Frequently Asked Questions 

The HIV Cure Program

  • American Gene Technologies® has officially completed its Phase 1 clinical trial for AGT103-T and submitted its final report to the FDA. Data about this first-in-human study was published in Frontiers in Medicine, a peer-reviewed scientific journal focused on medical advancement. 
  • No trial participants experienced any serious adverse events. The modified T cells engrafted, expanded after infusion and maintained reactivity to HIV in 100% of the trial participants. There was no rejection of the cells, which were detectable in patients, even without exposure to the virus, until the last measurement at 180 days. There were no observable differences in safety between the low-dose and high-dose gene therapy. 
  • After completing the Phase 1 human trial, we conducted a sponsor-initiated study called “Durable Anti-Retroviral Withdrawal Initiative (DARWIN)” with approval from the Independent Review Board (IRB) and our clinical trial investigators to test the therapy’s efficacy after cessation of patients’ antiretroviral treatment. Despite non-ideal conditions in the DARWIN study, we still measured impressive viral and immunological data in participants in the absence of their usual antiretroviral medication. 100% of the participants showed active immune responses to HIV, and the data showed that more than half of the participants achieved significant viral suppression. 
  • We have submitted the final Phase 1 trial report to the FDA and we are writing an article about our DARWIN study observations, to be submitted to a peer-reviewed journal. Our scientists continue to analyze the data to fully understand the mechanisms underlying these findings. All of these encouraging results give us optimism that we’re at an inflection point in our efforts to cure HIV. 
  • We are now designing the Phase 2 clinical trial protocol and working to identify manufacturers and trial sites. As we review the Phase 1 data with potential collaborators and partners, the response has been consistently enthusiastic to participate in the Phase 2. 
  • The Phase 2 clinical trial is expected to optimize the regimen and then treat 50 to 100 patients to demonstrate efficacy. We expect to discuss the Phase 2 with the FDA and hope to initiate the trial in 2024.
  • While the Phase 1 clinical trial only required six participants, a total of seven participants were infused with AGT103-T and were tracked to complete the trial. Six of those participants elected to enroll in an additional study where their antiretroviral medication was discontinued.
  • The primary endpoint (i.e., main purpose) of the Phase 1 trial was to establish initial safety aspects of AGT103-T. This is the first step in testing any new drug so that doctors know if there are any possible side effects, how common they are, and how to address them. In this case, since AGT103-T is made of the patient’s own T cells (which have been given a defensive anti-HIV gene therapy), it is important to show that these cells can live with the patient long-term without causing harm. Thus far, 100% of the participants experienced no serious adverse safety events, which gives us an optimistic outlook for the future of AGT103-T. Since AGT103-T is a living gene and cell therapy that acts over a long period of time, a good safety profile is critically important to this therapeutic modality.
  • American Gene Technologies developed AGT103, a gene therapy that turns normal CD4 T cells into AGT103-T cells. AGT103-T cells are almost identical to regular T cells, except they are highly resistant to HIV infection. Normally, HIV causes AIDS by depleting the body’s CD4 T cells, but with HIV-resistant AGT103-T cells, the immune system should be able to mount a durable response against HIV instead of simply succumbing to infection.

 

  • AGT103 is a lentivirus vector carrying a specialized combination of anti-HIV genes. When AGT103 modifies CD4 T cells, it protects the cell against CCR5- or CXCR4-tropic strains of HIV and prevents a latently-infected cell from releasing new HIV virus particles. By installing large numbers of HIV-targeting AGT103-T cells, we are creating conditions in which the immune system can fight HIV, but HIV cannot deplete the immune system. We are hopeful that this treatment could potentially reconstitute the immune system and restore its ability to mount vigorous antiviral immune responses, leading to natural HIV control.
  • Clinical trials, especially for first-in-class therapeutics such as AGT103-T, are uncharted territory. There are no guarantees during clinical trials, and accurately predicting the timeline of clinical development is difficult. Clinical trials come with risk, variability, and real-world difficulties.
  • The HIV epidemic has raged for over four decades, and we are aware that for many people, “when” is the most important question. Cases like “The Berlin Patient” show us that HIV can be cured, and now the race is on to develop a safe and scalable method of curing HIV which is suitable for mass use. While we cannot provide an exact answer to the question “when,” we can provide updates on our clinical progress. The Phase 1 trial took approximately two years including all patient recruitment, product manufacturing, release, and observation. We will continue to provide updates as we progress toward a Phase 2 clinical trial and through the rest of the human trials.
  • With more than 37 million people in the world living with HIV/AIDS, American Gene Technologies is working hard to develop a cure.
  • The company is in a clinical trial for a single-infusion gene therapy (AGT103-T) to functionally cure people living with HIV.
  • HIV has been cured before, for example, you may have heard of The Berlin Patient who was cured in 2008. In fact, more recent case studies such as The New York Patient and The Dusseldorf Patient made headlines in 2022 and 2023 as research teams shared the results of their work. In these patients, and others who have been declared cured, HIV levels in the blood remained undetectable even without antiretroviral medication for an extended period of time.
  • For AGT103-T, ultimate success will mean patients’ viral loads are low enough they aren’t contagious, their CD4 T cell counts are stable at a level so they can never develop into AIDS, can’t be reinfected, and they need no further antiretroviral treatments to suppress the virus or protect them from HIV-related health issues. 
  • In our clinical trials, we are tracking the levels of HIV and AGT103-T cells present in the blood, the function of those AGT103-T cells, and the overall health of the patient. In a nutshell, trial participants who exhibit viral suppression, the persistence of AGT103-T cells, and remain healthy would be good candidates for independent researchers to study.

About American Gene Technologies® (AGT™)

  • American Gene Technologies is a pioneering biotech company in Rockville, Maryland, that’s using gene therapy to cure humanity’s most deadly diseases. Founder & CEO Jeff Galvin’s vision: Repair the DNA, the operating system of the cell, with targeted therapy that fixes the cell’s underlying code, thus curing the disease without the toxic side effects typical of conventional therapies.

 

  • Jeff Galvin founded American Gene Technologies in 2008 after National Institutes of Health (NIH) scientist Roscoe Brady, MD, PhD, introduced him to cutting-edge viral vector technologies and gene therapies. 

 

  • Galvin quickly saw their potential and invested nearly everything he had to start the company and create the future he envisioned: a world without disease.
  • Think of the human cell as an organic computer that operates on instructions formed with four nucleotides symbolized by A, C, T, and G. That is how genes are coded. Genes are instructions to the cellular machinery to produce a specific cellular product (an enzyme or protein). These four symbols are similar to the binary symbols “0” and “1” that form the basis of computer software code. Just as the order of zeros and ones in computer software represents a specific digital command, the order of the A, C, T, and G nucleotides in a cell (each gene) represents a specific organic command.

 

  • Gene therapy is so effective at curing diseases because it allows us to “edit” a cell’s operating system (its DNA) to insert or delete commands. It operates directly on cellular mechanisms to produce potent therapeutic effects with high specificity and targeting.

 

  • In other words, we can now develop therapies that correct the underlying cause in the desired cells in the body and make precise, predictable changes to them. Furthermore, gene therapies can be delivered selectively to work in specific cells and tissues using viral vectors.
  • The cost of the American Gene Technologies gene therapy has not yet been established because the therapy is still in development. However, we expect the cost to be similar to “CAR T” therapies currently on the market. Our goal is to create a cure for HIV that is accessible and affordable for all. The first step is to demonstrate a safe, effective, and broadly applicable therapeutic. The next step will be to engineer economic efficiency into the manufacturing process. Whatever the initial price might be, it could decrease over time as we achieve production and distribution efficiencies. New inventions are affected by the same “technology curve” that turned giant expensive mainframe computers into compact, affordable laptop computers and smartphones. In the same way, successive iterations should drive costs down even as the technology improves.
  • American Gene Technologies believes gene therapy will cut the time and cost of developing life-saving therapies. Traditional drug development is expensive, time intensive, and stochastic. Meanwhile, the design-based development of gene therapies allows companies to converge on effective therapeutics quickly and with fewer expenses.

 

  • American Gene Technologies aims to create a technology platform that accelerates the development of life-changing gene therapies. The American Gene™ platform will make it simpler and less expensive for drug developers to create new cures.

 

  • The company works in a continually accelerating, constantly changing competitive landscape, and will always find ways to create tools and technologies that improve and accelerate cures that are better, faster, and cheaper than the treatments that exist today.

Who is Jeff Galvin?

  • Jeff Galvin is the CEO and Founder of American Gene Technologies. He sees the human cell as a biological computer and DNA as its software operating system. Glitches or errors in a cell’s operating system can result in life-threatening diseases. Jeff believes the best way to cure those diseases is by fixing the underlying DNA — literally tweaking the cell’s software code to eliminate the glitch. That vision, and the techniques for repairing a cell’s DNA, continually drive Jeff and American Gene toward more innovation.
  • Jeff Galvin is the CEO and Founder of American Gene Technologies. He earned his BA degree in Economics from Harvard in 1981. He has more than 30 years of business and entrepreneurial experience including founder or executive positions at a variety of Silicon Valley startups. Several of his companies were taken public and/or sold to public companies, including one in the medical-technology arena that was sold to Varian, the leading maker of linear accelerators used in cancer therapy. Following his startup experience, he retired to become an angel investor in real estate and high tech. He came out of retirement to found and fund AGT after meeting Dr. Roscoe Brady at the National Institutes of Health.

Who is on the AGT Leadership Team?

  • Our leadership team has three components: the management team, the board of directors, and professional advisors. You can see all their names and access their bios on our leadership team page. 
  • The management team includes Founder and CEO Jeff Galvin; Chief Science Officer Jeff Boyle, PhD; Chief Medical Officer Marcus A. Conant, MD; Director of HR & Administration Lola Liao; VP of Finance Andrew Miller, CPA; Chief Business Officer Drew Palin, MD; Chief of Staff Karen McCord; Head of Business Development Barry Wells, MD; Director of Marketing Grant Smith; Systems Development and IT Leader Chris Reid.
  • The board of directors includes Jeff Galvin, Jason T. Thompson, Aaron Galvin, and Qi Tang.
  • Our professional advisors include Tommy Thompson and Robert R. Redfield, MD.
  • We also have scientific advisory boards and groups for each of our cure programs, which you can see here.

Media Inquiries

  • We value our relationship with the media, public officials, and accredited investors.  Guided tours are available for special guests. Contact Marketing Director Grant Smith at gsmith@americangene.com for scheduling details.
  • Founder and CEO Jeff Galvin is our chief media spokesperson, but other executive leaders and scientists are also available for interview. Please contact Marketing Director Grant Smith at gsmith@americangene.com to schedule an interview with Jeff or one of AGT’s other subject matter experts.