Finding answers for genetic diseases

Bringing life-changing gene therapeutics to patients with diseases that have a high unmet medical need fuels our research and development pipeline. We 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

Pompe 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.


Limb-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 failure

Congestive 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 acidemia

Methylmalonic 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.

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.