Crispr Can Edit Directly Genes Inside Human Bodies

Calendar September 30, 2022 | Posted by admin

A decade ago, biologists Jennifer Doudna and Emmanuelle Charpentier published a landmark paper describing a natural immune system found in bacteria and its potential as a tool for editing the genes of living organisms. A year later, in 2013, Feng Zhang and his colleagues at the Broad Institute of MIT and Harvard reported that they’d harnessed that system, known as Crispr, to edit human and animal cells in the lab. The work by both teams led to an explosion of interest in using Crispr to treat genetic diseases, as well as a 2020 Nobel Prize for Doudna and Charpentier.

Many diseases arise from gene mutations, so if Crispr could just snip out or replace an abnormal gene, it could in theory correct the disease. But one of the challenges of turning test tube Crispr discoveries into cures for patients has been figuring out how to get the gene-editing components to the place in the body that needs treatment.

One biotech company, Crispr Therapeutics, has gotten around that issue by editing patients’ cells outside the body. Scientists there have used the tool to treat dozens of people with sickle cell anemia and beta thalassemia—two common blood disorders. In those trials, investigators extract patients’ red blood cells, edit them to correct a disease-causing mutation, then infuse them back into the body.

But this “ex vivo” approach has downsides. It’s complex to administer, expensive, and has limited uses. Most diseases occur in cells and tissues that can’t be easily taken out of the body, treated, and put back in. So the next wave of Crispr research is focused on editing “in vivo”—that is, directly inside a patient’s body. Last year, Intellia Therapeutics was the first to demonstrate that this was possible for a disease called transthyretin amyloidosis. And last week, the Cambridge, Massachusetts-based biotech company showed in-the-body editing in a second disease.

Source: https://www.intelliatx.com/
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https://www.wired.com/

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The First CRISPR Gene-Editing Drug on the Market by 2023

Calendar July 4, 2022 | Posted by admin

Until recently, CRISPR—the gene-editing technology that won scientists Jennifer Doudna and Emmanuelle Charpentier the 2020 Nobel Prize in chemistry—sounded more like science fiction than medicine; lab-created molecular scissors are used to snip out problematic DNA sections in a patient’s cells to cure them of disease. But soon we could see regulators approve the very first treatment using this gene-editing technology in an effort to combat rare inherited blood disorders that affect millions across the globe.

In a $900 million collaboration, rare disease specialist Vertex and CRISPR Therapeutics developed the therapy, dubbed exa-cel (short for exagamglogene autotemcel). It has already amassed promising evidence that it can help patients with beta thalassemia and sickle cell disease (SCD), both of which are genetic blood diseases that are relatively rare in the U.S. but somewhat more common inherited conditions globally.

Beta thalassemia is characterized by damaged or missing genes that cause the body to produce less hemoglobin (an essential protein that transports oxygen), potentially leading to enlargement of the liver, spleen, or heart, and malformed or brittle bones. It is estimated to afflict 1 in 100,000 people in the world, and regular blood transfusions are necessary to stave off its most serious effects.

While the exact statistics are unknown, SCD is estimated to affect 100,000 people in the U.S. and millions around the world; it is attributed to a defective gene that causes malformed hemoglobin that are stiff, sticky, and sickle-shaped (hence the name) and can thus block healthy blood cells from transporting oxygen around the body.

Exa-cel reportedly slashed the need for blood transfusions or incidence of serious, life-threatening medical events for months to years after patients received the treatment. New and impressive clinical trial results were announced at a major international medical conference in June and bolstered the companies’ prospect of producing the first gene-editing therapy of its kind to reach the broader market and patients.

The drug makers say they intend to submit exa-cel for regulatory approval in the U.S., U.K., and Europe by the end of this year, meaning the drug could receive marketing authorization sometime in 2023 as more and more biopharma companies pursue novel gene therapies.

Source: https://www.fastcompany.com/

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Nkarta and CRISPR Therapeutics Develop Natural Killer Cell Cancer Treatments

Calendar May 13, 2021 | Posted by admin

Biopharmaceutical company CRISPR Therapeutics has entered into a strategic research, development and commercialization partnership with cancer-focused Nkarta. The new collaboration will be geared toward advancing CRISPR/Cas9 gene-edited cell therapies for certain cancers.

In a statement on the collaboration, the companies state “their complementary cell therapy engineering and manufacturing capabilities” will join forces to advance “the development of a novel NK+T product candidate harnessing the synergies of the adaptive and innate immune systems.” Financial details of the agreement were not publicly disclosed.

According to terms of the agreement, both CRISPR Therapeutics and Nkarta plan to jointly develop and commercialize up to two CAR NK cell product candidates. One candidate will target the CD70 tumor antigen, while no specific target has been set for the additional product. Nkarta has obtained a license to CRISPR gene-editing technology under the agreement. This license will allow Nkarta to edit up to five gene targets using “an unlimited number” of the company’s own NK cell therapy products.

Additionally, the two companies will share equally the research and development costs as well as global profits related to the products born from the collaboration. While Nkarta will retain global rights to a product candidate using a CRISPR Therapeutics’ gene editing target but not developed through the collaboration, Nkarta will provide CRISPR Therapeutics milestone payments as well as royalties on all net sales of the non-collaboration product.

There is a three-year exclusivity on the new agreement, according to the announcement of the collaboration. Overall, the exclusivity agreement covers the research, development as well as commercialization of allogeneic, gene-edited, and donor-derived NK cells and NK+T cells. “This collaboration broadens the scope of our efforts in oncology cell therapy, and expands our efforts to discover and develop novel cancer therapies for patients,” according to a statement made by CRISPR Therapeutics’ Chief Executive Officer (CEO), Samarth Kulkarni, Ph.D.

“Uniting the best-in-class gene editing solution and allogeneic T cell therapy expertise of CRISPR with Nkarta’s best-in-class CAR NK cell therapy platform will be a major advantage to advancing the next wave of transformative cancer cell therapies,” said Nkarta’s CEO, Paul J. Hastings, in a statement. Hastings added that the partnership will enable the company to harness CRISPR’s deep knowledge of CD70 biology as well as experience in the clinical development of allogeneic T cell candidates, which may ultimately “deliver innovative treatments to patients that much faster.”

https://www.biospace.com/

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