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Excision BioTherapeutics' EBT-101 Granted FDA Fast Track Designation as a Promising CRISPR-Based Gene Therapy for Potentially Curing HIV-1

Friday, July 21, 2023

Excision BioTherapeutics, a clinical-stage biotechnology company dedicated to developing CRISPR-based therapies for viral infectious diseases, has received Fast Track designation from the US Food and Drug Administration (FDA) for its innovative CRISPR-based therapy, EBT-101. This therapy is designed as a dual-cut CRISPR-based gene therapy aimed at treating human immunodeficiency virus type 1 (HIV-1). EBT-101 is currently undergoing Phase 1/2 clinical trials to assess its safety and tolerability in adults aged 18 to 65 years old with HIV-1 who are receiving continuous antiretroviral therapy and have undetectable HIV RNA levels.

Fast Track designation is granted to facilitate the development and expedite the review of potentially significant new drugs for serious conditions with unmet medical needs. Therapies with this designation receive more frequent interactions with the FDA and may qualify for other benefits such as accelerated approval and priority review.

Daniel Dornbusch, Chief Executive Officer of Excision, expressed his satisfaction with the FDA's decision to grant Fast Track designation to EBT-101. This recognition emphasizes the importance of finding a cure for HIV and strengthens Excision's commitment to developing potentially curative therapies for significant medical needs.

HIV-1 affects approximately 38 million people worldwide, including 1.1 million in the US, and many individuals face significant health complications and social stigma associated with the disease. The current standard of care is antiretroviral therapy (ART), which patients must take throughout their lives. Although ART effectively reduces viral loads and the risk of HIV transmission, it cannot eliminate latent HIV in the body, leading to potential medical issues.

EBT-101 represents a unique in vivo CRISPR-based therapeutic designed to cure HIV infection after a single intravenous infusion. The therapy uses an adeno-associated virus (AAV) to deliver CRISPR-Cas9 and dual guide RNAs, allowing for multiplexed in vivo editing that targets three distinct sites within the HIV genome. This approach enables the excision of significant portions of the HIV genome, minimizing potential viral escape.

The ongoing Phase 1/2 trial of EBT-101 is an open-label, multi-center, single ascending dose study involving approximately nine participants with HIV-1 who are on antiretroviral therapy and have undetectable viral loads. The primary objective is to evaluate the safety and tolerability of a single dose of EBT-101. The trial will also conduct assessments of biodistribution, pharmacodynamics, and efficacy. Participants will be evaluated for eligibility for an analytical treatment interruption of their background ART at Week 12 post EBT-101 administration.

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