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.
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.
BY JENNA MCLAUGHLIN AND ZACH DORFMAN
YAHOO NEWS EXCLUSIVE
The Pentagon is advising members of the military not to use consumer DNA kits, saying the information collected by private companies could pose a security risk, according to a memo co-signed by the Defense Department’s top intelligence official.
BY EMILY MULLIN
MEDIUM / ONE-ZERO
Around this time last November, Chinese scientist He Jiankui stunned the world when he revealed the birth of the first known gene-edited babies. Working in relative secrecy, he had used CRISPR to modify human embryos in the lab and then established pregnancies with those embryos. Twin girls with edited genomes were born as a result.
The scientific community’s condemnation of He was harsh and swift. He had edited the germline, making a heritable genetic change. There were safety questions about the effects on the twins, and he had not meant to fix a genetic defect or prevent disease. Instead, he tweaked a gene in an attempt to bestow an uncommon, protective genetic trait: resistance to HIV.
BY ROBERT F SERVICE
SCIENCE MAGAZINE
Synthetic biologists have performed a biochemical switcheroo. They’ve re-engineered a bacterium that normally eats a diet of simple sugars into one that builds its cells by absorbing carbon dioxide (CO2), much like plants. The work could lead to engineered microbes that suck CO2 out of the air and turn it into medicines and other high-value compounds.
BY TIM CUSHING
TECHDIRT
Cops have discovered a new source of useful third-party records: DNA databases. Millions of people have voluntarily handed over personal information to a number of services in exchange for info on medical markers or distant family members.
Investigators are submitting DNA samples from cold cases in hopes of tracking down criminals who’ve managed to evade them for years. It has led to the closing of some cases, which is all agencies need to argue for continued access to DNA samples from millions of users.
BY ANTONIO REGALADO
MIT TECHNOLOGY REVIEW
Anxious couples are approaching fertility doctors in the US with requests for a hotly debated new genetic test being called “23andMe, but on embryos.”
The baby-picking test is being offered by a New Jersey startup company, Genomic Prediction, whose plans we first reported on two years ago.
The company says it can use DNA measurements to predict which embryos from an IVF procedure are least likely to end up with any of 11 different common diseases. In the next few weeks it’s set to release case studies on its first clients.
A private DNA
ancestry database that’s been used by police to catch criminals is a
security risk from which a nation-state could steal DNA data on a
million Americans, according to security researchers. Security flaws in the service, called GEDmatch,
not only risk exposing people’s genetic health information but could
let an adversary such as China or Russia create a powerful biometric
database useful for identifying nearly any American from a DNA sample. GEDMatch,
which crowdsources DNA profiles, was created by genealogy enthusiasts
to let people search for relatives and is run entirely by volunteers. It
shows how a trend toward sharing DNA data online can create privacy
risks affecting everyone, even people who don’t choose to share their
own information. “You
can replace your credit card number, but you can’t replace your
genome,” says Peter Ney, a postdoctoral researcher in computer science
at the University of Washington.
BY JOCELYN KAISER
SCIENCE
The U.S. Department of Justice (DOJ) released new rules yesterday governing when police can use genetic genealogy to track down suspects in serious crimes—the first-ever policy covering how these databases, popular among amateur genealogists, should be used in law enforcement attempts to balance public safety and privacy concerns.
BY DAVID SYRANOSKI
NATURE
A Russian scientist says he is planning to produce gene-edited babies, an act that would make him only the second person known to have done this. It would also fly in the face of the scientific consensus that such experiments should be banned until an international ethical framework has agreed on the circumstances and safety measures that would justify them.
An attempt to contain the populations of the yellow fever mosquito Aedes aegypti in Brazil may have failed. It appears that gene mutations have been transferred to the local population.
The British company Oxitec had released about 450,000 male mosquitoes every week in the city of Jacobina in the Bahia region with official permission over a period of 27 weeks. The experminet was designed to control the infectious diseases dengue, zika and yellow fever.
The gene modification called OX513A in the mosquitoes was designed in such a way that the first descendant generation of the mosquitoes, known as F1, would not reach the adult stage and thus not be able to reproduce.
