Products

Genethon teams develop gene therapy products for rare diseases, in collaboration with academic and industrial teams in France and abroad.

Several products from Genethon R&D (or to which Genethon has been closely associated) are currently in the clinical trial stage. These clinical trials are sponsored by Genethon or by partners in Europe or the United States.
These therapies deal with neuromuscular diseases, the immune system, ocular or metabolic 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
Pompe disease

In vivo Gene Therapy/AAV

Diseases of vision

Leber’s Hereditary Optic Neuropathy

In vivo Gene Therapy/AAV

Collaboration