Excision BioTherapeutics is a private corporation dedicated to creating innovative gene-editing therapies to address the medical needs of patients with life-threatening diseases caused by viral infections. The company's primary focus is on developing gene-editing medicines that can eliminate or disrupt viral genes in human patients. Their ultimate goal is to advance these gene-editing therapies into safe and effective treatments that will significantly enhance the lives of individuals affected by these diseases worldwide.

Tags

Infectious Diseases

Skin and Musculoskeletal Diseases

Nervous System Diseases

AAV based gene therapy

Gene therapy

CRISPR/Cas9

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Infectious Diseases	5
Nervous System Diseases	1
Immune System Diseases	1

Top 5 Drug Type	Count

AAV based gene therapy	3
Gene therapy	1
CRISPR/Cas9	1

Top 5 Target	Count

HSV- ICP0 x HSV- ICP27	1

Drugs associated with Excision BioTherapeutics, Inc.

EBT-101

Target-

Mechanism

Gene transference

Active Org.

Excision BioTherapeutics, Inc.Startup

Originator Org.

University of California, Berkeley [+1]

Active Indication

HIV Infections

Inactive Indication-

Drug Highest PhasePhase 1/2

First Approval Ctry. / Loc.-

First Approval Date-

EBT-104

Target

HSV- ICP0 x HSV- ICP27

Mechanism

HSV- ICP0 inhibitors [+2]

Active Org.

Excision BioTherapeutics, Inc.Startup

Originator Org.

University of California, Berkeley

Active Indication

Keratitis, Herpetic

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date-

EBT-105

Target-

Mechanism

Gene transference

Active Org.

Excision BioTherapeutics, Inc.Startup

Originator Org.

Excision BioTherapeutics, Inc.Startup

Active Indication

Herpes Simplex

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with Excision BioTherapeutics, Inc.

NCT05143307

/ Enrolling by invitationPhase 1

Long-Term Follow-Up Study of HIV-1 Infected Adults Exiting a Parent Study Where EBT-101 Was Administered

Participants who receive EBT-101 in a parent protocol will be eligible to participate in this long-term follow-up (LTFU) study (EBT-101-002).

Start Date11 Jul 2023

Sponsor / Collaborator

Excision BioTherapeutics, Inc.Startup

NCT05144386

/ CompletedPhase 1

A Phase 1/2a, Sequential Cohort, Single Ascending Dose Study of the Safety, Tolerability, Biodistribution, and Pharmacodynamics of EBT 101 in Aviremic HIV-1 Infected Adults on Stable Antiretroviral Therapy

This is a First in Human (FIH) study of EBT-101 administered IV to aviremic HIV-1 infected adults on stable antiretroviral therapy (ART).

Start Date24 Jan 2022

Sponsor / Collaborator

Excision BioTherapeutics, Inc.Startup

100 Clinical Results associated with Excision BioTherapeutics, Inc.

0 Patents (Medical) associated with Excision BioTherapeutics, Inc.

Literatures (Medical) associated with Excision BioTherapeutics, Inc.

01 Sep 2024·Molecular Therapy-Nucleic Acids

Suppression of HSV-1 infection and viral reactivation by CRISPR-Cas9 gene editing in 2D and 3D culture models

Article

Author: Sariyer, Ilker K ; Liao, Shuren ; Donadoni, Martina ; Gordon, Jennifer ; Khalili, Kamel ; Liu, Hong ; Sariyer, Rahsan ; Ruan, Guo-Xiang ; Çakır, Senem ; Bellizzi, Anna

Although our understanding of herpes simplex virus type 1 (HSV-1) biology has been considerably enhanced, developing therapeutic strategies to eliminate HSV-1 in latently infected individuals remains a public health concern. Current antiviral drugs used for the treatment of HSV-1 complications are not specific and do not address latent infection. We recently developed a CRISPR-Cas9-based gene editing platform to specifically target the HSV-1 genome. In this study, we further used 2D Vero cell culture and 3D human induced pluripotent stem cell-derived cerebral organoid (CO) models to assess the effectiveness of our editing constructs targeting viral ICP0 or ICP27 genes. We found that targeting the ICP0 or ICP27 genes with AAV2-CRISPR-Cas9 vectors in Vero cells drastically suppressed HSV-1 replication. In addition, we productively infected COs with HSV-1, characterized the viral replication kinetics, and established a viral latency model. Finally, we discovered that ICP0- or ICP27-targeting AAV2-CRISPR-Cas9 vector significantly reduced viral rebound in the COs that were latently infected with HSV-1. In summary, our results suggest that CRISPR-Cas9 gene editing of HSV-1 is an efficient therapeutic approach to eliminate the latent viral reservoir and treat HSV-1-associated complications.

01 Sep 2024·Molecular Therapy-Methods & Clinical Development

CRISPR-Cas9-mediated genome editing delivered by a single AAV9 vector inhibits HSV-1 reactivation in a latent rabbit keratitis model

Article

Author: Shipley, Mason ; Ruan, Guo-Xiang ; Bellizzi, Anna ; Donadoni, Martina ; Neumann, Donna ; Amrani, Nadia ; Luk, Kevin ; Sariyer, Ilker K ; Gordon, Jennifer ; Khalili, Kamel ; Singh, Pankaj ; Isik, Meltem ; Sariyer, Ilker K.

Herpes simples virus 1 (HSV-1) keratitis is a major cause of blindness globally. During primary infection, HSV-1 travels to the trigeminal ganglia and establishes lifelong latency. Although some treatments can reduce symptom severity and recurrence, there is no cure for HSV-1 keratitis. We used CRISPR-Cas9 to co-target gene sequences encoding two essential HSV-1 proteins, ICP0 and ICP27, as a potential therapy for HSV-1 keratitis. In HSV-1-infected Vero cells, the HSV-1 viral load and titer were significantly reduced by plasmid transfection or AAV2 vector transduction expressing Cas9 nuclease from Staphylococcus aureus (SaCas9) and paired guide RNAs (gRNAs). Off-target assessment showed minimal off-target editing activity from the selected gRNAs. We then tested our CRISPR-Cas9 gene editing approach in a latent rabbit model of HSV-1 keratitis. Corneal scarification with all-in-one AAV8(Y733F)-SaCas9 or AAV9-SaCas9 vector reduced viral shedding by over 50%. Interestingly, intravenous administration of the same AAV9-SaCas9 vector eliminated viral shedding in 92% of treated eyes. In addition, treated trigeminal ganglia showed a reduction in HSV-1 DNA and RNA expression. Our results support the utility of single-dose AAV9 all-in-one CRISPR-Cas9 gene editing as a safe and effective strategy for treating HSV-1 keratitis.

01 Jul 2024·Gene therapy

Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates

Author: Chen, Chen ; Thompson, Elaine E ; Donadoni, Martina ; Kaminski, Rafal ; Xu, Ethan Y ; Zhao, Huaqing ; Simonyan, Vahan ; Sariyer, Rahsan ; Cradick, Thomas J ; Gordon, Jennifer ; Lewis, Mark G ; Smith, Mandy D ; Khalili, Kamel ; Misamore, John ; Caocci, Maurizio ; Sariyer, Ilker K ; Mancuso, Pietro ; Huo, Wenwen ; Liao, Shuren ; Liu, Hong ; Burdo, Tricia H

Abstract: In this study, we demonstrate the safety and utility of CRISPR-Cas9 gene editing technol. for in vivo editing of proviral DNA in ART-treated, virally controlled simian immunodeficiency virus (SIV) infected rhesus macaques, an established model for HIV infection.EBT-001 is an AAV9-based vector delivering SaCas9 and dual guide RNAs designed to target multiple regions of the SIV genome: the viral LTRs, and the Gag gene.The results presented here demonstrate that a single IV inoculation of EBT-001 at each of 3 dose levels (1.4 x 10¹², 1.4 x 10¹³ and 1.4 x 10¹⁴ genome copies/kg) resulted in broad and functional biodistribution of AAV9-EBT-001 to known tissue reservoirs of SIV.No off-target effects or abnormal pathol. were observed, and animals returned to their normal body weight after receiving EBT-001.Importantly, the macaques that received the 2 highest doses of EBT-001 showed improved absolute lymphocyte counts as compared to antiretroviral-treated controls.Taken together, these results demonstrate safety, biodistribution, and in vivo proviral DNA editing following IV administration of EBT-001, supporting the further development of CRISPR-based gene editing as a potential therapeutic approach for HIV in humans.

News (Medical) associated with Excision BioTherapeutics, Inc.

03 Dec 2024

·www.globenewswire.com

Excision BioTherapeutics Announces Oral Presentation at the 5th Genome Editing Therapeutics Summit

SAN FRANCISCO, Dec. 03, 2024 (GLOBE NEWSWIRE) -- Excision BioTherapeutics, Inc. (“Excision”, the “Company”), a biotechnology company developing CRISPR-based therapies to cure serious viral infectious diseases, today announced that Dr. Ryo Takeuchi, Senior Director, Genome Editing, will deliver an oral presentation at the 5th Genome Editing Therapeutics Summit (formerly CRISPR 2.0), which is taking place from December 3-5, 2024, in Boston, MA. Dr. Takeuchi will also be chairing a session at the conference entitled, Developing Robust Nonclinical Safety Packages. Presentation and panel details: Oral presentation: “Leveraging EBT-101 Preclinical & Early Clinical Development for New In Vivo Multiplex Editing Programs”Date and Time: Wednesday, December 4th at 9:30 AM ET Dr. Takeuchi will also chair the following panel discussion: Panel title: Developing Robust Nonclinical Safety PackagesDate and Time: Tuesday, December 3rd at 9:30 AM ET About Excision BioTherapeutics, Inc.Excision BioTherapeutics develops CRISPR-based therapeutics designed to cure viral infectious diseases. The Company is pioneering treatments for herpes simplex virus-1 keratitis (HSV-1 keratitis), hepatitis B virus (HBV), and HIV that target and inactivate the viral DNA. Leveraging the Company's multiplexed gene editing approach, the Excision pipeline builds upon positive safety and tolerability data from a Phase 1/2 trial evaluating a first-generation therapy, EBT-101, in patients with HIV. Excision’s foundational technologies were developed by Dr. Kamel Khalili at Temple University and Dr. Jennifer Doudna at UC Berkeley. For more information, please visit www.excision.bio. Contact:John FrauncesLifeSci Advisors917-355-2395jfraunces@lifesciadvisors.com

Gene Therapy

16 Sep 2024

·www.globenewswire.com

Presentation at the 2024 International HBV Meeting Highlights Antiviral Activity of EBT-107 as a Potential Anti-HBV Therapy

SAN FRANCISCO, Sept. 16, 2024 (GLOBE NEWSWIRE) -- Excision BioTherapeutics, Inc. (“Excision”, the “Company”), a clinical-stage biotechnology company developing CRISPR-based therapies to cure deadly and debilitating viral infections, today announced that it presented data from its program for Hepatitis B virus, EBT-107, at the 2024 International HBV Meeting, that took place September 11-15, 2024, in Chicago, IL. “The in vitro and in vivo data presented at the 2024 International HBV Meeting advance the development of a new approach to treat and cure Hepatitis B virus,” said Daniel Dornbusch, Chief Executive Officer of Excision. “EBT-107, using a nanoparticle-delivered, dual guide CRISPR editing approach, demonstrated substantial suppression of HBV biomarkers in vitro and in vivo. These data support advancement to human clinical trials and are highly encouraging for the CRISPR field. We look forward to bringing this therapy to the millions of patients with HBV around the world.” HBV is the most common blood-borne and liver infection worldwide. Existing antiviral and immunomodulator treatments do not eliminate the source of the infection - covalently closed circular DNA (cccDNA) which enables persistent viral infection. Excision’s lead product candidate for treating HBV infection, EBT-107, is a CRISPR-based dual guide RNA gene therapy aimed toward effectively deactivating the virus and eliminating the source of persistent viral infection. About Excision BioTherapeutics, Inc. Excision BioTherapeutics develops CRISPR-based therapeutics designed to cure viral infectious diseases. The Company is pioneering treatments for herpes simplex virus-1 keratitis (HSV-1 keratitis), hepatitis B virus (HBV), and HIV that targets and inactivates the viral DNA. Leveraging the Company's multiplexed gene editing approach, the Excision pipeline builds upon positive safety and tolerability data from a Phase 1/2 trial evaluating a first-generation therapy, EBT-101, in patients with HIV. Excision’s foundational technologies were developed by Dr. Kamel Khalili at Temple University and Dr. Jennifer Doudna at UC Berkeley. For more information, please visit www.excision.bio. Contact: John FrauncesLifeSci Advisors917-355-2395jfraunces@lifesciadvisors.com

Gene TherapysiRNAClinical Study

09 Sep 2024

·www.globenewswire.com

Excision BioTherapeutics Announces Oral Presentation at the 2024 International HBV Meeting

SAN FRANCISCO, Sept. 09, 2024 (GLOBE NEWSWIRE) -- Excision BioTherapeutics, Inc. (“Excision”, the “Company”), a clinical-stage biotechnology company developing CRISPR-based therapies to cure serious latent viral infectious diseases, today announced that it will present data from its program for Hepatitis B virus, EBT-107, at the 2024 International HBV Meeting, taking place September 11-15, 2024, in Chicago, IL. EBT-107 is a CRISPR-based gene therapy that is being developed as a potential cure for Hepatitis B. EBT-107 uses dual guide RNAs to effectively deactivate the virus and prevent the emergence of escape variants. The details of the presentations are below: Title: Development of a multiplex gene editing therapy with paired guide RNAs for the cure of chronic hepatitis BSession Type: Oral presentationSession Title: Session XI: Therapeutics IIPresenter: Ryo Takeuchi, Excision BioTherapeuticsDate/Time: Saturday, September 14, 2024, 2:30 pm (CDT) About Hepatitis B (HBV) HBV is one of the most prevalent infectious diseases worldwide that lacks curative therapies. While existing antiviral and immunomodulator treatments slow liver damage by reducing viral load, they fail to eliminate covalently closed circular DNA (cccDNA) that enables persistent viral infection. Excision’s lead product candidate for the treatment of HBV infection, EBT-107, uses dual guide RNAs to excise large sections of viral DNA and effectively deactivate the virus. About Excision BioTherapeutics, Inc. Excision BioTherapeutics develops CRISPR-based therapeutics designed to cure viral infectious diseases. The Company is pioneering treatments for herpes simplex virus-1 keratitis (HSV-1 keratitis), hepatitis B virus (HBV), and HIV that targets and inactivates the viral DNA. Leveraging the Company's multiplexed gene editing approach, the Excision pipeline builds upon positive safety and tolerability data from a Phase 1/2 trial evaluating a first-generation therapy, EBT-101, in patients with HIV. Excision’s foundational technologies were developed by Dr. Kamel Khalili at Temple University and Dr. Jennifer Doudna at UC Berkeley. For more information, please visit www.excision.bio. Contact: John FrauncesLifeSci Advisors917-355-2395jfraunces@lifesciadvisors.com

Gene TherapysiRNA

100 Deals associated with Excision BioTherapeutics, Inc.

100 Translational Medicine associated with Excision BioTherapeutics, Inc.

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Pipeline

Pipeline Snapshot as of 25 Apr 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

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Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

EBT-101	HIV Infections More	Phase 1/2
EBT-104 ( HSV- ICP0 x HSV- ICP27 )	Keratitis, Herpetic More	Preclinical
EBT-103	Leukoencephalopathy, Progressive Multifocal More	Preclinical
EBT-107	Hepatitis B More	Preclinical
EBT-105	Herpes Simplex More	Preclinical

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