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confidence high
sentiment positive
materiality 0.55
Krystal Biotech reports positive ocular data from compassionate use of B-VEC in DEB patient
Krystal Biotech, Inc.
- Topical B-VEC achieved full corneal healing by 3 months and improved visual acuity from hand motion to 20/40 at 7 months.
- No drug-related adverse events observed; treatment was well tolerated in the patient.
- Patient had recurrent cicatrizing conjunctivitis; ocular complications affect >50% of recessive DEB patients.
- Company plans to engage regulators to expand B-VEC utility for ocular DEB manifestations.
- Ongoing preclinical studies evaluating intravitreal and subretinal deliveries for other ocular diseases.