Transforming medicine and changing lives

AskNews

October 20, 2020

Selecta Biosciences and AskBio Receive FDA Rare Pediatric Disease Designation for their Gene Therapy for Methylmalonic Acidemia

The FDA grants Rare Pediatric Disease Designation to MMA-101 for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations. ...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

Chief Medical Officer Summit 360

Oct 26

Oct 27

Join us at the 8th annual Chief Medical Officer Summit, the largest gathering of physicians in biotech, to be held virtually from October 26-27, 2020.

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