Our growing gene therapy pipeline
The AskBio pipeline is centered on patient-focused innovation, rigorous clinical standards, and research collaborations that drive a promising portfolio of gene therapies. We continue to advance current therapeutics toward potential regulatory approval and introduce pre-clinical candidates into our current pipeline, bringing hope to patients who suffer from neuromuscular and central nervous system genetic conditions.
| 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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| Neuromuscular | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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 clinical researchAs a result of extensive, pioneering research for Duchenne muscular dystrophy and hemophilia as well as AskBio’s development of self-complementary adeno-associated virus (scAAV) therapeutics, we have sold and licensed our technology to some of the world’s leading biopharma companies. We continue to collaborate with these and many other companies to advance potential therapies and AAV technology.
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