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 disease Bringing gene therapeutics to lifeWe have a world-class team of researchers and scientists who are diligently working to erase genetic disease.  Joining clinical trialsBecause many genetic disorders are progressive, time is of the essence and the importance of every clinical trial has greater meaning to all of us at RhiaPharmaceutical.  | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||