Excision BioTherapeutics
Excision BioTherapeutics is a clinical-stage biotechnology company focused on developing CRISPR-based therapies to cure viral infectious diseases, including HIV, herpes simplex viruses, and hepatitis B. The company is advancing innovative gene editing technologies, with its lead program EBT-101, a first-in-class in vivo CRISPR therapy for HIV. Excision aims to provide curative solutions through cutting-edge research and clinical trials, supported by collaborations and grants such as from the California Institute for Regenerative Medicine. The company's mission is to improve the lives of patients with viral diseases by pioneering gene editing therapies.
Industries
Nr. of Employees
small (1-50)
Excision BioTherapeutics
134 Coolidge Avenue, Watertown, MA 02472, USA
Patents
Gene editing methods and compositions for eliminating risk of JC virus activation and PML (progressive multifocal leukoencephalopathy) during immunosuppressive therapy
US-10279014-B2
View Details
Gene editing methods and compositions for eliminating risk of JC virus activation and PML (progressive multifocal leukoencephalopathy) during immunosuppressive therapy
US-10279014-B2
View DetailsProducts
Systemically delivered in vivo CRISPR therapeutic candidate for HIV
A research-stage, single-infusion in vivo CRISPR therapeutic candidate employing an adeno-associated viral vector to deliver a CRISPR nuclease and multiple guide RNAs designed to excise integrated proviral DNA and evaluate potential for sustained viral suppression after a single administration.
Platform-based preclinical candidates for other persistent viral infections
Research-stage in vivo genome-editing candidates targeting persistent viral DNA in other pathogens (for example, herpesviruses, hepatitis B, and polyomavirus), leveraging the same multiplexed editing and delivery approach.
Systemically delivered in vivo CRISPR therapeutic candidate for HIV
A research-stage, single-infusion in vivo CRISPR therapeutic candidate employing an adeno-associated viral vector to deliver a CRISPR nuclease and multiple guide RNAs designed to excise integrated proviral DNA and evaluate potential for sustained viral suppression after a single administration.
Platform-based preclinical candidates for other persistent viral infections
Research-stage in vivo genome-editing candidates targeting persistent viral DNA in other pathogens (for example, herpesviruses, hepatitis B, and polyomavirus), leveraging the same multiplexed editing and delivery approach.
Services
Design and execution of open-label, multi-center, single-ascending-dose Phase 1/2 studies to evaluate safety, tolerability, biodistribution, pharmacodynamics, and preliminary efficacy of systemically delivered in vivo gene editing therapeutics, including analytical treatment interruption and long-term follow-up.
Execution of transgenic rodent and non-human primate studies, including single-dose administration, necropsy, tissue analyses, and extended safety and tolerability monitoring to support translational development.
In-house bioinformatic design of genome-targeting candidates and off-target prediction compared against the reference human genome to reduce unintended edits and inform experimental validation.
Design and execution of open-label, multi-center, single-ascending-dose Phase 1/2 studies to evaluate safety, tolerability, biodistribution, pharmacodynamics, and preliminary efficacy of systemically delivered in vivo gene editing therapeutics, including analytical treatment interruption and long-term follow-up.
Execution of transgenic rodent and non-human primate studies, including single-dose administration, necropsy, tissue analyses, and extended safety and tolerability monitoring to support translational development.
In-house bioinformatic design of genome-targeting candidates and off-target prediction compared against the reference human genome to reduce unintended edits and inform experimental validation.
Expertise Areas
- In vivo gene editing for persistent viral infections
- AAV-mediated systemic delivery and intravenous dosing
- Multiplexed guide RNA design and genome-targeting strategies
- Preclinical transgenic rodent and non-human primate studies
Key Technologies
- CRISPR-based gene editing nucleases
- Adeno-associated virus (AAV) vector delivery
- Multiplexed guide RNA constructs
- In-silico off-target prediction pipelines
News & Updates
Excision announced positive interim results from its first-in-human trial of EBT-101, a CRISPR-based therapy for HIV, demonstrating safety and detectable levels in blood, with plans for dose escalation.
The company presented data supporting the development of EBT-101 and its potential to cure HIV.
A publication highlighting the development of EBT-101 as a potential cure for HIV.
Excision announced positive interim results from its first-in-human trial of EBT-101, a CRISPR-based therapy for HIV, demonstrating safety and detectable levels in blood, with plans for dose escalation.
The company presented data supporting the development of EBT-101 and its potential to cure HIV.
A publication highlighting the development of EBT-101 as a potential cure for HIV.