Scientific Publications

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February 8, 2024

Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa Published in the New England Journal of Medicine

Summary: The New England Journal of Medicine has published data on the application of beremagene geperpavec (B-VEC) to treat ocular complications in a patient with dystrophic epidermolysis bullosa (DEB).

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November 3, 2023

Localized airway delivery of Interleukins-12 and -2 via inhalation of a replication-defective HSV-1-based therapy for the treatment of primary and secondary lung tumors

Summary: During the SITC Annual Meeting & Pre-Conference Programs in San Diego, California from November 1-5, data was presented on preclinical data in multiple oncology models. One presentation showed that non-invasive inhalation of vector-encoded IL-2 and IL-12 was found to be both safe and effective in treating lung tumors in a metastasis model, resulting in long-term survival after single or repeated cancer cell challenge, suggestive of prolonged adaptive immunity.

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November 3, 2023

A replication-defective, HSV-1 based gene therapy for localized delivery of combinatorial Interleukins-12 and -2 for the treatment of cutaneous malignancies

Summary: During the SITC Annual Meeting & Pre-Conference Programs in San Diego, California from November 1-5, data was presented on preclinical data in multiple oncology models. One presentation showed combinatorial IL-2 and IL-12 expressed from Krystal’s platform technology provided a synergistic effect in a melanoma model, suppressing treated and non-treated tumor outgrowth, enhancing survival, and eliciting a durable memory response sufficient for recurrent tumor control.

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October 25, 2023

Nonclinical pharmacology of KB408, an HSV-1-based vector designed for the treatment of alpha-1 antitrypsin deficiency, in the SERPINA1 knockout mouse model

Summary: During the ESGCT 2023 Conference held in Brussels, Belgium from October 24-27, data was presented on the nonclinical pharmacology of KB408 for the treatment of alpha-1 antitrypsin deficiency, in the SERPINA1 knockout mouse model.

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June 14, 2023

Type VII Collagen Deficiency in the Oncogenesis of Cutaneous Squamous Cell Carcinoma in Dystrophic Epidermolysis Bullosa

Summary: The Journal of Investigative Dermatology has published an article that examines cutaneous squamous cell carcinoma pathophysiology in dystrophic epidermolysis bullosa with a focus on known oncogenesis pathways at play and explores the idea that therapeutic type VII collagen replacement may reduce cutaneous squamous cell carcinoma risk.

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May 11, 2023

Long Term Use of Topical Beremagene Geperpavec (B-VEC) in Two Patients with Dystrophic Epidermolysis Bullosa Presented at the International Societies for Investigative Dermatology (ISID) 2023 Conference

Summary: During the ISID 2023 Conference held in Tokyo, Japan from May 10-13, data was presented on the long term use of topical B-VEC in two patients with dystrophic epidermolysis bullosa.

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April 23, 2022

New Data on Topical Application of B-VEC to the Eye to Treat Ocular Complications in a Patient with Dystrophic Epidermolysis Bullosa

Summary: New data on topical application of B-VEC to the eye to treat ocular complications in a patient with dystrophic epidermolysis bullosa was presented at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting.

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December 15, 2022

Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa Published in the New England Journal of Medicine

Summary: The New England Journal of Medicine has published data from the pivotal Phase 3 (GEM-3) trial of beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB).

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November 07, 2022

NACF 2022 Presentation - Respiratory cell-type affinity and absolute CFTR expression in the primate airway upon nebulization of KB407

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September 29, 2022

Clinical characteristics, healthcare use, and annual costs among patients with dystrophic epidermolysis bullosa Published in Orphanet Journal of Rare Diseases

Summary: Orphanet Journal of Rare Diseases has published an article detailing dystrophic epidermolysis bullosa (DEB) as a serious, ultra-rare, genetic blistering disease that requires a multidisciplinary care team and lifelong, proactive disease management.

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June 29, 2022

A new era of in vivo gene therapy: the applicability of a differentiated HSV-1 based vector platform for redosable medicines Published in Cell & Gene Therapy Insights

Summary: A review of Krystal Biotech’s proprietary HSV-1 based platform which is being leveraged to develop treatments for patients with serious diseases, including rare diseases in skin, lung, and other organ systems, has been published in Cell & Gene Therapy Insights.

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June 15, 2022

Coverage of B-VEC Development Published in Nature Biotechnology

Summary: Nature Biotechnology has published an article covering beremagene geperpavec (B-VEC), an investigational, topical, redosable gene therapy, as the first HSV-1-based gene therapy in development for dystrophic epidermolysis bullosa (DEB).

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May 19, 2022

Safety and Immunogenicity Data from the GEM-3 Study of B-VEC Presented at SID 2022

Summary: During the Society for Investigative Dermatology (SID) Annual Meeting held May 18-21, 2022 in Portland, Oregon, new data on the safety and immunogenicity of beremagene geperpavec (B-VEC), an investigational, topical, redosable gene therapy, in the Phase 3 GEM-3 study in patients with dystrophic epidermolysis bullosa (DEB) were presented.

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March 28, 2022

In Vivo Topical Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa on Phase 1 and 2 published in Nature Medicine

Summary: Nature Medicine has published data from the Phase 1 and Phase 2 trial of beremagene geperpavec (B-VEC), an investigational, topical, redosable gene therapy in development for recessive dystrophic epidermolysis bullosa (RDEB).

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March 28, 2022

Pre-clinical Data and Results of the Phase 1/2 Study of B-VEC Published in Nature Medicine

Summary: The pre-clinical data and results from the GEM-1 Phase 1/2 study of beremagene geperpavec (B-VEC), an investigational, topical, redosable gene therapy, in patients with recessive dystrophic epidermolysis bullosa (RDEB) were published in Nature Medicine.

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March 26, 2022

New Results from the GEM-3 Phase 3 Study of B-VEC Presented at AAD 2022

Summary: Positive results of the GEM-3 Phase 3 study of beremagene geperpavec (B-VEC), an investigational, topical, redosable gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB), were presented during a late-breaking abstract session at the 2022 American Academy of Dermatology (AAD) Annual Meeting which took place March 25-29, 2022 in Boston, MA.

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November 19, 2021

Pre-clinical Data on KB303 Presented at ASDS 2021

Summary: Jeune Aesthetics, Inc., a subsidiary of Krystal Biotech, Inc., presented new pre-clinical data on KB303, an investigational, redosable gene therapy in development for the treatment of age- or environment-related loss in cutaneous elasticity, at the American Society for Dermatologic Surgery (ASDS) 2021 Virtual Meeting held November 19-21, 2021.

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November 2, 2021

New Non-Clinical Pharmacology Data on KB407 Presented at NACFC 2021

Summary: New pre-clinical data describing the pharmacology of nebulized KB407, an investigational, redosable gene therapy in development for the treatment of cystic fibrosis, was presented virtually at the North American Cystic Fibrosis Conference (NACFC) from November 2 – 5, 2021.

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October 20, 2021

Data on Preclinical pharmacology of KB408, an HSV-1-based gene therapy vector, for the treatment of alpha-1 antitripsin deficiency

Summary: New preclinical data on the pharmacology of KB408 indicated that KB408 efficiently transduced human cells, including clinically relevant primary human small airway epithelia cells (SAECs), resulting in production and secretion of full-length human AAT. (DEB).

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September 16, 2021

New Data on B-VEC and Dystrophic Epidermolysis Bullosa (DEB) Presented at the DEBRA International Conference 2021

Summary: During the DEBRA International Conference held virtually September 16-19, 2021, new data on use of beremagene geperpavec (B-VEC) in a murine corneal wound model, the proprietary Krystal Biotech HSV-1-based platform, burden of illness and socioeconomic costs of dystrophic epidermolysis bullosa (DEB) were presented.

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May 3, 2021

New Safety Data from the GEM-1 Phase 1/2 Study of B-VEC and the Phase 1 Study of KB301 Presented at SID 2021

Summary: During the Society for Investigative Dermatology (SID) Annual Meeting held virtually May 3-8, 2021, we presented new safety results from our GEM-1 Phase 1/2 study of beremagene geperpavec (B-VEC), an investigational, topical, redosable gene therapy, in patients with recessive dystrophic epidermolysis bullosa (RDEB) and from the Phase 1 study of KB301, an investigational, redosable gene therapy, for the treatment of aesthetic skin conditions.

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April 1, 2021

Preclinical Data on KB105, in Development for the Treatment of Autosomal Recessive Congenital Ichthyosis, Published in the Journal of Investigative Dermatology

Summary: Preclinical data on KB501, an investigational, topical redosable gene therapy in development for the treatment of autosomal recessive congenital ichthyosis (ARCI), has been published in the Journal of Investigative Dermatology.

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October 9, 2020

Preclinical Data for KB301 Presented at ASDS 2020

Summary: New in vitro and in vivo data on KB301, an investigational, redosable gene therapy in development for the treatment of aesthetic skin conditions, was presented at the American Society for Dermatologic Surgery (ASDS) 2020 Virtual Meeting held October 9-11, 2020.

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May 14, 2020

New Data Across the B-VEC and KB105 Programs Presented at SID 2020

Summary: Positive results from the GEM-1 Phase 1/2 study of beremagene geperpavec (B-VEC), an investigational, topical redosable gene therapy, in patients with recessive dystrophic epidermolysis bullosa (RDEB) along with data on KB105, an investigational, redosable gene therapy, from the Phase 1/2 study in patients with TGM1-related autosomal recessive congenital ichthyosis (ARCI) were presented virtually at the Society for Investigative Dermatology (SID) Annual Meeting held May 13-16, 2020.

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May 12, 2020

In Vitro Pharmacology Data on KB407 Presented at ASGCT 2020

Summary: New in vitro pharmacology data on KB407, an investigational, redosable gene therapy in development for the treatment of cystic fibrosis, was presented at the American Society of Genetic & Cell Therapy (ASGCT) Annual Meeting held virtually from May 12-15, 2020.

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June 24, 2019

GEM-2 KB103 Phase 2 Clinical Study Update

Summary: Additional data about the GEM-2 KB103 Phase 2 clinical study were presented.

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May 9, 2019

New Data on KB105 and KB104 Programs Presented at SID 2019

Summary: New preclinical data on KB105, an investigational, redosable gene therapy in development for autosomal recessive congenital ichthyosis (ARCI), and on KB104, an investigational, redosable gene therapy in development for Netherton Syndrome, was presented at the Society for Investigative Dermatology (SID) Annual Meeting held May 8-11, 2019 in Chicago, IL.

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May 18, 2018

New Preclinical Data on B-VEC Presented at the IID 2018

Summary: New in vivo data on B-VEC, an investigational, topical redosable gene therapy, being studied in recessive dystrophic epidermolysis bullosa (RDEB) skin was presented at the International Investigative Dermatology (IID) Meeting held in May 16-19, 2018 in Orlando, FL.

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September 24, 2017

HSV-1 mediated COL7A1 (KB103) delivery to keratinocytes and fibroblasts for recessive dystrophic epidermolysis bullosa (RDEB) therapy: preparations for Phase-I clinical trials

Summary: During the EB2017 Research Congress and the EB Clinet Conference, held September 24-27, 2017 in Salzburg, Austria, preclinical assessment data of HSV-1 mediated COL7A1 delivery in vitro and in vivo were presented.