Products

Genethon is currently sponsoring two gene therapy projects that have reached clinical trial phase, and is involved in several other projects in preclinical or research stages.

These projects, which are being developed either internally at Genethon or as part of partnerships, involve neuromuscular diseases, pathologies of the immune system, of the eye, and other rare diseases.

 

Technology

Research

Preclinical

Phase I/II

Muscle

Duchenne Muscular Dystrophy (exon)

In vivo Gene Therapy/AAV

Duchenne Muscular Dystrophy (microdystrophine)

In vivo Gene Therapy/AAV

Spinal Muscular Atrophy

In vivo Gene Therapy/AAV

Myotubular Myopathy

In vivo Gene Therapy/AAV

Collaboration
Limb-Girdle Muscular Dystrophy

In vivo Gene Therapy/AAV

Immune deficits and blood diseases

Wiskott-Aldrich Syndrome (WAS)

Ex vivo Gene Therapy/LV

Chronic Granulomatous Disease

Ex vivo Gene Therapy/LV

Radiosensitive Severe Combined Immunodeficiency (SCID-X1)

Ex vivo Gene Therapy/LV

Radiosensitive Severe Combined Immunodeficiency (RS-SCID Artemis gene mutation)

Ex vivo Gene Therapy/LV

Collaboration
Fanconi Anemia

Ex vivo Gene Therapy/LV

Collaboration
Sickle Cell Disease

Ex vivo Gene Therapy/LV

Liver diseases

Crigler-Najjar Syndrome

In vivo Gene Therapy/AAV

Collaboration

Diseases of vision

Leber’s Hereditary Optic Neuropathy

In vivo Gene Therapy/AAV

Collaboration