating but does not remove it from the body. That means patients stay on the treatment, which can cause serious side effects and affect quality of life. Now with the start of human testing, the real path to see if this new and lauded tech can accomplish this really begins.
HIV integrates its genetic material into the genome of a host cell, meaning available therapies just can’t remove it. A team of scientists at Temple University and the University of Nebraska Medical Center managed to remove the virus completely from mice during preclinical testing using a combination of CRISPR and antiretroviral therapy. They also found no adverse events that could be linked to the therapy in the study, published back in 2019.
“If you just make a single cut, the virus can mutate around it. We make multiple cuts to deactivate the viral genome,” CEO Daniel Dornbusch previously told Fierce Biotech in an interview.
EBT-101 has since been tested in nonhuman primates, which showed it reached every tissue in the body where HIV reservoirs reside.
Excision licensed the therapy from the universities with a goal of moving it into clinical trials. Now, the FDA is on board.
The biotech plans to initiate a phase 1/2 clinical trial later this year, according to the statement.
The technology used by Excision was licensed from the lab of famed CRISPR pioneer Jennifer Doudna. The company is also working on similar treatments for other viruses, including herpes and hepatitis B.
Excision’s news arrives the same week that Intellia announced a gene editing therapy for acute myeloid leukemia had been cleared for human trials by the FDA.
On June 26, 2000, President Bill Clinton announced the completion of the draft of the human genome at a press conference with the two project leads, Francis Collins and J. Craig Venter. A genome is all the genetic information of an organism. Scientists had conceived of the Human Genome Project in the 1980s, and, in the first half of the 1990s, expected it to be an endeavor that would go on for decades. But an unexpected technological revolution of faster computers and better chemistry accelerated the ten-year effort toward the finish line, just as the 20th century came to a close.
Britain’s drugs regulator has approved a groundbreaking treatment for two painful and debilitating lifelong blood disorders, which works by “editing” the gene that causes them.
The Medicines and Healthcare products Regulatory Agency (MHRA) has given the green light for Casgevy to be used to treat sickle cell disease and beta thalassemia.
It is the first medicine licensed anywhere that works by deploying gene editing that uses the “genetic scissors”, known as CRISPR, for which its inventors won the Nobel prize for chemistry.
Doctors have told health services to prepare for a new era of cancer screening after a study found a simple blood test could spot multiple cancer types in patients before they develop clear symptoms.
The Pathfinder study offered the blood test to more than 6,600 adults aged 50 and over, and detected dozens of new cases of disease. Many cancers were at an early stage and nearly three-quarters were forms not routinely screened for.
It is the first time results from the Galleri test, which looks for cancer DNA in the blood, have been returned to patients and their doctors, to guide cancer investigations and any necessary treatment.
The prospect of using the popular genome-editing tool CRISPR to treat a host of diseases in people is moving closer to reality.
Medical applications of CRISPR–Cas9 had a banner year in 2019. The first results trickled in from trials testing the tool in people, and more trials launched. In the coming years, researchers are looking ahead to more sophisticated applications of CRISPR genome editing that could lay the foundation for treating an array of diseases, from blood disorders to hereditary blindness. Continue Reading →
