Drug information

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VRC01
Other Names
VRC-HIVMAB060-00-AB, VRC-HIVMAB080-00-AB, VRC01LS (long-acting form of VRC01)
Drug Class
Broadly Neutralizing Antibodies
Registry Number
1412901-55-3 (CAS)
Organization:
NIAID Vaccine Research Center (VRC)
Phase of Development

VRC01 is in Phase 2 development as a broadly neutralizing antibody for HIV treatment, and the long-acting variant VRC01LS is in Phase 1/2 development for HIV treatment. VRC01 has also been studied in Phase 2b trials for HIV prevention.

(Compound details obtained from PubChem,1 Science Translational Medicine article,2 Treatment Action Group website,3 and ClinicalTrials.gov4,5)

What is VRC01?What is VRC01?

What is VRC01?

VRC01 is an investigational drug that is being studied as a possible strategy to treat people with HIV. It has also been studied for HIV prevention. VRC01 belongs to a group of HIV drugs called broadly neutralizing antibodies (bNAbs).3-5

A long-acting form of VRC01, called VRC01LS, has also been studied for HIV treatment and prevention.3,6

To learn how investigational drugs are tested during clinical trials, read the HIVinfo What is an Investigational HIV Drug? and HIV and AIDS Clinical Trials fact sheets.

How do broadly neutralizing antibodies work?How do broadly neutralizing antibodies work?

How do broadly neutralizing antibodies work?

Antibodies are proteins that the immune system makes to fight infection. A person with HIV produces specific antibodies against HIV. However, most of these antibodies do not stop HIV from multiplying in the body.7,8

Some people with HIV naturally produce rare types of HIV antibodies called broadly neutralizing antibodies (bNAbs). bNAbs are powerful antibodies that can work against different HIV strains. bNAbs can block HIV from entering healthy cells and activate other immune cells to help destroy infected cells.7,9,10

Researchers are investigating whether giving bNAbs to people with HIV can help them maintain undetectable levels of HIV without the need for daily antiretroviral therapy. Additionally, some bNAbs are being studied because they may be able to reduce the size of the latent HIV reservoir.9,11

Researchers are also trying to find out if giving bNAbs, such as VRC01, to people who do not have HIV can help protect them from getting the virus.4,5 This record discusses the study of VRC01 for both HIV treatment and prevention.

Select clinical trials of VRC01Select clinical trials of VRC01

Select clinical trials of VRC01

VRC01 for HIV treatment

Study Names: IMPAACT 2008; NCT03208231

Phase: 1/2
Status: This study has been completed.
Locations: Botswana, Brazil, Malawi, and Zimbabwe
Purpose: The purpose of this study was to evaluate the safety and efficacy of VRC01 plus antiretroviral therapy (ART) in clearing the latent HIV reservoir in infants who were starting ART.12
Selected Study Results: Results presented at AIDS 2022 showed that there were no safety concerns with VRC01 given subcutaneously in infants. VRC01 added to ART was no more effective than ART alone in reducing viral reservoir size. Researchers noted that baseline resistance to ART and VRC01, as well as low VRC01 trough concentrations in some infants, may have contributed to the reduced efficacy of VRC01.13
Additional Published Material:

Study Names: Tatelo Study; NCT03707977

Phase: 1/2
Status: This study has been completed.
Location: Botswana
Purpose: The purpose of this study was to evaluate the safety and efficacy of VRC01LS and the investigational bNAb 10-1074 in maintaining viral suppression in children who had received early ART treatment.14
Selected Study Results: Results published in Sci Transl Med (2023) and presented at CROI 2022 showed that treatment with VRC01LS and 10-1074 was safe and effective. Among study participants who underwent an analytical treatment interruption of ART, 44% of the participants maintained viral suppression through 24 weeks of bNAb-only treatment. Participants with a longer time on ART and bNAb overlap were more likely to maintain viral suppression along with participants who enrolled earlier in the study and had favorable clinical and reservoir characteristics before starting bNAb treatment.15,16
Additional Published Material:

Study Names: RV 397; NCT02664415

Phase: 2
Status: This study has been completed.
Location: Thailand
Purpose: The purpose of this study was to evaluate the safety of VRC01 and efficacy of VRC01 in preventing viral rebound in participants undergoing an analytical treatment interruption of ART.17
Selected Study Results: Results published in Lancet HIV (2019) showed that infusions of VRC01 given to individuals who had started ART during acute HIV infection were safe, with no serious side effects reported during the study. However, VRC01 did not have a significant impact on the number of participants maintaining viral suppression at 24 weeks after ART interruption.18

Study Names: (1) HVTN 804/HPTN 095; NCT04801758 and (2) HVTN 805/HPTN 093; NCT04860323

Phase: Not available
Status: HVTN 804/HPTN 095 and HVTN 805/HPTN 093 have been completed.
Locations: (1) HVTN 804/HPTN 095 – United States, Brazil, and Peru (2) HVTN 805/HPTN 093 – Sub-Saharan Africa
Purpose: The purpose of these studies was to evaluate the ability of the immune system to control viral load levels during an analytical treatment interruption of ART in participants who received VRC01 or placebo and acquired HIV while enrolled in the antibody-mediated prevention (AMP) studies, HVTN 704/HPTN 085 or HVTN 703/HPTN 081.19,20
Selected Study Results:
HVTN 804/HPTN 095: Early results from the HVTN 804/HPTN 095 trial were presented at HIVR4P 2024. Among 17 participants who got HIV during the HVTN 704/HPTN 085 AMP study, there was no evidence of viral control during analytical treatment interruption of ART. The time from when participants started ART treatment interruption to when they had viral rebound or met criteria for restarting ART was no different for participants who had received VRC01 and participants who had received placebo. One participant had a moderately severe side effect related to ART interruption and experienced symptoms of acute retroviral syndrome or acute HIV.21,22

HVTN 805/HPTN 093: Results from the HVTN 805/HPTN 093 trial were presented at AIDS 2022 and published in J Int AIDS Soc (2025). Eleven African women who got HIV during the HVTN 703/HPTN 081 AMP study completed treament interruption off ART. Two of 11 (18%) women had control of viral load levels for at least 32 weeks off ART. Among the participants who underwent interruption of ART, there was no significant difference in the time to viral rebound or time to meet ART restart criteria between those who had received VRC01 and those who had received placebo. No serious side effects related to ART interruption were reported, and no side effects of moderate severity or greater occurred.23,24

Additional early-phase studies investigating HIV treatment with VRC01 or VRC01LS have been or are being conducted. Some of these studies include:

  • RV 398 (NCT02591420): A Phase 1 study that evaluated the safety and antiviral effects of a single infusion of VRC01 in adults with acute HIV infection who received ART simultaneously or one week later. This study has been completed, and results are available from CROI 2023.25
  • VRC 607/ACTG A5378 (NCT02840474): A Phase 1 trial that evaluated the safety and antiviral effects of the long-acting bNAbs VRC01LS and VRC07-523LS in treatment-naive adults with HIV. This study has been completed and results are available from IAS 2019 and JCI Insight (2025).26
  • IMPAACT P1115 (NCT02140255): A Phase 1/2 trial evaluating the use of early intensive ART regimens to achieve HIV remission in infants. One of the regimens studied will include the use of VRC01 added to ART. This study is currently recruiting participants. Results thus far are available from CROI 2024, Lancet HIV (2024), Lancet HIV (2025), and CROI 2026 (Posters 839 and 840).27


VRC01 for HIV prevention

Study Names: (1) HVTN 704/HPTN 085 AMP Study; NCT02716675 and (2) HVTN 703/HPTN 081 AMP Study; NCT02568215

Phase: 2b
Status: These studies have been completed.
Location: (1) HVTN 704/HPTN 085 – United States, Brazil, Peru, and Switzerland (2) HVTN 703/HPTN 081 – Sub-Saharan Africa
Purpose: The purpose of these studies was to evaluate the safety and efficacy of VRC01 given every 8 weeks in preventing HIV among adults who were at risk of acquiring HIV.4,5,28
Selected Study Results: Results published in the New Engl J Med (2021) showed that VRC01 did not significantly protect participants from getting HIV in either trial. The overall lack of efficacy was due to a low number of VRC01-sensitive virus in the regions where the trials were conducted. When looking specifically at how well VRC01 prevented participants in both trails from getting VRC01-sensitive HIV strains, VRC01 was 75% effective. The number and severity of side effects were similar across treatment groups.28
Additional Published Material:

Additional studies evaluating VRC01 or VRC01LS for HIV prevention have been completed, including the Phase 1 IMPAACT P1112 trial (NCT02256631) that looked at the safety and pharmacokinetics of three bNAbs (VRC01, VRC01LS, and VRC07-523LS) in HIV-exposed infants who were at increased risk of mother-to-child HIV transmission.6 Results to this trial are available from J Infect Dis (2020), J Infect Dis (2021), and J Pediatric Infect Dis Soc (2025).

For more details on the studies listed above, see the Health Professional version of this drug summary.

What side effects might VRC01 cause?What side effects might VRC01 cause?

What side effects might VRC01 cause?

One goal of HIV research is to identify safe new drugs that have fewer side effects. The following side effects were observed in some of the studies of VRC01 listed above.

IMPAACT 2008 (NCT03208231)

In the Phase 1/2 IMPAACT 2008 trial, 61 infants initiating ART enrolled to receive either four subcutaneous doses of VRC01 plus ART or ART only. Ninety percent of infants had local injection-site reactions, all of which were of moderate intensity or lower.12,13

Tatelo Study (NCT03707977)

In this Phase 1/2 trial, 28 children received daily ART plus the bNAbs VRC01LS and 10-1074 given once every 4 weeks for at least 8 weeks. Thereafter, 25 of the 28 children stopped ART and continued dual bNAb therapy for up to 24 weeks. No infusion reactions were reported throughout the study. Five severe side effects occurred in three participants. One of the severe side effects (neutropenia) was possibly related to bNAb treatment. No life-threatening side effects occurred during the study, and no participants discontinued study treatment because of severe side effects.14,16

RV 397 (NCT02664415)

In this Phase 2 study, 14 participants received VRC01 monotherapy and five participants received placebo. Infusion-related side effects occurred with 30.3% of VRC01 infusions and with 37.5% of placebo infusions. Most infusion-related side effects were mild, except for one case of moderate infusion-site bruising and one case of severe hives, both of which occurred in a participant receiving VRC01. The case of hives occurred during the participant’s first dose of VRC01 and led to study withdrawal.17,18

HVTN 704/HPTN 085 (NCT02716675) and HVTN 703/HPTN 081 (NCT02568215)

In these two Phase 2b prevention studies, the number and severity of side effects that occurred during the trials was similar across VRC01 and placebo treatment groups. Also, the percentage of participants experiencing reactogenic side effects to VRC01 infusions was similar across groups.28 (Reactogenic side effects are short-term side effects that occur during or shortly after receiving a vaccine. Examples of reactogenic side effects include injection-site pain or tenderness and fever.)

Because VRC01 is still being studied, information on possible side effects of the drug is not complete. As testing of VRC01 continues, additional information on possible side effects will be gathered.

Where can I get more information about clinical trials studying VRC01?Where can I get more information about clinical trials studying VRC01?

Where can I get more information about clinical trials studying VRC01?

More information about VRC01-related research studies is available from ClinicalTrials.gov. (The ClinicalTrials.gov search can be modified so that you can get results that better match your interests.)

Some clinical trials may be looking for volunteer participants. Your health care provider can help you decide whether participating in a clinical trial is right for you. For more information, visit NIH Clinical Research Trials and You.

ReferencesReferences

References

  1. National Center for Biotechnology Information. PubChem Substance Record for SID 472419986, VRC-01, Source: FDA Global Substance Registration System (GSRS). Accessed March 5, 2026
  2. Lynch RM, Boritz E, Coates EE, et al. Virologic effects of broadly neutralizing antibody VRC01 administration during chronic HIV-1 infection. Sci Trans Med. 2015;7(319):319ra206. doi:10.1126/scitranslmed.aad5752. Accessed March 5, 2026
  3. Treatment Action Group website. Research toward a cure trials. Accessed March 5, 2026
  4. National Institute of Allergy and Infectious Diseases (NIAID). A Phase 2b study to evaluate the safety and efficacy of VRC01 broadly neutralizing monoclonal antibody in reducing acquisition of HIV-1 infection among men and transgender persons who have sex with men. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on March 17, 2016. NLM Identifier: NCT02716675. Accessed March 5, 2026
  5. National Institute of Allergy and Infectious Diseases (NIAID). A Phase 2b study to evaluate the safety and efficacy of VRC01 broadly neutralizing monoclonal antibody in reducing acquisition of HIV-1 infection in women in Sub-Saharan Africa. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on October 1, 2015. NLM Identifier: NCT02568215. Accessed March 5, 2026
  6. National Institute of Allergy and Infectious Diseases (NIAID). Open-label, dose-escalating, Phase I study to determine safety and pharmacokinetic parameters of subcutaneous (SC) VRC01, VRC01LS, and VRC07-523LS, potent anti-HIV neutralizing monoclonal antibodies, in HIV-1-exposed infants. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on October 1, 2014. NLM Identifier: NCT02256631. Accessed March 5, 2026
  7. HIV Vaccine Trials Network (HVTN). Using antibodies for HIV prevention. Accessed March 5, 2026
  8. Snow B. The rise of broadly neutralizing antibodies. AIDS Vaccine Advocacy Coalition (AVAC). Published May 17, 2018. Accessed March 5, 2026
  9. National Institute of Allergy and Infectious Diseases (NIAID). Sustained ART-free HIV remission. Accessed March 5, 2026
  10. National Institute of Allergy and Infectious Diseases (NIAID). Future directions for HIV treatment research. Accessed March 5, 2026
  11. Grobben M, Stuart RA, van Gils MJ. The potential of engineered antibodies for HIV-1 therapy and cure. Curr Opin Virol. 2019;38:70-80. doi:10.1016/j.coviro.2019.07.007. Accessed March 5, 2026
  12. National Institute of Allergy and Infectious Diseases (NIAID). Phase I/II multisite, randomized, controlled study of monoclonal antibody VRC01 with combination antiretroviral therapy to promote clearance of HIV-1-infected cells in infants. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered June 30, 2017. NLM Identifier: NCT03208231. Accessed March 5, 2026
  13. Khaitan A, Lindsey J, Capparelli E, et al. Phase I/II study of monoclonal antibody VRC01 with early antiretroviral therapy to promote clearance of HIV-1 infected cells in infants (IMPAACT 2008). Abstract presented at: International AIDS Conference; Montreal, Canada and Virtual; July 29–August 2, 2022. Abstract OALBB0102. Accessed March 5, 2026
  14. National Institute of Allergy and Infectious Diseases (NIAID). A clinical trial to evaluate the impact of broadly neutralizing antibodies VRC01LS and 10-1074 on maintenance of HIV suppression in a cohort of early-treated children in Botswana (dual bNAb treatment in children). In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). October 12, 2018. NLM Identifier: NCT03707977. Accessed March 5, 2026
  15. Shapiro RL, Maswabi K, Ajibola G, et al. Treatment with broadly neutralizing antibodies in children with HIV in Botswana (The Tatelo Study). Webcast presented at: Conference on Retroviruses and Opportunistic Infections (CROI); February 12-16, 2022; Virtual. Accessed March 5, 2026
  16. Shapiro RL, Ajibola G, Maswabi K, et al. Broadly neutralizing antibody treatment maintained HIV suppression in children with favorable reservoir characteristics in Botswana. Sci Transl Med. 2023;15(703):eadh0004. doi:10.1126/scitranslmed.adh0004. Accessed March 5, 2026
  17. National Institute of Allergy and Infectious Diseases (NIAID). Safety and therapeutic efficacy of the broadly neutralizing HIV-1 specific monoclonal antibody VRC01 during analytic treatment interruption in patients who initiated antiretroviral therapy during early acute HIV infection. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on January 20, 2016. NLM Identifier: NCT02664415. Accessed March 5, 2026
  18. Crowell TA, Colby DJ, Pinyakorn S, et al. VRC01 in acutely treated HIV-infected adults: a randomised, double-blind, placebo-controlled trial. Lancet HIV. 2019;6(5):e297-e306. doi:10.1016/S2352-3018(19)30053-0. Accessed March 5, 2026
  19. HIV Vaccine Trials Network. Antiretroviral analytical treatment interruption (ATI) to assess immunologic and virologic responses in participants who received VRC01 or placebo and became HIV-infected during HVTN 704/HPTN 085. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on March 1, 2021. NLM Identifier: NCT04801758. Accessed March 5, 2026
  20. HIV Vaccine Trials Network. Antiretroviral analytical treatment interruption (ATI) to assess immunologic and virologic responses in participants who initiated ART in early HIV infection after having received VRC01 or placebo in HVTN 703/HPTN 081. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on April 14, 2021. NLM Identifier: NCT04860323. Accessed March 5, 2026
  21. Karuna S, Gallardo-Cartagena J, De La Grecca R, et al. Analytical treatment interruption (ATI) in Peru among MSM, trans & gender non-conforming (GNC) individuals with early ART initiation +/- VRC01 proximate to HIV acquisition: stakeholder engagement & early clinical data. Abstract presented at: HIV Research for Prevention (HIVR4P); October 6-10, 2024; Lima, Peru. Abstract OA0605. Accessed March 5, 2026
  22. Gallardo-Cartagena J. Analytical treatment interruption (ATI) in Peru: stakeholder engagement & early clinical data. Slides presented at: HIV Research for Prevention (HIVR4P); October 6-10, 2024; Lima, Peru. Accessed March 5, 2026
  23. Karuna S, Bar K, DeCamp A, et al. Analytical treatment interruption (ATI) among African women with early ART initiation with or without VRC01 circulating at HIV acquisition: study design and early observations of viral rebound and control. Poster presented at: International AIDS Conference; July 29-August 2, 2022; Montreal, Canada and Virtual. Poster EPLBB08. Accessed March 5, 2026
  24. Karuna S, Laher F, Dadabhai S, et al. Analytical treatment interruption among women with HIV in southern Africa who received VRC01 or placebo in the Antibody Mediated Prevention Study: ATI stakeholder engagement, implementation and early clinical data. J Int AIDS Soc. 2025;28(6):e26495. doi:10.1002/jia2.26495. Accessed March 5, 2026
  25. National Institute of Allergy and Infectious Diseases (NIAID). Safety and virologic effect of a human monoclonal antibody (VRC01) administered intravenously to adults during early acute HIV infection. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on October 28, 2015. NLM Identifier: NCT02591420. Accessed March 5, 2026
  26. National Institute of Allergy and Infectious Diseases (NIAID). VRC 607-ACTG A5378: a phase 1, single dose study of the safety and virologic effect of an HIV-1 specific broadly neutralizing human monoclonal antibody, VRC-HIVMAB080-00-AB (VRC01LS) or VRC-HIVMAB075-00-AB (VRC07-523LS), administered intravenously to HIV-infected adults. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered July 19, 2016. NLM Identifier: NCT02840474. Accessed March 5, 2026
  27. National Institute of Allergy and Infectious Diseases (NIAID). Very early intensive treatment of HIV-infected infants to achieve hiv remission: a Phase I/II proof of concept study. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on May 13, 2014. NLM Identifier: NCT02140255. Accessed March 5, 2026
  28. Corey L, Gilbert PB, Juraska M, et al. Two randomized trials of neutralizing antibodies to prevent HIV-1 acquisition. N Engl J Med. 2021;384(11):1003-1014. doi:10.1056/NEJMoa2031738. Accessed March 5, 2026

Last Reviewed: March 5, 2026