Krystal Biotech, Inc., headquartered in Pittsburgh, Pennsylvania, is a clinical-stage gene therapy company focused on developing treatments for rare genetic skin disorders. Since its founding in 2015, the company has utilized a proprietary, non-integrating herpes simplex virus type 1 (HSV-1) vector platform to deliver functional copies of genes directly into skin cells. Krystal’s integrated research and manufacturing facility supports seamless progression of its product candidates from preclinical development through pivotal clinical trials.
The company’s lead product candidate, beremagene geperpavec (B-VEC), is designed for the treatment of dystrophic epidermolysis bullosa (DEB), a severe blistering disorder caused by mutations in the COL7A1 gene. B-VEC has received both Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration and is currently in late-stage clinical evaluation in the United States and Europe. In addition to B-VEC, Krystal’s pipeline includes early-stage gene therapies for Netherton syndrome and other single-gene dermal diseases, each leveraging its topical delivery platform.
In 2019, Krystal Biotech completed its initial public offering on the Nasdaq under the ticker KRYS, securing funding to advance its clinical programs and expand manufacturing capacity. The company collaborates with leading dermatology centers and academic institutions across North America and Europe to support patient enrollment, clinical execution, and eventual market preparation. Its end-to-end approach to vector design, process development and clinical trial management is intended to streamline regulatory pathways and reduce time to potential approval.
At the helm of Krystal Biotech is co-founder and Chief Executive Officer Amit D. Jain, whose expertise in molecular biology and gene therapy drives the company’s strategic vision. The executive team comprises seasoned professionals in regulatory affairs, manufacturing, clinical development and business development, all dedicated to bringing innovative, potentially transformative treatments to patients living with debilitating skin conditions worldwide.
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