On July 15, 2021, American Gene Technologies (AGT), a Maryland biotech company, announced that the safety data in its Phase 1 trial of AGT103-T revealed no adverse effect. Based on the data, the FDA’s Data and Safety Monitoring Board (DSMB) voted unanimously to continue AGT’s HIV cure program without modification.

AGT aims to develop genetic medicines through the application of viral vector technology. The AGT103-T pipeline is a cell therapy for treating HIV disease. The therapy is designed to induce durable viral suppression by delivering therapeutic genes to the recipient’s immune cells. The resulting immune cells are expected to survive attack by HIV and durably suppress the virus at undetectable levels without the need for antiretroviral treatment.

AGT has been collaborating with scientists at National Institute of Allergy and Infectious Disease (NIAID) of the National Institutes of Health (NIH). NIAID has helped AGT with characterizing of T cells in the cell therapy and illustrating the mechanism underlying AGT103-T’s therapeutic effect. A paper co-authored by AGT and NIAID researchers on the development of AGT-103-T was published in 2020.

The AGT103-T pipeline is currently in Phase 1 clinical trial, which is to understand the safety of AGT103-T by measuring key biomarkers and exploring surrogate markers of efficacy. The first patient was treated in May 2021. The DSMB voting allows the company to continue the clinical trial with more participants. AGT will also be able to take blood samples from the trial participants to further test the safety and efficacy of the cell therapy.

“Safety represents a critical milestone in the project and gives us additional confidence that HIV can be cured,” said Jeff Galvin, CEO. “This milestone supports AGT’s mission to relieve suffering and premature death from serious human diseases. AGT’s HIV cure program is based on a platform that has the capacity to treat other chronic viral infections, as well as monogenic disorders and cancers…A success in HIV would provide sustained funding to accelerate dozens of therapeutics that are within the scope of AGT’s technology platform.”

AGT now has a patent portfolio with patents and pending applications covering its lentiviral vectors delivering genes and microRNAs and related methods of use in treating HIV, cancer, and phenylketonuria. AGT also received FDA’s orphan-drug designation for its lentivirus based therapy for treating phenylketonuria.

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