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SB-007

Stargardt disease

Phase 1/2Active

Key Facts

Indication

Stargardt disease

Phase

Phase 1/2

Status

Active

Company

SpliceBio

About SpliceBio

SpliceBio is a private, clinical-stage biotech leveraging its novel Protein Splicing platform to overcome the payload limitations of standard AAV vectors, enabling the delivery of large genes for gene therapy. The company's lead asset, SB-007 for Stargardt disease, is in clinical development, supported by over $200 million in venture funding from top-tier investors. With a seasoned leadership team and a clear focus on inherited retinal diseases and beyond, SpliceBio aims to create a new class of genetic medicines for patients with limited treatment options.

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Therapeutic Areas

Ophthalmology Genetic Diseases

Other Stargardt disease Drugs

Drug	Company	Phase	Status
KIO-301	Kiora Pharmaceuticals	Preclinical	Active
EIR-0205	EIR Biopharma	Preclinical	Active
Emixustat HCl	Kubota Vision	Phase 3	Active
Gildeuretinol (ALK-001)	Alkeus Pharmaceuticals	Phase 3	Active
MCO-010	Nanoscope Therapeutics	Phase 2	Active
LBS-008	Lin BioScience	Phase 3	Active
OCU410ST	Ocugen	Phase 2/3	Active
ABO-504	Abeona Therapeutics	Preclinical	Active