Transforming medicine and changing lives

AskNews

November 19, 2020

AskBio and Selecta Biosciences Receive Orphan Drug Designation for MMA-101 to Treat Methylmalonic Acidemia

The FDA has granted orphan drug and rare pediatric disease designations to AskBio's gene therapy, MMA-101, for the treatment of methylmalonic acidemia. ...read the news

We are on a never-ending quest to advance genetic technology

and life-saving AAV gene therapy

In 1984, Dr. Jude Samulski, one of AskBio’s visionary founders, asked if a virus could be used to erase genetic disease. Discovering the answer cleared the path for historic advances in genetic medicine, and AskBio is leading the way.

Learn about AskBio »

gene therapy

Revolutionizing gene therapy

Cures for life-altering genetic diseases depend on new technology to lower the cost of treatment and increase supply for every patient who needs it.

Explore our technology »

Clinical Pipeline

Changing lives

Our clinical pipeline is the direct result of listening to patients, their families, their care teams and disease-advocacy organizations.

Discover our pipeline »

Putting patient care first

The voices of those affected by genetic disorders drive everything we do. They propel our research and the urgent need to find cures.

For Patients »

Work with us

Advancing research

Today’s genetic discoveries are some of the most awe-inspiring advances in medical history. We welcome collaboration with innovative minds in genetic R&D.

Building careers

Help transform the face of healthcare with life-changing gene therapy. Every life enriched by our scientific advancements is a result of people like you.

Events

2nd Annual Gene Therapy for Neurological Disorders

Dec 8

Dec 10

Jude Samulski and Krystof Bankiewicz will present at the 2nd Annual Gene Therapy for Neurological Disorders conference on Dec. 10, 2020.

News