2020-12-16| Technology

CRISPR Therapeutics Scores Prestigious Grant for Advancing Gene Therapies for HIV

by Tulip Chakraborty
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CRISPR Therapeutics, a transformative biopharmaceutical company, and leader in developing gene-based medicines for critical diseases announced on December 14th, that they have received a grant from the Bill & Melinda Gates Foundation for investigating in vivo gene editing therapies for HIV.

CRISPR is a Nobel Prize-winning technology that enables scientists to precisely edit DNA in an organism, including humans. Due to its fast, effective, and easy to use nature, CRISPR has the potential to impact various facets of our life, starting from disease prevention to human evolution. However, for the same reason, the gene-editing technology has been the focus of several debates and controversies in the past few years.

Tony Ho, M.D., Executive Vice President and Head of Research & Development at CRISPR Therapeutics said, “While we have demonstrated the promise of CRISPR/Cas9 gene-editing ex vivo in sickle cell disease and beta-thalassemia, an in vivo approach to editing hematopoietic stem cells could allow the transformative benefit of CRISPR/Cas9 to reach a broader array of patients, including those in low resource settings that lack sufficient infrastructure for stem cell transplantation. We look forward to working on new therapies that could contribute to the global effort to reduce the burden of HIV”


CRISPR Therapeutics’ Pipeline

CRISPR Therapeutics and Vertex Pharmaceuticals were the first companies to sponsor a human trial of the gene-editing technology CTX001, CRISPR Therapeutics’ candidate for treating sickle cell disease and beta-thalassemia. Doctors use a patient’s cells from their bone marrow and edit the defective gene using CTX001, after which the cells are infused back into the body. The four-year $105 million deal included upfront cash of $75 million by Vertex and a $30 million stake in CRISPR Therapeutics alongside license to up to six gene-editing programs.

At the 62nd American Society of Hematology Annual Meeting & Exposition this year, the companies presented pathbreaking results from two pioneering early-stage clinical studies in sickle cell disease and beta-thalassemia, potentially revolutionizing the field of therapeutics for monogenic disorders.

The company has also partnered with a private company called ViaCyte to develop a treatment for Type 1 diabetes. The duo hopes that gene-editing can protect ViaCyte’s replacement pancreatic stem cells from the body’s immune system. Results from clinical trials are expected in 2021.


Competition in the Market

The use of CRISPR for diagnostic purposes in the pharmaceutical industry has turned the industry on its head. Several CRISPR-based treatments are already in clinical trials and are expected to shed more light on this evolving technique. Multiple pharmaceutical giants including Merck, Excision BioTherapeutics, Editas Medicine, Regeneron, and Vertex Pharmaceuticals among others are investing a ton of money in developing treatments for Duchenne muscular dystrophy, cancer, HIV, and other diseases using CRISPR. As the businesses navigate through these unprecedented times, it needs to be seen how the off-target effects of CRISPR coupled with the rapid advancement of the technology shape the market for future investments.

By T. Chakraborty, Ph.D.



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