Working together to find a cure.

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

AAV (Adeno-Associated Virus) Gene Therapy

Preclinical
Research
Clinical
Marketed
ABO-101 (sc AAV9 NAGLU): Sanfilippo Syndrome Type B (mucopolysaccharidosis (MPS) III)
Current
ABO-102 (sc AAV9 SGSH): Sanfilippo Syndrome Type A (mucopolysaccharidosis (MPS) III)
Current
ABO-201 (sc AAV9 CLN3): Juvenile Batten Disease (JBD)
Current
ABO-301 (AAV LK19 FANCC): Fanconi Anemia (FA)
Current
Crisper-CAS9 AAV: Rare Blood Diseases-Undisclosed
Current

SDF™ (Salt Diafiltration Process)

Preclinical
Research
Clinical
Marketed
SDF Alpha™ (alpha-1 protease inhibitor): Panacinar emphysema or COPD
Current
SDF IVIG™ (intravenous immunoglobulin): Autoimmune, infectious, and idiopathic diseases
Current