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CRISPR gene editing therapy for HIV is heading into human testing after FDA clearance

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BY ANNALEE ARMSTRONG
FIERCE BIOTECH

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. Continue Reading →

Filed under: emerging technologies

U.S. scientists try 1st gene editing in the body

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The Associated Press
CBC.ca

Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person’s DNA to try to cure a disease.

The experiment was done Monday in California on 44-year-old Brian Madeux. Through intravenous (IV), he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.

“It’s kind of humbling” to be the first to test this, said Madeux, who has Hunter syndrome, a metabolic disease. “I’m willing to take that risk. Hopefully it will help me and other people.”

Signs of whether it’s working may come in a month; tests will show for sure in three months.

If it’s successful, it could give a major boost to the fledgling field of gene therapy. Scientists have edited human genes before, altering cells in the lab that are then returned to patients. There also are gene therapies that don’t involve editing DNA.

But these methods can only be used for a few types of diseases. Some give results that may not last. Some others supply a new gene like a spare part, but can’t control where it inserts in the DNA, possibly causing a new problem like cancer.

This time, the gene tinkering is happening in a precise way inside the body. It’s like sending a mini-surgeon along to place the new gene in exactly the right location.

“We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.” Continue Reading →

Filed under: emerging technologies

DIY Gene Editing: Fast, Cheap—and Worrisome

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BY AMY DOCKSER MARCUS
WALL STREET JOURNAL

Kian Sadeghi has postponed homework assignments, sports practice and all the other demands of being a 17-year-old high-school junior for today. On a Saturday afternoon, he is in a lab learning how to use Crispr-Cas9, a gene-editing technique that has electrified scientists around the world—and sparked a widespread debate about its use. Scientific breakthroughs often raise big ethical questions. Moral concerns around the 1996 cloning of Dolly the sheep or the 2000 announcement of a rough draft of the human genome still reverberate today. The public benefits from scientific advances, particularly in improving health. Continue Reading →

Filed under: professional regulation

Gene-edited ‘micropigs’ to be sold as pets at Chinese institute

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BY DAVID CYRANOSKI
NATURE

Cutting-edge gene-editing techniques have produced an unexpected byproduct — tiny pigs that a leading Chinese genomics institute will soon sell as pets.

BGI in Shenzhen, the genomics institute that is famous for a series of high-profile breakthroughs in genomic sequencing, originally created the micropigs as models for human disease, by applying a gene-editing technique to a small breed of pig known as Bama. On 23 September, at the Shenzhen International Biotech Leaders Summit in China, BGI revealed that it would start selling the pigs as pets. The animals weigh about 15 kilograms when mature, or about the same as a medium-sized dog.

At the summit, the institute quoted a price tag of 10,000 yuan (US$1,600) for the micropigs, but that was just to “help us better evaluate the market”, says Yong Li, technical director of BGI’s animal-science platform. In future, customers will be offered pigs with different coat colours and patterns, which BGI says it can also set through gene editing.

With gene editing taking biology by storm, the field’s pioneers say that the application to pets was no big surprise. Some also caution against it. “It’s questionable whether we should impact the life, health and well-being of other animal species on this planet light-heartedly,” says geneticist Jens Boch at the Martin Luther University of Halle-Wittenberg in Germany. Boch helped to develop the gene-editing technique used to create the pigs, which uses enzymes known as TALENs (transcription activator-like effector nucleases) to disable certain genes.

How to regulate the various applications of gene-editing is an open question that scientists are already discussing with agencies across the world. BGI agrees on the need to regulate gene editing in pets as well as in the medical research applications that make up the core of its micropig activities. Any profits from the sale of pets will be invested in this research. “We plan to take orders from customers now and see what the scale of the demand is,” says Li. Continue Reading →

Filed under: emerging technologies

Henrietta Lacks: Family win recognition for immortal cells

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BBC News

Henrietta Lacks, a poor black woman from Maryland, had cells removed from her by doctors when she was being treated for terminal cancer in 1951. Researchers found they were the first human cells that could be grown indefinitely in a laboratory. The historic breakthrough paved the way for countless medical treatments. The story of how an African-American tobacco farmer unwittingly transformed biomedicine was made famous by a 2010 best-seller, The Immortal Life of Henrietta Lacks. ‘Left in the dark’

She was 31 years old when she died of cervical cancer at Baltimore’s Johns Hopkins Hospital. Continue Reading →

Filed under: genomic privacy