Abeona Therapeutics

Abeona Therapeutics

ABEOPhase 3
Cleveland, United Statesabeonatherapeutics.com

Abeona Therapeutics is dedicated to delivering transformative cell and gene therapies for patients with severe rare diseases. The company has achieved a significant milestone with the commercial launch of ZEVASKYN® and maintains a diverse pipeline spanning preclinical to Phase 1/2 development, primarily targeting inherited retinal diseases and neurological conditions. Its strategy is supported by in-house manufacturing capabilities at The Elisa Linton Center for Rare Disease Therapies and key partnerships with Ultragenyx and Taysha Gene Therapies.

Focus
BiologicsRNA & Gene Therapy

ABEO · Stock Price

USD 5625.0021250.00 (-79.07%)

Historical price data

AI Company Overview

Abeona Therapeutics is dedicated to delivering transformative cell and gene therapies for patients with severe rare diseases. The company has achieved a significant milestone with the commercial launch of ZEVASKYN® and maintains a diverse pipeline spanning preclinical to Phase 1/2 development, primarily targeting inherited retinal diseases and neurological conditions. Its strategy is supported by in-house manufacturing capabilities at The Elisa Linton Center for Rare Disease Therapies and key partnerships with Ultragenyx and Taysha Gene Therapies.

Technology Platform

Proprietary AIM™ (Abeona Integration Machine) vector platform based on adeno-associated virus (AAV) technology, designed for enhanced gene delivery and transduction efficiency in target tissues.

Pipeline Snapshot

9

9 drugs in pipeline, 4 in Phase 3

DrugIndicationStage
ofloxacin + MSI-78Diabetic Foot UlcersPhase 3
ofloxacin + MSI-78Diabetic Foot UlcersPhase 3
EB-101Epidermolysis BullosaPhase 3
EB-101 Surgical application of RDEB woundsEpidermolysis BullosaPhase 3
EcoNail™ (econazole 5%/SEPA® 18% nail lacquer)OnychomycosisPhase 2

Opportunities

Significant growth opportunities exist in expanding the commercial footprint of ZEVASKYN® and advancing the promising preclinical retinal disease pipeline (ABO-503, ABO-504, ABO-505) into clinical trials.
Successful data readouts from partnered programs with Ultragenyx and Taysha could also unlock substantial value through milestone payments and royalties.

Risk Factors

Key risks include the commercial execution challenge for ZEVASKYN®, the high failure rate inherent in drug development for the preclinical/clinical pipeline, potential future dilution from capital raises, and intense competition in the gene therapy space from larger, better-funded rivals.

Competitive Landscape

Abeona faces competition across its therapeutic areas, including from established gene therapy leaders like Spark/Roche in retinal diseases and various biotechs in neurological disorders. Its differentiation is based on the proprietary AIM™ vector platform and integrated in-house manufacturing, but it must demonstrate clinical superiority and execute effectively in a crowded market.

Company Info

TypeTherapeutics
LocationCleveland, United States
StagePhase 3
RevenueEarly Revenue

Trading

TickerABEO
ExchangeNASDAQ

Therapeutic Areas

Rare DiseasesInherited Retinal DiseasesNeurological Disorders

Partners

Ultragenyx Pharmaceutical Inc.Taysha Gene Therapies, Inc.
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