VIRAL GENE EDITING PIPELINE

Excision BioTherapeutics' research and development program identifies and evaluates investigational compounds and biologics
that show the potential to advance the treatment of life-threatening diseases in areas of unmet medical need.

HIV CRISPR Cas9/gRNA multiplex biologic (EBT101)

Excision's flagship therapeutic is the HIV CRISPR Cas9/gRNA multiplex biologic (EBT101) for the eradication of latent HIV. It has been demonstrated for the first time in CD4+ cells grown in culture and multiple animal models that integrated HIV-1 can be eradicated without off-targeting effects in a single low concentration dose. Excision is currently completing IND enabling studies with the goal to reach human clinical trials by 2019.


JC Virus CRISPR Cas9/gRNA multiplex biologic (EBT103)

Excision's primary orphan-eligible therapeutic for the treatment of individuals with PML is our JCV CRISPR Cas9/gRNA multiplex biologic (EBT103). It has been shown that gRNA targeting a critical region of JC Virus prevents its activation, resulting in considerable reduction of viral loads in vitro. Excision is currently conducting IND-enabling animal studies with the goal to advance EBT 103 into human clinical trials by 2020.


HSV-1/2 CRISPR Cas9/CasY/gRNA multiplex biologic (EBT104)

EBT 104 is a CRISPR biologic that targets critical regions of HSV-1/2 preventing virion maturation. It has been shown and verified in peer-reviewed publications that disruption of these critical viral elements with EBT104 impedes HSV replication. Excision is currently conducting animal studies with plans to be in the clinic by 2021.


HBV CRISPR CasX/gRNA Duplex biologic (EBT106)

Excision has recently initiated a viral gene editing eradication program that targets Hepatitis B. This program is the first vial program in the industry to utilize a CRISPR gene editor that is not a Cas9 nuclease. Using our ViraSuiteTM platform, Excision scientists have developed a proprietary gRNA targeted approach to eliminate the virus through a knockout-excision strategy.