HIV CRISPR Therapy Fast-Tracked by the FDA

This article previously appeared on our sister site, CGTLive.

The FDA has granted Fast Track designation to Excision Biotherapeutics’ CRISPR-based, multiplexed in vivo gene-editing therapy EBT-101 for the potential treatment of human immunodeficiency virus type 1 (HIV-1).1

“We are pleased with the FDA’s decision to grant Fast Track designation to EBT-101. This designation underscores the importance of finding a cure for people living with HIV and bolsters Excision’s efforts to rapidly develop potentially curative therapies for significant unmet medical needs,” Daniel Dornbusch, chief executive officer, Excision, said in a statement.1

There is no available cure for HIV-1, which affects 38 million people worldwide, including 1.1 million in the United States and lifelong antiretroviral therapy (ART) is the current standard of care. EBT-101 is a potentially curative, 1-time treatment which uses an adeno-associated virus (AAV) to deliver CRISPR-Cas9 and dual guide RNAs to target 3 sites in the HIV genome, make 2 cuts in integrated retroviral DNA that remove large portions of the HIV genome, and ultimately prevent viral escape and reproduction.

The therapy is being evaluated in an open-label, multicenter, single ascending dose phase 1/2 trial to evaluate the safety, tolerability, and preliminary efficacy of EBT-101. The trial is enrolling approximately 9 participants aged 18 years to 60 years currently receiving ART with an undetectable viral load to receive 1 of 3 dose levels. Investigators will also assess biodistribution and pharmacodynamics.

At week 12 after administration, all participants will be assessed for eligibility for an analytical treatment interruption (ATI) of their background ART. All participants will be enrolled into a long-term follow up protocol following the initial 48-week follow up period.The trial dosed its first patient with EBT-101 in September 2022 and the study has an expected completion date of March 2025.2

READ MORE: WHO Updates Recommendations for HIV, Hepatitis, STI Interventions in Prisons

“Having spoken to some clinicians, they realized that only a handful of people in the world have been cured of HIV ever, so they understand the transformative nature of the therapy. And we believe that the trial will provide important information on the path to a potential functional cure for people living with HIV and meets an area of high unmet medical need. It’s really exciting that we have this groundbreaking therapy for infectious diseases. This is the first time it’s been administered to a participant, and I think it is really exciting to be moving forward,” TJ Cradick, chief scientific officer, Excision BioTherapeutics, told CGTLive™.

Excision is not the only company making strides in search of a cure for HIV. American Gene Technologies’ AGT103-T autologous T-cell therapy has completed its phase 1 trial and shown positive preliminary results in that trial and a follow-up (NCT04561258, NCT05529342) after withdrawing patients from ART.3 Trailing behind is Caring Cross’s Anti-HIV DuoCAR-T cell therapy (LVgp120duoCAR-T), which is being evaluated in a phase 1/2a clinical trial (NCT04648046) that dosed its first patient with HIV in September 2022.4

1. Excision BioTherapeutics receives FDA Fast Track Designation for EBT-101, a first-in-class CRISPR-based gene therapy candidate to functionally cure HIV-1. News release. Excision Biotherapeutics. July 20, 2023. Accessed July 21, 2023.
2. Excision Biotherapeutics doses first participant in EBT-101 phase 1/2 trial evaluating EBT-101 as a potential cure for HIV. News release. Excision BioTherapeutics, Inc. September 15, 2022. Accessed July 21, 2023.
3. American Gene Technologies’ HIV cure clinical trial enters critical phase: withdrawing participants from antiretrovirals. News release. American Gene Technologies. July 28, 2022.
4. Caring cross announces dosing of first participant in anti-hivduocar-t cell therapy phase 1/2a clinical trial. News release. Caring Cross. October 11, 2022.

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