Caring Cross Announces Promising Clinical Data with Single-Dose anti-HIV CAR-T Cell Therapy

BOSTON, May 12, 2026 /PRNewswire/ -- Today, at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Boston, Massachusetts, Caring Cross announced early clinical data from a first-in-human Phase I/IIa trial of a novel CAR-T cell strategy in people living with HIV.

The findings, presented by principal investigator Dr. Steven Deeks of the University of California, San Francisco (UCSF), provide preliminary data suggesting that anti-HIV CAR-T cell therapy could lead to durable viral control, particularly in those who started HIV treatment early in the course of their infection.

"Although the primary focus of the study was safety, a few participants experienced better-than-expected outcomes following interruption of antiretroviral therapy. We are particularly interested in two individuals who have maintained control of HIV with undetectable viral loads for extended periods."

— Dr. Steven Deeks, Professor of Medicine, UCSF; Clinical Principal Investigator

"While this level of viral control can rarely occur in the absence of intervention, the depth of viral suppression observed after our clinical trial in two consecutive patients in the conditioning arm is especially compelling."

— Dr. Mehrdad Abedi, Professor of Medicine and Director, Alpha Stem Cell Clinic, UC Davis; Clinical Co-Investigator

"This work represents the culmination of years of scientific and clinical effort to develop a therapy that harnesses the body's own immune cells to fight HIV. While the clinical data are encouraging, the vector used to generate the anti-HIV duoCAR-T cells reflects a first-generation design — an important step toward a definitive therapeutic solution. We have already advanced next-generation versions that we expect will further enhance the potency and durability of the anti-HIV response, bringing us closer to a lasting, potentially one-time treatment."

— Dr. Boro Dropulić, Founder and Executive Director, Caring Cross

THE GLOBAL BURDEN OF HIV: WHY A CURE REMAINS ESSENTIAL

HIV continues to be one of the world's most significant public health challenges, with approximately 37 million people living with the virus globally. While antiretroviral therapy (ART) has transformed HIV into a manageable chronic condition for many, it is not curative; lifelong treatment is required to maintain viral suppression and prevent disease progression.

Major challenges persist:

• An estimated 1.3 million new HIV infections occur each year
• More than 700,000 people die annually from HIV-related causes
• In some regions, reductions in funding for HIV programs have contributed to a resurgence in new infections
• Sustained treatment adherence remains difficult in many regions due to cost, limited access, side effects, and stigma

Despite decades of progress, these limitations underscore the urgent need for a durable, scalable solution. Achieving a functional cure – one that enables long-term viral control without continuous therapy – remains a central objective in HIV research.

CLINICAL RESULTS

The Phase I/IIa clinical trial is evaluating the safety and preliminary efficacy of an anti-HIV duoCAR-T therapy in ART-suppressed individuals. The therapy uses a participant's own T cells, which are genetically engineered using Caring Cross's proprietary duoCAR technology and reinfused. To date, nine participants have received therapy in this first-in-human dose-escalation study.

The study had three cohorts. In Cohort 1, no conditioning regimen was used. There were no detectable CAR-T cells in the blood after the infusion and all three participants experienced rapid rebound after stopping antiretroviral drugs.

In the second and third cohorts, participants received mild, non-myeloablative conditioning, followed three days later by the infusion of a low dose of cells (Cohort 2) or a high dose of cells (Cohort 3). Among the six participants in these cohorts, two have maintained undetectable to very low viral loads after stopping ART, one for nearly a year and the other for nearly two years. A third participant had transient control for nearly three months.

Importantly, all three started antiretroviral drugs early in the course of their infection and hence were more likely to respond to any potentially curative intervention.

Several participants who did not achieve sustained control showed a delayed viral rebound compared to typical kinetics, suggesting some degree of immune-mediated suppression even in partial responders.

There were no serious adverse events attributed to the duoCAR-T cell product. One participant had a drop in blood counts related to the bone marrow conditioning regimen.

These findings suggest that early initiation of ART may play an important role in enabling durable viral control following CAR-T therapy, potentially by limiting the size and distribution of the viral reservoir.

These early results provide encouraging evidence that a one-time CAR-T therapy may enable durable immune-mediated control of HIV without ongoing treatment in some individuals. The trial is ongoing. Future studies will explore whether modifications such as adjusting the timing of ART interruption or administering multiple infusions could further improve outcomes. Larger-scale Phase II data will be needed to confirm and extend these findings.

For more information and updates on the clinical trial, visit https://clinicaltrials.gov/study/NCT04648046.

THE RESEARCH TEAM

• Dr. Steven Deeks, Professor of Medicine, UCSF – Principal Investigator and presenting author at ASGCT.
• Dr. Mehrdad Abedi, Professor of Medicine and Director, Alpha Stem Cell Clinic, University of California, Davis (UC Davis) – Patient enrollment and treatment administration. Clinical co-investigator.
• Jane Reese Koc, Cellular Therapy Operations Director, National Center for Regenerative Medicine, Case Western Reserve University – Head of Manufacturing for the duoCAR-T cell product.
• Dr. Boro Dropulić, Founder and Executive Director, Caring Cross – Developer of the duoCAR-T cell therapy platform.

The study was made possible by funding from the California Institute for Regenerative Medicine (CIRM), a state of California Agency that funds regenerative medicine, stem cell, and gene therapy research. (Grant Number CLIN2-12090).

About Caring Cross – Caring Cross is a 501(c)(3) nonprofit dedicated to accelerating the development of and ensuring global access to advanced therapies. To achieve its mission, Caring Cross develops technologies and therapeutic candidates that improve the accessibility and affordability of advanced medicines like CAR-T therapy and stem cell gene therapy. Lentiviral vectors used to manufacture Caring Cross advanced therapy medicinal products are manufactured by Vector BioMed, a public benefit corporation and CDMO specializing in rapid manufacturing solutions to provide the industry with affordable, high-quality GMP lentiviral vectors. For more information on Caring Cross, visit https://caringcross.org. For more information on Vector BioMed, visit https://vectorbiomed.com.

About the University of California, San Francisco (UCSF) – UCSF is exclusively focused on the health sciences and is dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. For more information, visit https://www.ucsf.edu.

About Case Western Reserve University (CWRU) – CWRU is one of the country's leading private research institutions. Located in Cleveland's University Circle, CWRU offers a unique combination of, and intense focus on education and research in a variety of fields, including engineering, law, medicine, nursing, dental medicine, and arts and sciences. U.S. News & World Report consistently ranks Case Western Reserve among the top 50 national universities in the U.S. For more information, visit case.edu.

About the University of California, Davis (UC Davis) – UC Davis is one of the most academically comprehensive universities in the field of cell and gene therapy. We are known for our outstanding cell and gene therapy programs across many areas of medicine focused on curing complex disorders through scientific innovations. For more information, visit https://www.ucdavis.edu.

About the California Institute for Regenerative Medicine (CIRM) – CIRM is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs. Since 2004, Californians have entrusted CIRM with $8.5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state's biotechnology economy, and expand access to transformative treatments. For more information, visit www.cirm.ca.gov.

Media Contact: Kathy Fowler / On The Marc Media / [email protected] / +1 (410) 963-2345

SOURCE Caring Cross