Phase 1 Clinical research program for functional cure of HIV with EBT-101, in-vivo gene therapy

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Application Title:

Phase 1 Clinical research program for functional cure of HIV with EBT-101, in-vivo gene therapy

Public Abstract:
Therapeutic Candidate or Device

EBT-101 is a novel genome editing therapeutic targeting integrated HIV-1 genome to achieve sustained virologic reduction to enable functional cure.


EBT-101 is intended to deplete the reservoir of integrated HIV-1 proviral DNA in immune reconstituted virally suppressed HIV-1 positive individuals.

Therapeutic Mechanism

EBT-101 is an in vivo gene therapy product intended to excise the replication-competent proviral HIV from latently infected cells. It will be delivered by an AAV9 vector containing a genomic payload of HIV-1 specific CRISPR Cas9gRNA editing transgene that is delivered through transduction of cells. EBT-101 is a single dose gene therapy product to target excision of replication-competent proviral HIV, and remove latent virus that leads to active infection and could lead to a "functional cure".

Unmet Medical Need

Even in the presence of suppressive ART with undetectable viral load in the plasma, HIV virus persists in the body in the form of integrated proviral DNA in latently infected cells. There is an unmet need to remove this latent reservoir towards functional cure of HIV without the need for chronic ART

Project Objective

Assess safety/biodistribution/excision of EBT-101

Major Proposed Activities

  • Clinical study enrollment and completion of study objectives: safety, biodistribution and excision of latent proviral HIV-1 DNA.
  • Implementation of biomarkers for the assessment of EBT-101 immunogenicity, HIV-1 reservoir persistence, and HIV-1 proviral DNA excision.
  • Completion of EBT-101 manufacturing.
Statement of Benefit to California:
In California there were 132,911 people living with HIV in 2019, including 4,354 who were newly diagnosed that year. HIV infection continues to disproportionately impact marginalized populations. Two experienced HIV centers in California will participate in this study: UCSF (San Francisco) and Ruane Clinical Research Group (Los Angeles). These California based centers recognize the importance of diverse representation of clinical study participants to assess this potentially curative therapy.