Our growing gene therapy pipeline
Our pipeline is the result of patient-focused innovation, rigorous clinical standards and research collaborations that drive a promising portfolio of gene therapies. We continue to advance therapeutics toward potential regulatory approval and introduce preclinical candidates to bring hope to patients who suffer from neuromuscular, central nervous system and other genetic disorders.
| Disease | Discovery | Preclinical | Phase I/II |
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| Neuromuscular | |||
| Pompe disease |
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| LGMD (2i/R9) |
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| CNS | |||
| Huntington’s disease |
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| Angelman syndrome |
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| Parkinson’s disease* |
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| Cardiovascular | |||
| Congestive heart failure |
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| Metabolic | |||
| Methylmalonic acidemia (MMA) |
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CNS |
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Cardiovascular |
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Metabolic |
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* In partnership with Brain Neurotherapy Bio, Inc.* Pompe diseasePompe disease is a rare genetic and often fatal metabolic disorder. Due to a deficiency in the acid alpha-glucosidase (GAA) enzyme, glycogen is not broken down causing damage to muscle and nerve cells throughout the body. Various organs and tissues are affected, particularly the muscles. LGMD 2iLimb-girdle muscular dystrophy type 2I (LGMD2I) is caused by mutations in the FKRP gene. It is a form of limb-girdle muscular dystrophy, which refers to a group of conditions that cause weakness and wasting of the muscles in the arms and legs. Congestive heart failureCongestive heart failure (CHF) is a chronic, progressive condition where the heart cannot pump sufficient amounts of blood and oxygen to the body. Gene therapy to express an active form of protein I-1c may provide a new approach to treating heart failure. Methylmalonic acidemiaMethylmalonic acidemia (MMA) is a rare disease in which the body cannot break down certain fats and proteins. This metabolic disorder, caused by genetic mutations, can lead to long-term complications including growth delay, intellectual disability, kidney disease and pancreatitis. Out-licensed clinicals & technologyWe have licensed our technology to some of the world’s leading biopharmaceutical companies, which further validates our science. We have ongoing licensing agreements for clinical indications in hemophilia (Chatham Therapeutics acquired by Takeda in 2014) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer in 2016). In 2015, AskBio granted AveXis a non-exclusive, worldwide license for the use of our self-complementary DNA technology (scAAV) for the treatment of spinal muscular atrophy (SMA). We continue to collaborate with these and many other companies to advance potential therapies and AAV technology.
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