Finding answers for genetic diseases and complex disorders
Bringing 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 potentially innovative gene therapies in our current clinical pipeline include:
Pompe disease
LGMD 2I/R9
Congestive heart failure
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 diseases may present later in life.
Bringing gene therapeutics to life
We have a world-class team of researchers and scientists who are diligently working to develop and investigate therapeutics that target genetic diseases.
AskBio AAV technology platform
Working to create gene therapeutics to reach millions of people in need requires advanced technology for research, clinical translation, and manufacturing.
