Krystal Biotech Announces Completion of the GEM-3 Pivotal Phase III Study Evaluating B-VEC for the Treatment of Dystrophic Epidermolysis Bullosa

Krystal Biotech, Inc. announced that the last participant has completed the GEM-3 study, Krystal’s pivotal Phase III clinical trial evaluating investigational beremagene geperpavec (B-VEC) as a topical gene therapy for the treatment of dystrophic epidermolysis bullosa.
[Krystal Biotech, Inc.]
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Increased Abundance of Cbl E3 Ligases Alters PDGFR Signaling in Recessive Dystrophic Epidermolysis Bullosa

Investigators indicated that elevated TGFβ signaling led to an attenuated response to growth factors, which contributed to impaired dermal wound healing in recessive dystrophic epidermolysis bullosa.
[Matrix Biology]
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Krystal Biotech and GeneDx Announce Collaboration to Provide No-Charge Genetic Testing for Patients with Suspected Dystrophic Epidermolysis Bullosa (DEB)

Krystal Biotech, Inc. and GeneDx, Inc. announced a collaboration offering no-charge genetic testing for all types of Epidermolysis Bullosa.
[Krystal Biotech, Inc.]
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Pro-Inflammatory Immunity Supports Fibrosis Advancement in Epidermolysis Bullosa: Intervention with Ang-(1–7)

Longitudinal analyses of the tissue proteome through the course of naturally developing disease in recessive dystrophic epidermolysis bullosa mice revealed that increased pro-inflammatory immunity associates with fibrosis evolution.
[EMBO Molecular Medicine]
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Gain-of-Function Mutation in Ubiquitin-Ligase KLHL24 Causes Desmin Degradation and Dilatation in hiPSC-Derived Engineered Heart Tissues

Dynamically loaded engineered heart tissues (dyn-EHTs) were generated from human induced pluripotent stem cell (hiPSC)-derived cardiomyocytes from two patients and three (non)familial controls. KLHL24 RNA interference or direct desmin overexpression recovered desmin protein levels, restoring morphology and function in patient-derived dyn-EHTs.
[Journal of Clinical Investigation]
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The Utility of Dermal Fibroblasts in Treatment of Skin Disorders: A Paradigm of Recessive Dystrophic Epidermolysis Bullosa

The authors review literature about ongoing and completed clinical trials that applied fibroblasts and bioengineered fibroblasts as therapeutic agents for various skin disorders.
[Dermatologic Therapy]
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Single-Keratinocyte Transcriptomic Analyses Identify Different Clonal Types and Proliferative Potential Mediated by FOXM1 in Human Epidermal Stem Cells

Scientists reported that single-cell transcriptome analysis of primary human epidermal cultures identifies categories of genes clearly distinguishing the different keratinocyte clonal types, which are hierarchically organized along a continuous, mainly linear trajectory showing that stem cells sequentially generate progenitors producing terminally differentiated cells.
[Nature Communications]
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Onconova Announced First Patient Dosed in Investigator-Initiated Phase II Study of Rigosertib in Recessive Dystrophic Epidermolysis Bullosa-Associated Squamous Cell Carcinoma

Onconova Therapeutics, Inc., announced that the first patient has been dosed in an investigator-initiated Phase II study to assess the efficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa-associated locally advanced/metastatic squamous cell carcinoma.
[Onconova Therapeutics, Inc. (GLOBE NEWSWIRE)]
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Intravenous Allogeneic Umbilical Cord Blood–Derived Mesenchymal Stem Cell Therapy in Recessive Dystrophic Epidermolysis Bullosa Patients

In a Phase I/IIa trial, scientists evaluated the safety and possible clinical efficacy of intravenous infusion of allogeneic human umbilical cord blood–derived mesenchymal stem cells in patients with recessive dystrophic epidermolysis bullosa.
[JCI Insight]
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Abeona Therapeutics Announces Successful Type B Meeting with FDA for Pivotal Phase 3 VIITAL™ Study of EB-101 in Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Abeona Therapeutics Inc. held a successful Type B meeting with the US FDA to align with the Agency on the company’s proposal regarding co-primary endpoints for the pivotal Phase III VIITAL™ study of EB-101 in recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder where those inflicted have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin.
[Abeona Therapeutics Inc.]
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Growth, Lifetime, Directional Movement and Myosin-Dependent Motility of Mutant Keratin Granules in Cultured Cells

Scientists used epithelial cells producing mutant keratin itermediate filament polypeptides that have been identified as the cause of the hereditary blister-forming skin disease epidermolysis bullosa simplex.
[Scientific Reports]
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