Neurology Conference Presentations Hub

Welcome to our Neurology Conference Presentations Hub, a dedicated space with access to the presentations Sangamo delivers at industry leading conferences. Learn about our latest preclinical research and results while gaining insights into our wealth of neurology knowledge.

Chronic Neuropathic Pain
Prion
Tauopathies
Synucleinopathies
Amyotrophic Lateral Sclerosis (ALS)
Neurodevelopmental Disorders
Neuromuscular Disorders
Huntington’s Disease
Viral Engineering for CNS Delivery: Intrathecal Delivery
Viral Engineering for CNS Delivery: Intravenous Delivery

Chronic Neuropathic Pain

Publication bioRxiv

Potent and selective repression of SCN9A by engineered zinc finger repressors for the treatment of neuropathic pain
CRL Biotechnology Symposium 2023

Preclinical Development of a Zinc Finger Transcriptional Repressor Targeting the SCN9A Gene as a Novel Therapy for Peripheral Neuropathic Pain
American College of Toxicology 2023

Preclinical Development of a Novel Gene Therapy for the Treatment of Peripheral Neuropathic Pain Through Zinc Finger Repressor- Mediated Repression of the SCN9A Gene
Annual Pain Therapeutics Summit 2023

Zinc finger mediated epigenetic repression of SCN9A gene as a therapeutic approach for painful peripheral neuropathies
ASGCT 2023

Zinc Finger Transcriptional Regulator Mediated Repression of SCN9A Gene as a Therapeutic Approach for Painful Peripheral Neuropathies

Prion

Tauopathies

Synucleinopathies

Amyotrophic Lateral Sclerosis (ALS)

Neurodevelopmental Disorders

Neuromuscular Disorders

Huntington’s Disease

Nature Medicine

Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

Viral Engineering for CNS Delivery: Intrathecal Delivery

Viral Engineering for CNS Delivery: Intravenous Delivery

Sangamo Announces Capsid License Agreement with Lilly to Delivery Genomic Medicines for Diseases of the Central Nervous System

Neurology Conference Presentations Hub

Potent and selective repression of SCN9A by engineered zinc finger repressors for the treatment of neuropathic pain

Preclinical Development of a Zinc Finger Transcriptional Repressor Targeting the SCN9A Gene as a Novel Therapy for Peripheral Neuropathic Pain

Preclinical Development of a Novel Gene Therapy for the Treatment of Peripheral Neuropathic Pain Through Zinc Finger Repressor- Mediated Repression of the SCN9A Gene

Zinc finger mediated epigenetic repression of SCN9A gene as a therapeutic approach for painful peripheral neuropathies

Zinc Finger Transcriptional Regulator Mediated Repression of SCN9A Gene as a Therapeutic Approach for Painful Peripheral Neuropathies

Zinc Finger Repressors mediate widespread PRNP lowering in the nonhuman primate brain and profoundly extend survival in prion disease mice

Single Intravenous Treatment with Zinc Finger Repressor Leads to Brain-wide Reduction of Prion in Nonhuman Primates and Significantly Prolongs Survival in the RML Mouse Model

Epigenetic Regulation of Human Prion Expression as a Potential One-Time Treatment for Prion Disease

Neuron-Specific Expression of Zinc-Finger Repressors Mediate Widespread Prion Reduction in the Brain for the Potential Treatment of Prion Disease

Engineered Zinc Finger Transcriptional Regulators Specifically Reduce Prion Expression and Extend Survival in an Aggressive Prion Disease Model

ZF-transcription factors for Prion Disease

Widespread CNS Tau Knockdown for the Potential Treatment of Alzheimer’s Disease and other Tauopathies

Persistent repression of tau in the brain using engineered zinc finger protein transcription factors

SNCA Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for Parkinson’s Disease

Zinc Finger Protein Transcription Factors Targeting Alpha-synuclein - A Promising Therapeutic Strategy for Parkinson’s Disease

SOD1 Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for SOD1-Mediated ALS

Selective repression of C9ORF72 repeat expansion-containing sense and antisense transcripts for the treatment of ALS

Shank3 Gene Activation Mediated by Zinc Finger Transcriptional Activators (ZFA) as a Therapeutic Approach for Phelan-McDermid Syndrome

Restoration of Normal Gene and Protein Expression in Mouse and Human Disease Models of SCN2A Haploinsufficiency Using Zinc Finger Activators

A Zinc Finger Activator Platform to Restore Normal Gene & Protein Expression in Cellular Models of Dravet Syndrome

UBE3A Gene Activation Mediated by Zinc Finger Activators (ZFAs) as a Therapeutic Approach for Angelman Syndrome

Development of a Robust Zinc Finger Activation Platform for Treatment of Neurological Disorders

Zinc Finger Activators restore normal gene and protein expression in a mouse model of SCN2A haploinsufficiency

Shank3 Gene Activation Mediated by Zinc Finger Activators (ZF-As) as a Therapeutic Approach for Phelan-McDermid Syndrome

PMP22 Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for CMT1A

Zinc Finger Mediated Repression and Replacement of MFN2 Leads to the Rescue of Cellular Disease Phenotype in CMT2A Patient-Derived Cells

Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

Fitness Maturation of Engineered AAV Capsids STAC-102 and STAC-103 Enhances Central Nervous System Transduction after CSF Administration in Cynomolgus Macaques

Engineered AAV Capsids Exhibit Improved Transduction of the Central Nervous System After CSF Administration in Adult Cynomolgus Macaques

Identification and Characterization of STAC-BBB, an Engineered AAV Capsid That Exhibits Widespread Transduction of the Central Nervous System in Cynomolgus Macaques

Development of Blood-Brain Barrier Penetrant AAVs through Receptor-Targeted Capsid Engineering

Process and Formulation Development for a Novel Blood-Brain Barrier Penetrant AAV Capsid

Evolution of Blood-Brain Barrier Penetrant AAV Capsids in Non-Human Primates Using a Multiplexed Transcription Dependent Capsid Engineering Platform