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Treatment of Dystrophic Epidermolysis Bullosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Receives Orphan Drug Designation from the Japanese MHLW for Beremagene Geperpavec-svdt (B-VEC)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]
Find Clinical Drug Pipeline Developments & Deals by Krystal Biotech
KB707 is a modified HSV-1 vector designed to deliver genes encoding both human interleukin-12 (IL-12) and interleukin-2 (IL-2) to the tumor microenvironment and promote systemic immune-mediated tumor clearance. It is being developed for lung cancer, non-small cell.
Vyjuvek (beremagene geperpavec-svdt) is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds.
Vyjuvek (beremagene geperpavec-svdt) is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds.
KB408 is an inhaled (nebulized) formulation of the Company’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment of AATD.
KB408 is an inhaled (nebulized) formulation of the Company’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment of AATD.
The Company was awarded the PRV following the FDA accelerated approval of Vyjuvek (beremagene geperpavec-svdt), , the first-ever redosable gene therapy, for the treatment of recessive or dominant dystrophic epidermolysis bullosa (DEB) for patients 6 months of age and older.
KB707 is a modified HSV-1 vector designed to deliver genes encoding both human IL-12 and IL-2 to the tumor microenvironment and promote systemic immune-mediated tumor clearance.
KB407 is an investigational, redosable gene therapy designed to molecularly correct the underlying cause of CF by delivering two copies of the CFTR gene directly to the airways via nebulization.
Vyjuvek (beremagene geperpavec-svdt) is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. Vyjuvek received FDA Approval for the First-Ever Redosable Gene Therapy for DEB treatment.
The proceeds will fund the Vyjuvek (beremagene geperpavec-svdt) launch, future operations, and the advancement of company's pipeline. Vyjuvek is the first FDA-approved gene therapy treatment for Dystrophic epidermolysis bullosa, a rare and serious genetic skin disorder.
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