Rare & Orphan Diseases
Bringing hope to patients with rare diseases through innovative therapeutic development
Orphan Drug Development
Oracle BioTech is committed to developing treatments for rare diseases that affect small patient populations but have significant unmet medical needs, leveraging regulatory incentives and innovative approaches.
Genetic Disorders
Gene therapies and precision medicines targeting inherited genetic disorders with life-altering potential.
- Inherited metabolic disorders
- Muscular dystrophies
- Lysosomal storage diseases
- Primary immunodeficiencies
Ultra-Rare Conditions
Developing treatments for ultra-rare diseases affecting fewer than 1 in 50,000 people, often with no existing therapies.
- Progeria syndromes
- Rare neurological disorders
- Metabolic pathway defects
- Autoimmune rare diseases
Rare Disease Pipeline
Research
8 ProgramsTarget identification and mechanism of action studies.
Preclinical
5 ProgramsDisease model validation and lead optimization.
Phase I/II
4 ProgramsCombined safety and efficacy studies in rare diseases.
Phase III
2 ProgramsPivotal trials for regulatory approval.
Approved
3 ProductsFDA and EMA approved orphan drugs.