Nat Commun: The dawn of a complete cure for AIDS: FDA approves the first clinical trial of CRISPR gene editing to treat AIDS in humans

AIDS (AIDS), the full name is Acquired Immune Deficiency Syndrome, which is a very harmful infectious disease caused by the human immunodeficiency virus (HIV)

According to UNAIDS data, the number of HIV carriers and AIDS patients worldwide has increased from 34.

Although the continuous progress of antiretroviral therapy (ART) has greatly extended the lifespan and prognosis of AIDS patients, it will also bring serious side effects and the emergence of drug resistance

More importantly, as a retrovirus, HIV can integrate its genome into the chromosome of the host cell.

Recently, Excision BioTherapeutics announced that its EBT-101 therapy based on CRISPR gene editing technology has been approved by the US FDA and will begin human clinical trials for the treatment of AIDS

Excision said that the existing standard treatment for AIDS is antiretroviral therapy, which can prevent the replication of HIV virus in the body, but cannot eliminate HIV virus.

In November 2020, researchers from Temple University and the University of Nebraska Medical Center published research papers in the journal Nature Communications

This research breakthrough is very close to clinical research, which means that mankind has found a new method that is expected to completely end the AIDS nightmare

Excision has been authorized by Temple University to conduct clinical research and development.

As we all know, CRISPR-Cas9 gene editing can accurately cut DNA double-strands, resulting in DNA double-strand breaks.

Previous tests in non-human primates have shown that the CRISPR-Cas9 system delivered using adeno-associated virus (AAV9) vectors can reach a wide range of tissues where the bone marrow, lymph nodes, spleen and other viruses are located, especially the important host of the HIV virus.

It is understood that Excision will officially launch Phase 1/2 clinical trials later this year

Excision is committed to curing viral infections through CRISPR gene editing technology.

In addition to CRISPR-based AIDS therapy, the company’s R&D pipeline also includes the use of CRISPR technology to eliminate hepatitis B virus (HBV), herpes simplex virus (HSV), papilloma vacuole virus (JCV), and the current outbreak of new crowns Virus (SARS-CoV-2)

Excision's R&D pipeline

In addition, Moderna, a vaccine development company that has exploded due to mRNA vaccines, also recently announced that it has developed an mRNA-based experimental AIDS vaccine that has been proven to trigger the production of neutralizing antibodies against HIV-like viruses in monkeys.

Original source:

Mancuso, P.