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, cardiovascular, metabolic and other disorders.
| Disease | Discovery | Preclinical | Phase 1 / Phase 2 |
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| Neuromuscular | |||
| Pompe disease |
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| LGMD (2i/R9) |
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| CNS | |||
| Parkinson’s disease |
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| Multiple system atrophy |
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| Huntington’s disease |
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| Cardiovascular | |||
| Congestive heart failure |
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| Neuromuscular | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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CNS |
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Cardiovascular |
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Pompe diseaseLGMD 2iCongestive heart failureMultiple System AtrophyParkinson’s diseaseOut-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) in Zolgensma® for the treatment of spinal muscular atrophy (SMA).
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