One-Stop AAV Gene Therapy Drug Development, Manufacturing, and Clinical Research Platform-AAV Service-CRO Service

Genevoyager is dedicated to advancing gene therapy by developing AAV viral vectors. We offer integrated services for gene therapy drug development and efficacy evaluation, with a strong focus on building an AAV-based translational medicine platform. We have established a comprehensive patent portfolio, which includes 16 granted invention patents in China, 10 under substantive examination, 11 PCT applications, and 17 international patents. Our portfolio, anchored by the proprietary One-bac 4.0 system, forms a strong patent foundation for AAV vector development and clinical translation.

Drug Development Platform

Manufacturing and Quality Control System

Genevoyager GMP Quality System

Drug Development Services

Genevoyager is committed to improving patient health by integrating clinical insights and addressing real-world needs. We focus on the clinical application of gene therapy, providing one-stop solutions - from research and validation to manufacturing - to meet the challenges faced by patients and healthcare providers.

Drug Development

Genevoyager offers a well-established one-stop platform for AAV vector design, construction, packaging, and testing, empowering precise targeted therapy for diseases through various gene engineering technologies and administration routes.

Current Clinical Applications of Gene Therapy in Major Human Diseases

Category	Disease
Neurological disorders	Spinal Muscular Atrophy (SMA), Amyotrophic Lateral Sclerosis (ALS), Temporal Lobe Epilepsy (TLE), Frontotemporal Dementia (FTD), Huntington’s Disease (HD), Parkinson’s Disease (PD), Alzheimer’s Disease (AD), Neuronal Ceroid Lipofuscinosis (NCL), Batten Disease, Canavan Disease, Dravet Dyndrome (DS), Giant Axonal Neuropathy (GAN), GM1 Gangliosidosis, GM2 Gangliosidosis, Metachromatic Leukodystrophy (MLD), Multiple System Atrophy (MSA), CLN5-NCL, Spastic Paraplegia 50 (SPG50), etc.
Neuromuscular diseases	Limb-Girdle Muscular Dystrophy (LGMD), Duchenne Muscular Dystrophy (DMD), Charcot-Marie-Tooth (CMT), Becker Muscular Dystrophy (BMD), Friedreich Ataxia (FA), X-Linked Myotubular Myopathy (X-MTM), etc.
Ocular diseases	Leber Congenital Amaurosis (LCA), Leber Hereditary Optic Neuropathy (LHON), X-Linked Retinoschisis (XLRS), X-Linked Retinitis Pigmentosa (XLRP), CNGA3/CNGB3-mutated Achromatopsia (ACHM), Wet Age-related Macular Degeneration (Wet AMD), Dry Age-related Macular Degeneration (Dry AMD, map atrophy), Choroideremia (CHM), Retinitis Pigmentosa (RP), Stargardt Disease (STGD), etc.
Metabolic diseases	Mucopolysaccharidosis (MPS), Gaucher Disease (GD), Fabry Disease (FD), Glycogen Storage Disease (GSD), Aromatic L-Amino Acid Decarboxylase Deficiency (AADCD), Ornithine Transcarbamylase Deficiency (OTCD), Krabbe Disease (KD), Hepatolenticular Degeneration (HLD), Homozygous Familial Hypercholesterolaemia (HoFH), Familial Hypertriglyceridemia (FHTG), Familial Lipoprotein Lipase (LPL) Deficiency, Methylmalonic Acidemia (MMA), Phenylketonuria (PKU), Pompe Disease (PD), etc.
Cardiovascular diseases	Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), Hypertrophic Cardiomyopathy (HCM), Chronic Heart Failure (CHF), Critical Limb Ischemia (CLI), Dilated Cardiomyopathy (DCM), Hereditary Angioedema (HAE), Familial Hypercholesterolemia (FH), Ischemic Cardiomyopathy (ICM), PKP2-related ARVC, etc.
Hematologic diseases	Hemophilia A (HA), Hemophilia B (HB), etc.
Otologic disease	Autosomal Recessive Deafness 9 (DFNB9), etc.
Chronic inflammatory diseases	Osteoarthritis (OA), etc.
Cancer	Gastric Cancer (GC), etc.

Process Highlights

▎High Yield, Cost-Effective

The One-Bac 4.0 system ensures stable AAV yields across various production scales, with initial yields reaching up to 1~5E+15 vg/L and batch yields exceeding 1E+18 vg. Reduced manufacturing costs and higher production capacity facilitate meeting the medical needs of a broad patient population.

▎High Expression Level

Results from in vitro and in vivo experiments demonstrate that One-Bac 4.0-derived rAAV exhibits equivalent or higher expression level than HEK293 rAAV.

▎High Passage Stability

The Baculovirus Expression Vector (BEV) employed in the One-Bac 4.0 system maintains high stability for multiple passages, ensuring consistent high yield and expression level of AAV batches during large-scale production.

▎High Safety Profile

The AAV produced using the One-Bac 4.0 system features high full capsid ratio, minimal impurities, and is free of replication-competent AAV (rcAAV).

CRO Service

One-Stop AAV Gene Therapy Drug Development, Manufacturing, and Clinical Research Platform

Drug Development Platform

Manufacturing and Quality Control System

Drug Development Services

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