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KB803
KB803 is a gene therapy that delivers a functional COL7A1 gene to restore type VII collagen production in patients with recessive dystrophic epidermolysis bullosa.
KB803 is a gene therapy that delivers a functional COL7A1 gene to restore type VII collagen production in patients with recessive dystrophic epidermolysis bullosa. Used for Recessive dystrophic epidermolysis bullosa (RDEB).
At a glance
| Generic name | KB803 |
|---|---|
| Sponsor | Krystal Biotech, Inc. |
| Drug class | Gene therapy |
| Target | COL7A1 gene |
| Modality | Biologic |
| Therapeutic area | Dermatology / Rare Genetic Disorders |
| Phase | Phase 3 |
Mechanism of action
KB803 uses an ex vivo approach where patient fibroblasts are transduced with a modified adeno-associated virus (AAV) carrying the COL7A1 gene, then reintroduced into the skin to produce functional type VII collagen. This addresses the underlying genetic defect in recessive dystrophic epidermolysis bullosa (RDEB), a severe blistering disorder caused by mutations in the COL7A1 gene that encodes collagen VII.
Approved indications
- Recessive dystrophic epidermolysis bullosa (RDEB)
Common side effects
- Application site reactions
- Infection at treatment site
- Scarring or fibrosis
Key clinical trials
- A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- KB803 CI brief — competitive landscape report
- KB803 updates RSS · CI watch RSS
- Krystal Biotech, Inc. portfolio CI