PIPELINE

Thousands of patients are affected by monogenic CNS diseases.

Our deep and sustainable pipeline aims to address this devastating need.

We are advancing our extensive AAV gene therapy pipeline to bring cures to those living with monogenic CNS disease, including for rare and large patient populations. Every one of our gene therapy candidates targets the unique, underlying biology of each indication.

GRT: Gene replacement therapy | miRNA: microRNA | shRNA: short hairpin RNA

Program

Indication

Discovery

Preclinical

Phase 1/2

Pivotal

Neurodegenerative Diseases

TSHA-120

GRT

Giant Axonal Neuropathy
Preclinical
TSHA-101

GRT

GM2 Gangliosidosis
Phase 1/2
TSHA-118

GRT

CLN1 Disease
Phase 1/2
TSHA-119

GRT

GM2 AB Variant
Preclinical
TSHA-104

GRT

SURF1-Associated Leigh Syndrome
Preclinical
TSHA-112

miRNA

APBD
Preclinical
TSHA-111-LAFORIN

miRNA

Lafora Disease
Preclinical
TSHA-111-MALIN

miRNA

Lafora Disease
Preclinical
TSHA-113

miRNA

Tauopathies
Discovery
TSHA-115

miRNA

GSDs
Discovery
Undisclosed

GRT/shRNA

Undisclosed
Discovery
Undisclosed

GRT

Undisclosed
Discovery

Neurodevelopmental Disorders

TSHA-102

Regulated GRT

RETT Syndrome
Preclinical
TSHA-106

shRNA

Angelman Syndrome
Preclinical
TSHA-114

GRT

Fragile X Syndrome
Discovery
TSHA-116

shRNA

Prader-Willi Syndrome
Discovery
TSHA-117

Regulated GRT

FOXG1 Syndrome
Discovery
TSHA-107

GRT

Autism Spectrum Disorder
Discovery
TSHA-108

GRT

Inborn Error of Metabolism
Discovery
TSHA-109

GRT

Inherited Metabolism Disorder
Discovery
Undisclosed

GRT

Undisclosed
Discovery
Undisclosed

mini-gene

Undisclosed
Discovery

Genetic Epilepsies

TSHA-103

GRT

SLC6A1 Haploinsufficiency Disorder
Preclinical
TSHA-105

GRT

SLC13A5 Deficiency
Preclinical
TSHA-110

mini-gene

KCNQ2*
Discovery
Undisclosed

mini-gene

Undisclosed
Discovery

⁺Anticipated milestone

*Option rights

GRT: Gene replacement therapy; miRNA: microRNA;  shRNA: short hairpin RNA

TSHA-101

TSHA-101 is being developed for the treatment of GM2 gangliosidosis, a fatal lysosomal storage disease that includes Tay-Sachs disease and Sandhoff disease. We are developing TSHA-101 as a bicistronic HEXBP2A-HEXA transgene packaged into an AAV9 vector under the control of a CAG promoter.

TSHA-102

TSHA-102 is being developed for the treatment of Rett syndrome, one of the most common genetic causes of severe intellectual disability. TSHA-102 is constructed from a neuronal specific promoter, MeP426, coupled with the miniMECP2 transgene, a truncated version of MECP2, and miRNA-Responsive Auto-Regulatory Element, or miRARE, our novel miRNA target panel, packaged in self-complementary AAV9.

TSHA-118

TSHA-118 is being developed for the treatment of CLN1 disease, also known as infantile Batten disease, a rapidly progressing rare lysosomal storage disease with no approved treatment. TSHA-118 is a self-complementary AAV9 viral vector that expresses human codon-optimized CLN1 complementary deoxyribonucleic acid under control of the chicken ß-actin hybrid promoter.

Publications

Gray, SJ. Timing of Gene Therapy Interventions: The Earlier, the Better. Mol. Ther. 2016 Jun; 24(6): 1017-1018

Copyright 2021- Taysha GTx