Global epigenetic regulation and capsid delivery license agreement with Genentech
Click here to view
Careers
Core Values
Diversity, Equity and Inclusion
Open Positions
Employee Benefits
Clinical Trials
Contact
Science
Technology Platforms
Zinc Finger Platform
Delivery Platform
Modular Integrase (MINT) Platform
Therapeutic Applications
Neurology
Oncology
Presentations
Publications
Programs
Pipeline
Clinical Programs
Hemophilia A
Fabry Disease
Clinical Trials
Collaborations & Partners
Patients
Our Commitment
Expanded Access Policy
Bioethics
Patient Stories
Bryan – Fabry Disease
Lori – Fabry Disease
Matt – Fabry Disease
Elizabeth – Fabry Disease
Israel – Hemophilia A
Miles – Hemophilia A
Trevor – Prion Disease
Sharron – Renal Transplant
Patient Focus
Resources
Education
Organizations
Investors & Media
Overview
News Releases
Events
Presentations
ESG
About
Overview
Mission & Vision
Executive Leadership
Board of Directors
Latest News
News Releases
Events
Presentations
Contact
Science
Technology Platforms
Zinc Finger Platform
Delivery Platform
Modular Integrase (MINT) Platform
Therapeutic Applications
Neurology
Oncology
Presentations
Publications
Programs
Pipeline
Clinical Programs
Hemophilia A
Fabry Disease
Clinical Trials
Collaborations & Partners
Patients
Our Commitment
Expanded Access Policy
Bioethics
Patient Stories
Bryan – Fabry Disease
Lori – Fabry Disease
Matt – Fabry Disease
Elizabeth – Fabry Disease
Israel – Hemophilia A
Miles – Hemophilia A
Trevor – Prion Disease
Sharron – Renal Transplant
Patient Focus
Resources
Education
Organizations
Investors & Media
Overview
News Releases
Events
Presentations
ESG
About
Overview
Mission & Vision
Executive Leadership
Board of Directors
Latest News
News Releases
Events
Presentations
Contact
LinkedIn
Twitter
ASGCT 2023
Zinc Finger Transcriptional Regulator Mediated Repression of SCN9A Gene as a Therapeutic Approach for Painful Peripheral Neuropathies
May 17th, 2023
Engineered Zinc Finger Transcriptional Regulators Specifically Reduce Prion Expression and Extend Survival in an Aggressive Prion Disease Model
May 12th, 2023
Engineered Zinc Finger Transcriptional Regulators Enable Robust and Reliable Epigenetic Regulation in the Mouse Brain
May 12th, 2023
A Robust and Flexible Baculovirus-Insect Cell System for AAV Vector Production with Improved Yield, Capsid Ratios and Potency
May 12th, 2023
Overcoming the Effect of Previous Enzyme Replacement Therapy on the Detection of Anti-Transgene Protein Antibodies After Treatment with Gene Therapy
May 12th, 2023
Gene Activation Mediated by Zinc Finger Transcriptional Regulators (ZF-TRs) as a Therapeutic Approach for CNS Disorders
May 12th, 2023
Evolution of Blood-Brain Barrier Penetrant AAV Capsids in Non-Human Primates Using a Multiplexed Transcription Dependent Capsid Engineering Platform
May 12th, 2023
Strategic Formulation Development for AAV Delivered Gene Therapies – a Case Study
May 12th, 2023
Development of a Competitive Ligand-Binding Assay to Detect Neutralizing Antibodies Against Chimeric Antigen Receptor of Regulatory T Cells
May 12th, 2023
Cell-Type Specific Reduction of Prion Expression in Neurons and Astrocytes Using Engineered Zinc Finger Transcriptional Regulators
May 12th, 2023