Inherited Retinal Disease
Splicebio S.L. focuses on Stargardt disease, with particular interest in Stargardt disease type 1 linked to ABCA4 gene mutations and the resulting retinal degeneration seen in inherited macular disorders.
- Stargardt disease
- Stargardt disease type 1
- ABCA4 mutation-associated retinal disease
The sponsor’s clinical activity is directed toward ophthalmic conditions affecting central vision and retinal structure.
Gene-Linked Ophthalmology
Its research interest includes genetically confirmed retinal disease, with emphasis on patients carrying biallelic ABCA4 mutations and the therapeutic implications of correcting disease driven by a defined molecular defect.
- Genotype-confirmed retinal disorders
- Biallelic ABCA4 mutations
- Molecularly targeted eye disease
This places the sponsor within the field of precision-oriented ophthalmic genetics.
Retinal Gene Therapy
Splicebio S.L. is engaged in retinal gene therapy centered on subretinal administration for a degenerative retinal condition, reflecting therapeutic interest in restoring retinal function through localized ocular intervention.
- Subretinal delivery
- Ocular gene-based treatment
- Retinal function preservation
The sponsor’s program is aligned with treatment strategies for macular degeneration driven by inherited disease mechanisms.
Ophthalmic Safety and Tolerability
Its clinical research also addresses safety, tolerability, and early efficacy assessment in an ocular setting, supporting development in diseases where visual impairment is progressive and treatment options remain limited.
- Ocular safety
- Tolerability assessment
- Preliminary efficacy in retinal disease
These interests are concentrated in vision-threatening inherited retinal disorders.
