New amfAR Grants Advance Innovative Gene Therapy Approaches to Curing HIV
FOR IMMEDIATE RELEASE
New amfAR Grants Advance Innovative Gene Therapy
Approaches to Curing HIV
NEW YORK, July 19, 2023 — amfAR, The Foundation for AIDS Research, has awarded new grants totaling $2.4 million to research teams in the U.S. and Europe developing and testing a range of innovative gene therapy approaches to curing HIV.
Globally, 39 million people are living with HIV, but one-quarter of them–roughly 10 million people–still don’t have access to lifesaving antiretroviral therapy. A cure would save millions of lives and could herald the beginning of the end of the HIV pandemic.
“Gene therapy is a tremendously exciting field with enormous, but as-yet untapped, potential against HIV,” amfAR Chief Executive Officer Kevin Robert Frost said. “This round of grants will advance projects being undertaken by some of the world’s most eminent gene therapy researchers, who have the skills and now, we hope, the resources to make real progress toward an accessible cure for people living with HIV.”
A leading gene therapy researcher at Hannover Medical School, Germany, and a past president of the European Society of Gene and Cell Therapy, Dr. Hildegard Büning was co-principal investigator of a consortium of gene therapy scientists that previously received funding from amfAR to develop a complex, three-pronged attack on the HIV reservoir, the main barrier to a cure.
The three components of the strategy have now been developed and fine-tuned and will be tested in an animal study for which Dr. Büning has been awarded an amfAR grant of $1.3 million. The strategy involves: antibodies capable of neutralizing a broad range of HIV subtypes; CAR stem cells—cells genetically reprogrammed to recognize and attack disease cells—and molecular scissors targeting the virus. At the end of this proof-of-concept study, the researchers will determine the efficacy of each intervention, alone and in combination.
A second grant of $480,000 was awarded to Dr. Hans-Peter Kiem of Fred Hutchinson Cancer Center in Seattle for a project that seeks to develop a “portable gene therapy treatment” within a patient’s body as part of an HIV cure intervention. The five cases of HIV cure documented to date have been achieved by a stem cell transplant introducing donor cells with the CCR5-delta32 mutation, which renders them effectively immune to HIV infection. In an effort to avoid this complex and high-risk procedure, and to design a cure that could be applicable to low-resource countries, Dr. Kiem will test engineered virus-like particles that can deliver gene-editing enzymes directly to the blood, liver, brain, etc., in an animal model in an attempt to disrupt the CCR5 receptor in blood-forming stem cells.
A world-renowned pioneer in stem-cell and gene therapy and in the development of new gene-editing technologies, Dr. Kiem was part of the research consortium co-led by Dr. Büning.
Genetically engineered CAR T cells have been remarkably successful in treating certain types of cancer, but their potential against HIV remains unfulfilled for several reasons: CAR T cells are themselves vulnerable to HIV infection, and, even when they remain uninfected, they cannot achieve broad enough coverage or persist long enough to be effective. Dr. Martin Tolstrup of Aarhus University in Denmark was awarded a grant of $477,000 to attempt to overcome these obstacles using CRISPR gene-editing technology and other strategies.
Dr. Daniel Claiborne of The Wistar Institute in Philadelphia will also attempt to optimize a CAR T cell approach for use against HIV with a grant of $120,000. Dr. Claiborne will engineer a panel of CARs targeting non-traditional portions of the HIV envelope and test them in a mouse model.
amfAR, The Foundation for AIDS Research, is one of the world’s leading nonprofit organizations dedicated to the support of AIDS research, HIV prevention, treatment education, and advocacy. Since 1985, amfAR has invested more than $635 million in its programs and has awarded more than 3,500 grants to research teams worldwide. Learn more at www.amfAR.org
Robert Kessler, Program Communications Manager