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 Friday, genetic testing company 23andMe announced that hackers accessed the personal data of 0.1% of customers, or about 14,000 individuals. The company also said that by accessing those accounts, hackers were also able to access “a significant number of files containing profile information about other users’ ancestry.” But 23andMe would not say how many “other users” were impacted by the breach that the company initially disclosed in early October.
As it turns out, there were a lot of “other users” who were victims of this data breach: 6.9 million affected individuals in total.
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.
BY EMILY WALTZ
NATURE
After a decade of fighting for regulatory approval and public acceptance, a biotechnology firm has released genetically engineered mosquitoes into the open air in the United States for the first time. The experiment, launched this week in the Florida Keys — over the objections of some local critics — tests a method for suppressing populations of wild Aedes aegypti mosquitoes, which can carry diseases such as Zika, dengue, chikungunya and yellow fever.
Two scientists have been awarded the 2020 Nobel Prize in Chemistry for developing the tools to edit DNA. Emmanuelle Charpentier and Jennifer Doudna are the first two women to share the prize, which honours their work on the technology of genome editing. Their discovery, known as Crispr-Cas9 “genetic scissors”, is a way of making specific and precise changes to the DNA contained in living cells. They will split the prize money of 10 million krona (£861,200; $1,110,400). Biological chemist Pernilla Wittung-Stafshede, commented: “The ability to cut DNA where you want has revolutionised the life sciences.”
Researchers led by Shan-Lu Liu at the Ohio State University say there is “no credible evidence” of genetic engineering. The virus’s genome has been sequenced, and if it had been altered, we would expect to see signs of inserted gene sequences. But we now know the points that differ from bat viruses are scattered in a fairly random way, just as they would be if the new virus had evolved naturally.
BY MATT DAVIS
BIG THINK
All salamanders are gifted at regeneration, but the axolotl takes this capability to the extreme. In addition to growing back its limbs, axolotl can grow back organs like their eyes and even their brains.
BY JIJI
JAPAN TIMES
An Osaka University team said it has carried out the world’s first transplant of cardiac muscle cells created from iPS cells in a physician-initiated clinical trial.
BY HARMEET KAUR
CNN HEALTH
About 15 years ago, an Amish family in the eastern US was hit by an unexplainable tragedy — one of their children died suddenly while playing and running around. Just a few months later, the same fate befell another one of their children.
