About American Gene Technologies
American Gene Technologies is a clinical-stage cell and gene therapy company focusing on treatments and cures for acquire or inherited diseases and cancer. Operating in Rockville, MD, AGT designs and develops, and tests novel genetic medicines using a gene-delivery platform that accelerates early-stage testing and enables rapid translation to the clinic. The company has an extensive patent portfolio covering gene therapies for HIV disease, Phenylketonuria , and solid tumor forms of epithelial cancer.
For more information on AGT’s Phase 1 clinical trial, visit clinicaltrials.gov .
Fda Approves First Human Trial For Potential Crispr
The first-in-human clinical trial for a candidate treatment for individuals living with human immunodeficiency virus type 1 is starting soon after its maker, Excision BioTherapeutics, today received an Investigational New Drug clearance from the U.S. Food and Drug Administration .
The FDA’s IND approval sets the stage for the very first Phase I/II trial to evaluate EBT-101 as a functional cure for chronic HIV based on the endpoints of safety, tolerability, and efficacy.
EBT-101, an in vivo, CRISPR-based drug that targets HIV proviral DNA, is a unique gene therapy that leverages CRISPR’s viral defense capability against bacteria. It uses an adeno-associated virus to deliver a one-time treatment to functionally cure HIV infections. Preclinical studies show that it can excise HIV proviral DNA in multiple cell lines, including both human primary cells and multiple animal models and non-human primates.
CRISPR is a tool that has the ability to snip out HIV that has managed to coil itself into a patient’s DNA cell. It is HIV’s snake-like characteristic that has made it almost impossible to remove over the past decades.
CRISPR’s “genetic scissors,” which is what Drs Jennifer Doudna and Emmanuelle Charpentier won the Nobel Prize for chemistry in 2020, would prevent the need for patients to take virus suppressant drugs to prevent HIV from developing into AIDS.
An Hiv Vaccine Is One Of The Holy Grails Of Medicine
The antigens being tested in this new trial were successful in an earlier proof of concept trial in February 2021 in which a team of researchers at IAVI and Scripps Research used the vaccine to successfully stimulate the production of rare immune cells needed to start the process of generating antibodies against the fast-mutating HIV virus.
It is very exciting that mRNA technology already is being used to create another vaccine of global importance, says William Schaffner, MD, a professor of medicine in the division of infectious diseases at Vanderbilt University Medical Center in Nashville, Tennessee.
Developing a vaccine against HIV the AIDS virus is one of the holy grails of vaccine science. Numerous past attempts have met with only limited success, says Dr. Schaffner. The public health community is eagerly awaiting the results of these early clinical trials and hoping for success, he adds.
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Gsk Aim For Hiv Cure By 2030
Following its recent approval of an injectable HIV-1 treatment from the National institute for Health and Care Excellence , GlaxoSmithKline has high hopes in developing a cure for the virus in the future.
UK pharmaceutical company GSK is looking to begin human clinical trials of its potential cure for HIV in 2022. The company shared that it is aiming to develop a cure for the disease by 2030.
Our ultimate goal is always a cure of HIV, said Kimberly Smith, head of research and development at GSKs HIV arm ViiV Healthcare. She hoped the company would produce a cure by 2030 if not sooner. The potential treatment may give those who have been infected by the virus a chance to heal completely.
The news follows approval from NICE in support of a long-acting injectable HIV-1 treatment, also developed by GSK, which ensures that treatment for HIV only occurs 6 days a year, instead of 365 days with the use of daily pills. GSK is a science-led global healthcare company, pioneering treatments in the HIV virus that can lead to AIDS. GSK and Shionogi additionally entered into a deal in September 2021 in an attempt to extend the time between injections for HIV patients to three months or longer.
What Is An Hiv And Aids Clinical Trial
HIV and AIDS clinical trials help researchers find better ways to prevent, detect, or treat HIV and AIDS. Every HIV medicine was first studied through clinical trials.
Examples of HIV and AIDS clinical trials include:
- studies of new medicines to prevent or treat HIV and AIDS
- studies of vaccines to prevent or treat HIV
- studies of medicines to treat infections related to HIV and AIDS
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American Gene Technologies Hiv Cure Clinical Trial Enters Critical Phase: Withdrawing Participants From Antiretrovirals
AMERICAN GENE TECHNOLOGIESROCKVILLE, Md.
American Gene Technologies, a clinical-stage biotechnology company, has reached a turning point in its HIV cure clinical trial: it began withdrawing participants from their antiretroviral drugs earlier this month. This clinical protocol, technically known as analytic treatment interruption , may ultimately determine if this gene therapy treatment can cure HIV.
This is the next logical step in the trials progression, explained Dr. Marcus A. Conant, Chief Medical Officer at American Gene Technologies. By withdrawing these patients antiretrovirals we can demonstrate whether our AGT103-T gene therapy will lead to a cure.
In February 2022, the independent Institutional Review Board overseeing the clinical trial approved the company to proceed with the ATI. Seven patients have received AGT103-T so far. None have experienced any serious adverse events, thereby demonstrating the therapys safety. Blood markers in the treated patients have provided a view into how the cell therapy is responding in their bodies. So far, the data has confirmed that the cells are properly engrafting, persisting, and seem to be remaining uninfected while reacting to, and likely fighting HIV the way scientists expected.
Phase 1 Trial Background
Video About American Gene Technologies Work:
How Can One Find An Hiv And Aids Clinical Trial Looking For Volunteer Participants
There are several ways to find an HIV and AIDS clinical trial looking for volunteer participants.
- Use the find a study search feature on ClinicalTrials.gov to find HIV and AIDS studies looking for volunteer participants.
- Call a Clinical Info health information specialist at 1-800-448-0440 or email .
- Join ResearchMatch, which is a free, secure online tool that makes it easier for the public to become involved in clinical trials.
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More Than 37 Million People Around The World Have Hiv
HIV attacks the bodys immune system, and if it goes untreated, it can lead to AIDS .
People with AIDS have badly damaged immune systems, which makes them vulnerable to a number of severe illnesses, called opportunistic infections. If AIDS goes untreated, the survival rate is about three years, according to the Centers for Disease Control and Prevention .
There were approximately 37.7 million people around the world with HIV in 2020, and 680,000 AIDS-related deaths, according to HIV.gov.
It’s estimated that 1.2 million people in the United States have HIV. In 2019, there were an estimated 34,800 new infections, with the highest rates of new diagnoses in the South, according to HIV.gov.
A Possible Functional Cure For Hiv Will Soon Begin Human Trials
A biotech company is set to begin human trials on a groundbreaking new HIV treatment that could offer what they believe is a breakthrough cure for the virus.
The Food and Drug Administration gave approval to Excision Biotherapeutics to use its innovative HIV treatment in Phase I/II human trials, according to a press release from the company. Excision Biotherapeutics specifically utilizes CRISPR gene-editing technology in its research against viruses. The Nobel Prize-winning technology allows for the modification of human DNA and is considered revolutionary for its treatment of many diseases.
Excisions HIV treatment, known as EBT-101, was developed in partnership with researchers at Temple University in Philadelphia and involves cutting out several pieces of the HIV genome, which the company believes will render it incapable of mutating inside the body.
If you just make a single cut, the virus can mutate around it, Excision CEO Daniel Dornbusch told Biopharma publication Fierce Biotech. We make multiple cuts to deactivate the viral genome.
EBT-101 has already proved effective in removing HIV proviral DNA a latent form of the virus that inserts itself into human cells and replicates along with them from both lab-isolated human cells and non-human primates, according to Excision. Researchers believe it, thus, has the potential to functionally cure HIV in humans.
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Nih Launches Clinical Trial Of Three Mrna Hiv Vaccines
Phase 1 study is among first to examine mRNA technology for HIV.
The National Institute of Allergy and Infectious Diseases , part of the National Institutes of Health, has launched a Phase 1 clinical trial evaluating three experimental HIV vaccines based on a messenger RNA platforma technology used in several approved COVID-19 vaccines. NIAID is sponsoring the study, called HVTN 302, and the NIAID-funded HIV Vaccine Trials Network , based at Fred Hutchinson Cancer Research Center in Seattle, is conducting the trial.
Finding an HIV vaccine has proven to be a daunting scientific challenge, said Anthony S. Fauci, M.D. NIAID director. With the success of safe and highly effective COVID-19 vaccines, we have an exciting opportunity to learn whether mRNA technology can achieve similar results against HIV infection.
The specific mRNA sequences contained in the vaccines were designed and developed by investigators at the NIAID-funded Scripps Consortium for HIV/AIDS Vaccine Development at the Scripps Research Institute and the Bill & Melinda Gates Foundation-funded IAVI Neutralizing Antibody Center at Scripps, in collaboration with scientists at Cambridge, Massachusetts-based Moderna, Inc. Moderna manufactured the investigational vaccines through a NIAID-supported contract.
NIHTurning Discovery Into Health®
Acceptance Of An Hiv Functional Cure Trial
Should there be an HIV functional cure trial, the vast majority would consider joining. Therapy safety , advice from healthcare professionals , and credibility of the research institution were the top three factors participants took into account when considering whether to join such a trial. Participants interested in joining the trial were more likely to have been diagnosed with an STI in the past year , and to take advice from healthcare professionals when making such a decision . When deciding whether to participate in an HIV functional cure trial, view and support from family and peers were important for those who ceased to have sex and who had attained lower education level . Credibility of the research institution was important to those who had received higher education . Participating in such a trial is not without concerns. Major concerns included the potential risk of developing AIDS and complications , HIV viral load going up and adverse effects of the therapy . They were concerned about adverse effects on their CD4 level and complications after joining the trial. Treatment-experienced participants were less likely to be concerned about AIDS or complications , whereas participants aged below 30 years , those who had attained post-secondary level education , and who had a CD4 cell count lower than 500 cells/uL , expressed concerns about the risk of developing AIDS or related complications after or during trial participation.
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Hiv Cure Program Releases Initial Clinical Trial Data
Rockville, Maryland, UNITED STATES
ROCKVILLE, Md., July 15, 2021 — American Gene Technologies announced today that the Data and Safety Monitoring Board voted unanimously to continue AGTs HIV cure program without modification, after safety analysis of the participants data revealed no adverse effects from the treatment. AGTs Phase 1 trial of AGT103-T, a new cell and gene therapy for HIV disease, is designed to induce durable viral suppression by delivering therapeutic genes to the recipient’s immune cells. The cells are collected by leukapheresis, modified outside the body, then re-infused. Once infused, the cells are expected to durably suppress the virus at undetectable levels without the need for antiretroviral treatment.
Phase 1 in the Maryland/DC Area
AGTs Phase 1 RePAIR trial for AGT103-T is currently underway at trial sites in the Maryland / Washington, DC area. The recruitment status of the clinical trial, and information on the trial sites can be found on ClinicalTrials.gov under the identifier NCT04561258. The RePAIR trial is the companys first step in clinical testing of cell and gene therapies for HIV, cancer, and rare diseases.
A video accompanying this announcement is available at
About American Gene Technologies
For more information on AGT’s Phase 1 clinical trial, visit clinicaltrials.gov .
What Is A Clinical Trial
A clinical trial is a research study that evaluates new medical approaches in people. These approaches include:
- new medicines or new combinations of medicines
- new medical devices or surgical procedures
- new ways to use an existing medicine or device
- new ways to change behaviors to improve health
Clinical trials are conducted in several phases to determine whether new medical approaches are safe and effective in people. Results from a Phase 1 Trial, Phase 2 Trial, and Phase 3 Trial are used to determine whether a new drug should be approved for sale in the United States. Once a new drug is approved, researchers continue to track its safety in a Phase 4 Trial.
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Mrna Technology Could Be Used To Fight Many Different Diseases
So far, millions of Americans have received a COVID-19 mRNA vaccine. Unlike previously developed vaccines, which use a version of the virus to teach the body to recognize and mount an immune response, the Pfizer-BioNTech and Moderna COVID-19 vaccines work by introducing a piece of mRNA that corresponds to a viral protein. The mRNA enables cells to make the type of protein that will trigger an immune response.
In addition to the current HIV trial, researchers around the world are studying ways that mRNA technology could be used to produce proteins to prevent, treat, or even cure a variety of other diseases.
Moderna is currently developing 24 different mRNA-based vaccines or therapies, according to a January 2021 announcement, and both Moderna and Pfizer have ongoing clinical trials testing the efficacy and safety of mRNA flu vaccines.
Anticipation Of Hiv Functional Cure
Among all participants, the median anticipation score for HIV functional cure was 10 with more than half giving a score of 10, and it did not differ between ART treatment-naïve and experienced participants. Among treatment-experienced MSM, those having been diagnosed with HIV for more than 8 years had higher odds of scoring 10 for their level of anticipation for HIV cure . Those anticipating HIV functional cure were more likely to be of age 50 years or above , and to have a lower awareness of the idea . Higher level of anticipation was associated with the consideration of not needing to take long-term HIV medications following functional cure but negatively with untransmittability of HIV .
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First Clinical Trial Of Crispr
The first clinical trial of a gene-editing therapy for human immunodeficiency virus type 1 infection is now underway, thanks to a major collaborative effort between scientists at the Lewis Katz School of Medicine at Temple University and investigators at Excision BioTherapeutics, Inc. The trial, designed to evaluate the safety, tolerability, and efficacy of EBT-101, a unique gene-editing treatment that could alter the future of HIV therapeutics, marks a major step forward in the effort to find a cure for HIV/AIDS.
This is an unprecedented and exciting time for all, particularly for those who have been searching for solutions to the HIV/AIDS pandemic over the last 40 years, including scientists and health care providers, said Kamel Khalili, PhD, Laura H. Carnell Professor and Chair of the Department of Microbiology, Immunology, and Inflammation, Director of the Center for Neurovirology and Gene Editing, and Director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine. Dr. Khalili is also a cofounder of Excision BioTherapeutics. The trial will take place at multiple locations, but Temple is not one of the sites.
For more information, see ClinicalTrials.gov identifiers NCT05144386 and NCT05143307 .
Awareness Of Hiv Functional Cure
Overall, less than half of the participants were aware of HIV functional cure. Compared with those having no knowledge of it, treatment-experienced MSM were more likely to have heard of HIV functional cure but without detailed knowledge . Awareness was associated with STI history in the past year , and being sexually active . Those who were aware of HIV functional cure did not consider restoration and stabilization of effective immune function an important impact , nor were they concerned about potential adverse effects of therapies for functional cure .
Table 4 Crude and adjusted odds ratio for awareness and anticipation of HIV functional cure and willingness in participating in a trial among MSM
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Perceived Benefits Of Hiv Cure
Perceptions on HIV cure differed between the treatment-experienced and naïve group . The most commonly perceived impact of HIV cure was restoration and stabilization of effective immune function , with lower odds among those on ART compared to ART-naïve participants . About half considered untransmittability as an important impact of curing HIV infection. Lowered risk of AIDS or related morbidity was considered more important in the treatment-naïve group , whereas reduced need for clinic visit was accorded higher importance among treatment-experienced participants . Those who considered immune function stabilization as an important impact of HIV cure had a lower CD4 cell count and were less likely to have recently been engaging in sex . Perceived importance of reduction of clinic visits was associated with being sexually active and a higher CD4 level . Participants having attained post-secondary education level and younger than 40 years of age regarded being free from the risk of AIDS or HIV-related morbidity as an important impact for HIV cure. Older adults did not consider reduced transmittability important . De-labelling was an important impact for those with a higher education level .
Table 2 Participants perceptions of the impacts of HIV cure, perception and attitudes towards HIV functional cure, and consideration about HIV functional cure trial, by treatment status