Image: AGT. The company is developing innovative treatments and says it has a promising treatment for AIDS.

Maryland, August 11th, 2020–US biotechnology firm American Gene Technologies (AGT) on Tuesday received approval from the US Food and Drug Administration (FDA) to start a phase-one clinical trial of a potential cure for the human immunodeficiency virus (HIV), which causes acquired immunodeficiency syndrome (AIDS).

The trial, which will take place in Baltimore, Maryland, and Washington, DC, is expected to begin enrollment in September and will test the safety of AGT103-T, a single-dose gene therapy developed to cure HIV.

The treatment aims to restore CD4 T cells, which help regulate immune responsesand consists of a one-time intravenous infusion that is claimed can restore the patient’s immune system and effectively “cure” AIDS.

In HIV disease, the HIV-specific CD4 T cells are activated soon after infection similar to other viral diseases. However, HIV targets these cells for destruction because this insidious virus binds to the same CD4 molecule that identifies the helper T cell subset,” AGT  explained on its website, noting that the HIV-specific CD4 T cells become compromised after an HIV infection because they fall below the necessary levels required for effective immune function.

By that point, only antiretroviral medication is effective for controlling HIV, and infected individuals who seek treatment begin a daily drug regimen that must be continued without interruption for life,” the firm adds.

The potential cure was developed in collaboration with the US National Institute of Allergy and Infectious Diseases. AGT hopes to report initial findings from the study before the end of the year.

“This is momentous news that we have FDA approval to launch Phase 1 and conduct our first human trials. We are beyond excited to reach this milestone. This brings us closer to our goal of transforming lives with genetic medicines,” AGT Chief Science Officer David Pauza said in the release. “Based on our successful commercial-scale product manufacturing runs and features of the product observed in our labs, this therapy has a high potential to be effective.”

However this claim is not without controversy in the industry, mainly because of the use of the word “cure” at an very early stage of testing for the treatment.

Back in November, 2019 one critic, Derek Lowe told the company to “tone it down”:

There are a lot of things that can go wrong with gene therapy, and there are a lot of things that we don’t yet understand about its applications. AGT knows this; anyone who works in the field knows this. And that’s why I say that using “cure” for what are actually just interesting ideas in preclinical development is just not right.”

There are a lot of very sick people out there; AGT and the rest of the biopharma industry are trying to find new medicines to help them. But no one is well served by setting off “Single-dose cure!” headlines before a treatment has gone into a single human being. To AGT’s management and press office, this message: raising hopes like that is not some sort of PR coup – it’s cruelty.”

Well, now the first stage of human testing has been approved, and many people will be interested in the results.

According to UNAIDS, around 38 million people across the world in 2019 were living with HIV. An estimated 1.7 million people worldwide acquired the virus last year.

On the stock market there was very little excitement yesterday and the American Gene Technology stock was slightly down on the NASDAQ stock market on a day when stocks in general were up. If the results are promising, the company might become a target for a takeover by an industry giant.