Krystal Biotech
Krystal Biotech is a pioneering biotechnology company dedicated to developing and delivering genetic medicines through innovative gene therapies for severe, life-threatening, or rare diseases with limited or no approved treatments. Their mission is to create accessible, effective, and redosable gene delivery therapies, leveraging a proprietary platform to transform the treatment landscape for genetic diseases. The company's first commercial product, VYJUVEK®, is the only redosable gene therapy approved for dystrophic epidermolysis bullosa, and they are actively advancing a pipeline of genetic therapies across various therapeutic areas including dermatology, respiratory, oncology, ophthalmology, and aesthetics. Krystal emphasizes supporting the healthcare community through partnerships, clinical research, and patient support programs such as Krystal Connect.
Industries
Nr. of Employees
large (251-1000)
Krystal Biotech
Pittsburgh, Pennsylvania, United States
Patents
Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
US-11717547-B2
View Details
Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
US-11717547-B2
View DetailsProducts
Redosable gene therapy for dystrophic epidermolysis bullosa (DEB)
A redosable gene therapy intended to treat dystrophic epidermolysis bullosa by delivering corrective transgene expression to affected skin tissue.
Beremagene geperpavec
An investigational topical herpes simplex virus type 1 (HSV-1)-based gene therapy designed to restore type VII collagen protein by delivering COL7A1 for the treatment of dystrophic epidermolysis bullosa.
Beremagene Geperpavec (B-VEC)
An HSV-1 based topical in vivo gene therapy designed to restore functional collagen VII via COL7A1 gene delivery for the treatment of dystrophic epidermolysis bullosa.
VYJUVEK
FDA-approved gene therapy for dystrophic epidermolysis bullosa designed to deliver missing or defective genes to the skin.
HSV-1–based Gene Therapy Platform
Krystal's engineered replication deficient HSV-1-based platform enables the development of redosable gene therapies that can be administered non- or minimally-invasively.
KB303
An innovative and minimally invasive HSV-1-based gene therapy designed to enable local elastin replacement and improve skin elasticity in aged or photodamaged skin.
Redosable gene therapy for dystrophic epidermolysis bullosa (DEB)
A redosable gene therapy intended to treat dystrophic epidermolysis bullosa by delivering corrective transgene expression to affected skin tissue.
Beremagene geperpavec
An investigational topical herpes simplex virus type 1 (HSV-1)-based gene therapy designed to restore type VII collagen protein by delivering COL7A1 for the treatment of dystrophic epidermolysis bullosa.
Beremagene Geperpavec (B-VEC)
An HSV-1 based topical in vivo gene therapy designed to restore functional collagen VII via COL7A1 gene delivery for the treatment of dystrophic epidermolysis bullosa.
VYJUVEK
FDA-approved gene therapy for dystrophic epidermolysis bullosa designed to deliver missing or defective genes to the skin.
HSV-1–based Gene Therapy Platform
Krystal's engineered replication deficient HSV-1-based platform enables the development of redosable gene therapies that can be administered non- or minimally-invasively.
KB303
An innovative and minimally invasive HSV-1-based gene therapy designed to enable local elastin replacement and improve skin elasticity in aged or photodamaged skin.
Services
On-site GMP production and scale-up support for viral vector drug substance and drug product to support clinical and commercial supply.
Patient-facing support services for therapy education, benefits investigation, treatment planning and care coordination delivered via phone and online resources.
On-site analytical development, validated release assays and QC testing for viral vector products, supported by AD/QC laboratory infrastructure and validated titering methods.
On-site GMP production and scale-up support for viral vector drug substance and drug product to support clinical and commercial supply.
Patient-facing support services for therapy education, benefits investigation, treatment planning and care coordination delivered via phone and online resources.
On-site analytical development, validated release assays and QC testing for viral vector products, supported by AD/QC laboratory infrastructure and validated titering methods.
Expertise Areas
- Redosable gene therapy approaches
- HSV-1-based viral vector platform development
- GMP viral vector manufacturing and scale-up
- Process development and pilot-to-commercial transfer for viral vectors
Key Technologies
- HSV-1-based viral vectors
- Episomal non-integrating DNA delivery
- High-capacity viral vectors (>30 kb payload)
- Cell and master virus banking
News & Updates
FDA Approval of VYJUVEK®
Krystal Biotech's VYJUVEK® received FDA approval, becoming the only redosable gene therapy approved for dystrophic epidermolysis bullosa.
FDA Approval of VYJUVEK®
Krystal Biotech's VYJUVEK® received FDA approval, becoming the only redosable gene therapy approved for dystrophic epidermolysis bullosa.