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

Phase 2/3

Neurodegenerative Diseases

TSHA-101

GRT

GM2 Gangliosidosis
Preclinical
TSHA-118***

GRT

CLN1
Preclinical
TSHA-104

GRT

SURF1 Deficiency
Preclinical
TSHA-112

GRT/miRNA

APBD
Discovery
TSHA-111

GRT/miRNA

LaFora
Discovery
TSHA-113

miRNA

Tauopathies
Discovery
TSHA-115

miRNA

GSDs
Discovery

Neurodevelopmental Disorders

TSHA-102

Regulated GRT

RETT Syndrome
Preclinical
TSHA-106

shRNA

Angelman Syndrome
Discovery
TSHA-114

GRT

Fragile X Syndrome
Discovery
TSHA-116

shRNA

Prader-Willi Syndrome
Discovery
TSHA-117

Regulated GRT

FOXG1 Syndrome
Discovery
TSHA-107

GRT

Undisclosed Target
Discovery
TSHA-108

GRT

Undisclosed Target
Discovery
TSHA-109

GRT

Undisclosed Target
Discovery

Genetic Epilepsies

TSHA-103

GRT

SLC6A1
Preclinical
TSHA-105

GRT

SLC13A5
Discovery
TSHA-110

GRT

KCNQ2*
Discovery

*Option Rights

**Taysha has exclusive options to acquire 4 additional development programs from UT Southwestern

***Formerly known as ABO-202

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.

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