Finding answers for genetic diseases and complex disorders

Bringing life-changing gene therapeutics to patients with diseases that have a high unmet medical need fuels our research and development pipeline. We also have ongoing research collaborations for various neuromuscular and central nervous system conditions with particular attention on rare diseases.

Diseases with innovative new gene therapies in our current clinical pipeline include: 

Pompe disease

LGMD 2i

Congestive heart failure

Methylmalonic acidemia

Multiple System Atrophy

Parkinson’s disease

Understanding genetic disease

There are various types of genetic diseases that result from missing genes or mutations in the genome. Some show signs right after birth, while other onset forms of disease show later in life. 

Bringing gene therapeutics to life

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

AskBio AAV technology platform

Creating powerful gene therapeutics to reach millions of people in need requires advanced technology for research, clinical translation, and manufacturing.