Working together to find a cure.

Abeona (ey-bee-ohn-uh): The Roman Goddess who protects children as they take their first steps away from home.

What is Gene Therapy?

In many disorders, particularly genetic diseases caused by a single genetic defect, gene therapy aims to treat a disease by delivering the correct copy of DNA into a patient’s cells.

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Rare Diseases in Children

While rare diseases can affect any age group, about 50% of people affected are children; and rare diseases account for 35% of deaths in first year of life. Find out what we’re doing to help.

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Research & Development

Abeona Therapeutics is working on a number of exciting technologies and products aimed at curing various rare diseases. Find out what we’re working on and what we have planned.

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Pipeline

Gene Therapy

Research
Preclinical
Phase I/II
Marketed
ABO-102 (sc AAV9-SGSH): Sanfilippo Syndrome Type A
Current
ABO-101 (AAV9-NAGLU): Sanfilippo Syndrome Type B
Current
ABO-201 (sc AAV9-CLN3): Juvenile Batten Disease (JNCL)
Current
ABO-301 (AAV-FANCC): Fanconi Anemia (FA)
Current
CRISPR-CAS9-AAV: Rare Blood Diseases TBA
Current

Plasma Protein Therapy

Research
Preclinical
Phase I/II
Marketed
SDF Alpha™ (alpha-1 protease inhibitor): Inherited COPD
Current
SDF IVIG™ (intravenous immunoglobulin): Autoimmune, infectious, and idiopathic diseases
Current