Product Description
ABO-503, composed of a functional human RS1 packaged in the novel AIMâ"¢ capsid AAV204, has shown preclinical efficacy following delivery to the retina in a mouse model of XLRS. Preclinical studies have demonstrated robust RS1 expression in the retina, improved cone photoreceptor density and overall photoreceptor cell survival, as well as a restoration of outer retina architecture. (Sourced from: https://investors.abeonatherapeutics.com/press-releases/detail/246/abeona-therapeutics-announces-update-on-aav-ophthalmology)
Mechanisms of Action: Gene Therapy, RS1
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Company: Abeona
Company Location: Eastern America
Company Founding Year: 2013
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
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Recent & Upcoming Milestones
Highest Development Phases
Phase 0: Retinoschisis
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