Transforming medicine and changing lives

AskNews

May 25, 2021

AskBio Announces IND for LION-101, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Cleared to Proceed by U.S. FDA

The U.S. FDA has cleared AskBio's Investigational New Drug (IND) application for LION-101 to proceed in a Phase 1/2 clinical study. ...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 diseases depend on new technology to lower the cost of treatment and increase supply for every patient who needs it.

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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 patients 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 someone like you.

Events

BIO International Convention — BIO Digital

Jun 16

For 2021, the BIO International Convention will be held virtually as BIO Digital. BIO Digital will feature sessions on key sessions, including a discussion on gene therapy, including an industry panel on new approaches and challenges to developing and delivering genetic medicines.

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