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 THERAPIES

Preclinical
Phase I/II
Phase III
Marketed

METABOLIC

ABO-102 (sc AAV-SGSH) : Sanfilippo Syndrome Type A (MPS IIIA)
Current
ABO-101 (AAV-NAGLU) : Sanfilippo syndrome Type B (MPS IIIB)
Current
ABO-201 (sc AAV-CLN3) : Juvenile Batten disease (CLN3)
Current
ABO-202 (AAV-CLN1): Infantile Batten disease (CLN1)
Current

DERMATOLOGY

EB-101 (LZRSE-Col7A1) : Recessive dystrophic epidermolysis bullosa (RDEB)
Current
EB-201 (AAV-DJCol7A1): Epidermolysis bullosa (EB)
Current

HEMATOLOGY

ABO-301 (AAV-FANCC): Fanconi anemia (FA)
Current
ABO-302 (CRISPR-Cas9): Rare Blood diseases
Current

AAV VECTOR PLATFORM

Preclinical
Phase I/II
Phase III
Marketed
AIM™ Vectors: 2nd Gen and New AAV Products
Current

Orphan Drug Designation (FDA) Orphan Drug Designation (EU) Rare Pediatric Disease Designation (FDA) Fast Track Designation (FDA)

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PLASMA THERAPIES

Research
Preclinical
Phase I/II
Marketed
SDF™ ALPHA (AIPI): Inherited COPD
Current
SDF™ GAMMA (IVIG): Autoimmunie, infectious diseases
Current
Alpha-1 antitrypsin deficiency (Alpha-1) is a rare (1 in 1,500 to 3,500) genetic (inherited) autosomal disorder that may cause lung disease and liver disease from retained misfolded protein. Abeona Therapeutics is developing PTB-101 SDF Alpha™ (alpha1-proteinase inhibitor) for chronic augmentation and maintenance therapy in adults with clinically evident emphysema due to severe deficiency of alpha1-proteinase inhibitor.