Drug Profile

SB 007

Alternative Names: SB-007

Latest Information Update: 16 Jan 2026

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At a glance

Originator Splice Bio
Class Eye disorder therapies; Gene therapies
Mechanism of Action ABCA4 protein replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Stargardt disease
New Molecular Entity No

Highest Development Phases

Phase I/II Stargardt disease

Most Recent Events

08 Jan 2026 SB 007 receives Fast Track designation for Stargardt disease [Subretinal,Injection] (In adolescents, In the elderly, In adults) in USA
08 Jan 2026 SB 007 receives Orphan Drug status for Stargardt disease in Europe
11 Feb 2025 Phase-I/II clinical trials in Stargardt disease (In adolescent, In adults, In elderly) in USA (Subretinal) (NCT06942572)

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