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 I/II
Neuromuscular
Pompe disease
LGMD (2i/R9)
CNS
Parkinson’s disease
Multiple system atrophy
Huntington’s disease
Cardiovascular
Congestive heart failure
Neuromuscular
Pompe disease
Discovery Preclinical Phase I/II
LGMD (2i/R9)
Discovery Preclinical Phase I/II
CNS
Parkinson’s disease
Discovery Preclinical Phase I/II
Multiple system atrophy
Discovery Preclinical Phase I/II
Huntington’s disease
Discovery Preclinical Phase I/II
Cardiovascular
Congestive heart failure
Discovery Preclinical Phase I/II

Congestive heart failure

Multiple System Atrophy

Parkinson’s disease

Out-licensed clinicals & technology

We 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).

Disease Discovery Phase I/II Pivotal Marketing Approval
DMD (Pfizer)
Hemophilia (Takeda)
SMA (AveXis/Novartis)
DMD (Pfizer)
Discovery Phase I/II Pivotal Marketing Approval
Hemophilia (Takeda)
Discovery Phase I/II Pivotal Marketing Approval
SMA (AveXis/Novartis)
Discovery Phase I/II Pivotal Marketing Approval

Bringing gene therapeutics to life

We have a world-class team of researchers and scientists who are diligently working to erase genetic disease.

Clinical trials

Joining clinical trials

Because 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 AskBio.

Our clinical trials »