FDA First Approved Human Clinical Trials of CRISPR Gene Editing For The Treatment of AIDS

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FDA First Approved Human Clinical Trials of
CRISPR Gene Editing For The Treatment of AIDS

AIDS (acquired immune deficiency syndrome) is a very harmful infectious disease
caused by the human immunodeficiency virus (HIV). The HIV
virus attacks and damages the human immune system severely. Therefore, AIDS patients
often suffer from secondary infections or tumors due to insufficient immunity, which also
makes the mortality rate of AIDS patients extremely high.

According to UNAIDS data, the number of HIV carriers and AIDS patients worldwide has
increased from 34.3 million at the end of 2013 to 38 million at the end of 2018, and the
number is still growing rapidly.

Although the continuous advancement 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.

What's more, as a retrovirus, HIV can integrate its genome into the chromosome of
the host cell. This means that current antiretroviral therapy can only suppress the virus
and cannot completely eliminate the HIV virus in the body. There will be no cure for AIDS.

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

According to Excision, 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.
Patients need to receive treatment for a long time, which will cause serious side effects
and affect patients' quality life. The therapy based on CRISPR gene editing technology is
expected to replace the current antiretroviral therapy and achieve a "complete cure" for
AIDS.

In November 2020, researchers from Temple University and the University of Nebraska
Medical Center published research papers in the journal Nature
Communications titled CRISPR Based Editing of SIV proviral DNA in ART treated
non-human primates. The researchers successfully edited and eliminated the SIV-a
virus closely related to HIV in the genomes of non-human primates, which is an
important step in AIDS research. This breakthrough also makes mankind closer than ever
to the development of a complete cure for AIDS.

This research breakthrough is very close to clinical research, which means that mankind

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has found a new method that is expected to completely eliminate AIDS.

Excision has been authorized by Temple University for clinical research and development.
Today, the FDA has also approved its human clinical trials.

As we all know, CRISPR-Cas9 gene editing can accurately cut DNA double-strands,
resulting in DNA double-strand breaks. Based on this principle, we can use CRISPR-Cas9
to cut the HIV virus genome that has been integrated into the human chromosome.
However, if only one site is cut, they are easy to repair, and it is easy to cause genetic
mutation near the cut site. This therapy uses multiple cuttings to completely smash the
HIV virus genome, thereby achieving complete elimination of the HIV virus.

Previous tests in non-human primates have shown that the CRISPR-Cas9 system,
delivered using an adeno-associated virus (AAV9) vector, can reach tissues where the
virus is widely present, including bone marrow, lymph nodes and spleen, and in particular
CD4+T cells, an important host cell of HIV, to clear the virus.

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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.
Its CRISPR technology is authorized by Nobel Prize winner Jennifer Doudna.

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 novel coronavirus
(SARS-CoV-2).

Excision's R&D pipeline

In addition, Moderna, a vaccine development company known for its 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. It is reported that Moderna's method is different from that of
previous AIDS vaccines. It prevents HIV virus from entering cells by producing broadly
neutralizing antibodies against the virus. This vaccine based on mRNA technology also
brings new hope to the repeatedly failed AIDS vaccine research.

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References:
[1] CRISPR based editing of SIV proviral DNA in ART treated non-human primates
[2] Excision's CRISPR gene editing therapy for HIV is heading into human testing after
FDA clearance
[3] mRNA Therapeutic Areas in Infectious Diseases