The HIV gene medicines company, Calimmune, announced today that the first patient has begun treatment in a Phase I/II clinical trial designed to determine whether a pioneering genetic medicine approach can help to protect individuals infected with HIV from the effects of the virus. The study, “Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection,” utilizes a gene medicine called Cal-1, developed in the lab of Nobel Laureate Dr. David Baltimore and by Calimmune.
In the study, 12 HIV-positive participants will be infused with their own T cells and stem cells (hematopoietic stem cells, HSC), which have been modified to block the HIV receptor CCR5, and to prevent HIV fusion. The procedure is designed to prevent the virus from entering and damaging protected cells. The dual approach used in the study is designed to reduce the possibility that HIV can develop resistance to the procedure.
The goal of the study is to assess the safety, feasibility, and tolerability of Cal-1 in HIV-infected individuals who have previously been on highly active antiretroviral therapy (HAART) but are not currently taking any antiretroviral agent. In addition to routine clinical and laboratory assessments to monitor general health and HIV infection, the study will monitor the presence of Cal-1 protected cells in various cell types in the blood and lymphoid tissue. Other analyses will monitor the safety of Cal-1. The first patient was treated in the study in late June. Data from this study are expected in 2015.
All participants in the study’s three arms will receive the Cal-1 gene transfer. Participants in two of the three study arms will also receive different doses of a preconditioning drug known as busulfan, which may make the therapy more effective.
“This study is an early but important step in an emerging area of scientific exploration, representing the culmination of more than a decade of research and development,” said Calimmune Chief Executive Officer Louis Breton. “We are optimistic that what we learn from this study may bring us closer to the day when a one-time treatment could provide an alternative to a lifetime of antiretroviral therapy.”
The study has been partially funded by the California Institute for Regenerative Medicine (CIRM). The study will take place at clinical trial sites in Los Angeles and San Francisco, Calif., under the direction of Principal Investigators Ron Mitsayasu, M.D., of UCLA and Jacob P. Lalezari, M.D., of Quest Clinical Research.
For more information, visit www.clinicaltrials.gov.
Calimmune is a clinical-stage HIV gene medicines company focused on developing innovative cell-based therapies for HIV. The company’s stem cell technology was discovered in the labs of Nobel Laureate Dr. David Baltimore (Caltech) and Dr. Irvin Chen (UCLA AIDS Institute). Calimmune is also developing a rich product candidate pipeline to address the needs of different types of individuals at different states of HIV infection and with different levels of treatment experience.
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