To treat HIV, CRISPR gene editing therapy targeting multiple sites enters clinical trials

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Click on the picture and sign up for the conference September 21, 2021/MedClub News/eMedClub News/ --Although antiretroviral therapy provides an effective treatment for human immunodeficiency virus (HIV), there is currently no cure for this disease because the HIV virus is permanently integrated into the host genome and cannot be eliminated by antiviral therapy.

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The discovery of gene editing technology has brought hope for the cure of HIV patients
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CRISPR/Cas9 can make permanent and precise changes to the genome.
In theory, a treatment can be effective for life
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Recently (September 15, 2021), Excision BioTherapeutics announced that the US FDA has approved the IND application for CRISPR-based HIV candidate therapy EBT-101
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Excision plans to launch the first phase 1/2 human clinical trial of EBT-101 later this year to evaluate the safety, tolerability and efficacy of EBT-101 in people infected with human immunodeficiency virus type 1 (HIV-1)
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EBT-101 is a therapeutic drug based on CRISPR gene editing in vivo, designed to remove the proviral DNA that HIV integrates into the human cell genome
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EBT-101 uses AAV9-packaged CRISPR-Cas9 and two gRNAs to target three sites in the HIV genome, thereby removing most of the HIV genome to minimize potential virus escape
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▲ Picture source: Excision's preclinical studies show that EBT-101 has shown the ability to excise HIV proviral DNA in a variety of cell lines, including human primary cells and a variety of animal models including non-human primates
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Since CRISPR gene editing may produce off-target effects, and the gene repair process after the removal of the proviral DNA may also introduce gene mutations, this clinical trial will initially focus on the safety of the therapy and the efficiency of CRISPR to remove the proviral DNA.
Test it
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But in the end, Excision also hopes to observe whether patients will relapse after treatment
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For more details, scan the QR code for a complete read.
In addition, Excision and scientists are also exploring a combination therapy of EBT-101 to improve the immune system's ability to remove infected cells
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Lisa Danzig, Chief Medical Officer of Excision, said: "EBT-101 has demonstrated the removal of proviral DNA in multiple animal models and provides a possible cure for HIV-infected patients
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We look forward to working with major researchers, scientific consultants, and regulators.
This important collaboration between the authors has brought hope to diseases that were previously considered incurable
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” ▲ Lisa Danzig, Chief Medical Officer of Excision (Source: Endpoints) Concluding remarks There are currently many companies developing CRISPR gene editing and base editing technologies in the world.
The pioneers are mainly companies founded by several pioneers of CRISPR technology, and the indications are mostly sickle cell disease and β-thalassemia
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Recommended reading: 8 companies, about the entrepreneurial stories of three CRISPR pioneersFengke analysis.
However, as gene editing therapy companies and technologies continue to mature, including the continuous maturity of the industry, more and more products will be Developed
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The general trend of gene editing therapy development is from simple to complex, from diseases caused by single gene mutations to complex diseases caused by multiple gene mutations or disorders
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It is hoped that in the near future, gene editing technology can bring treatments to epidemic diseases (such as HIV) in a larger patient population
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Recommended reading: The breakthroughs and challenges of CRISPR therapy, the CRISPR market has a bright futureYimai Meng broke the news that CRISPR won the Nobel Prize! A comprehensive inventory of the research directions of gene editing, this article will take you to understand the past and present of the gene magic scissorsThe new observation of medical wheat reference materials: 1.
https:// -ind-for-phase-1-2-trial-of-ebt-101-crispr-based-therapeutic-for-treatment-of-hiv/2.
https://endpts.
com/fda-clears-first-of -its-kind-trial-to-see-if-crispr-gene-editing-can-cure-hiv/3.
Chen Jia Comments | From CRISPR gene editing to base editing, the next round of medical breakthroughs