AGT’s HIV Cure Cell Therapy Clears First Safety Hurdle with DSMB Nod

American Gene Technologies’ goal to develop a cure for HIV moved one step closer to reality. After infusing the first patient with its cell therapy candidate AGT103-T, the company announced the Data and Safety Monitoring Board unanimously supported the continuation of the clinical study without modification.

AGT Chief Executive Officer Jeff Galvin expressed his excitement in the green light from the board.

“Being able to safely administer the cell therapy moves the company closer to its goal of actually developing a cure for a disease that has claimed the lives of millions of people across the globe. The nod from the DSMB is a significant milestone in the clinical development of the gene therapy,” Galvin said.

AGT’s Gag-specific AGT103-T is a genetically modified cell product made from an individual’s own cells administered to patients with an ex vivo lentiviral vector-based gene therapy. The cells used in the process are collected through a process known as leukapheresis, modified outside the body, and then re-administered into the patient. The therapy is developed over an 11 –day process to increase the number of HIV-specific T-cells resistant to HIV in order to better fight the virus. In preclinical studies, AGT103-T demonstrated the ability to clear HIV and HIV-infected cells. The research was replicated in collaboration with the National Institute of Allergy and Infectious Disease (NIAID). 

AGT103-T is designed to repair depleted CD4 cells or helper T cells. The company believes this cell therapy can ultimately restore the immune system’s ability to remove infected cells from the patient and decrease or eliminate the need for lifelong antiretroviral treatment. AGT researchers expect the number of HIV-specific immune cells protected and returned to the participants will be nearly 2000 times the number that was achieved in previous efforts.

AGT, which is based in Rockville, Maryland, is conducting the first phases of the Phase I RePAIR (Restore Potent Antiviral Immune Responses) trial in the Maryland and Washington, D.C. area, which is known as a “hot spot” for HIV infection. It is estimated that the area has a rate of 46 infections per 100,000 people each year. According to the Maryland State Department of Health, Maryland also ranks fifth among U.S. states in HIV diagnosis rates, with nearly 1,000 new cases in 2018 alone.

When the Phase I trial is completed, AGT should know if the patients who received the cell therapy are functionally cured. That means patients would no longer show symptoms of the disease and will not be able to transmit the virus to others. 

“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. He said the trial’s success will provide a pathway to receive sustained funding that can support the development of dozens of therapeutics within the scope of AGT’s technology platform,” Galvin said.

The RePAIR trial is the company’s first step in clinical testing of cell and gene therapies for HIV, cancer, and rare diseases.

“Our ImmunoTox program has yielded pre-clinical data showing the potential for revolutionizing solid tumor cancer treatment, and our Phenylketonuria gene therapy may be a one-and-done cure for patients suffering from that inherited disease. Success in HIV would provide sustained funding to accelerate,” Galvin said.AGT is manufacturing its gene therapy products for HIV and oncology at its recently-expanded 27,000 square-foot facility in the Maryland life sciences corridor near the National Cancer Institute and Johns Hopkins Shady Grove Campus.