Platform and Pipeline

Pipeline Introduction

Gritgen provides transformative gene therapies by fundamental understanding of the genetics and underlying biology of diseases. The robust pipeline spans from rare diseases to common diseases.

Research Development Progress

Therapeutic areas

Indication

Discovery

Pre-POC

PCC

IND enabling

Phase I

Pivotal trials

Liver

Hemophilia A

GS1191

GS1191 is the Leading AAV Gene Therapy in China for Hemophilia A. GS1191 has demonstrated excellent safety and sustained bleed control efficacy.

Metabolic disease

GS1168

GS1168 is the First-in-class Dual-mechanism AAV Gene Therapy for target indication. GS1168 has Shown deeper target amino acid reduction with much lower minimum effective dose.

HAE

GS1196

GS1196 is the Best-in-class AAV Gene Therapy for HAE. GS1196 has liver-specific high-efficiency AAV expression cassette caters to high threshold of C1-INH activity required in HAE treatment.

Rare disease

Central nervous system

Common disease

GS6235

Rare disease

Common disease

Others

Common disease

GS5258

Common disease

Liver

Discovery

Pre-POC

PCC

IND enabling

Phase I

Pivotal trials

Hemophilia A

GS1191

GS1191 is the Leading AAV Gene Therapy in China for Hemophilia A. GS1191 has demonstrated excellent safety and sustained bleed control efficacy.

Metabolic disease

GS1168

GS1168 is the First-in-class Dual-mechanism AAV Gene Therapy for target indication. GS1168 has Shown deeper target amino acid reduction with much lower minimum effective dose.

HAE

GS1196

GS1196 is the Best-in-class AAV Gene Therapy for HAE. GS1196 has liver-specific high-efficiency AAV expression cassette caters to high threshold of C1-INH activity required in HAE treatment.

Rare disease

Central nervous system

Discovery

Pre-POC

PCC

IND enabling

Phase I

Pivotal trials

Common disease

GS6235

Rare disease

Common disease