This page tracks new and upcoming treatment options for epidermolysis bullosa (EB). What’s in development? What’s promising? What’s available soon? Current topics include AP103, gene therapy, EB-101, Filsuvez, AGLE-102, stem cells, B-VEC, KB-103, PTR-01, QR-313, FCX-007, INM-755, and more. Projects
,Last updated: 4 years ago
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News
- Aegle Therapeutics Receives Rare Pediatric Disease Designation from FDA for AGLE-102 for Patients with Dystrophic Epidermolysis Bullosa Oct 6, 2020
- Orphan Drug Status in Europe Likely for AP103, DEB Gene Therapy Sep 30, 2020
- A new approach to understanding the biology of wound healing Sep 15, 2020
- Positive Opinion from EMA on Orphan Drug Designation for Amryt’s RDEB Gene Therapy Candidate, AP103 Sep 15, 2020
- Amryt Eyes Approval for Epidermolysis Bullosa Drug Following Phase III Data Sep 9, 2020
- Amryt Announces Positive Top Line Results from Pivotal Phase 3 “EASE” Trial of Filsuvez® in Epidermolysis Bullosa Sep 9, 2020
- Abeona Therapeutics to provide update on EB-101 program at upcoming virtual debra Care Conference (DCC) on September 8th Sep 3, 2020
- Second patient treated in Abeona’s EB-101 pivotal Phase 3 VIITAL™ study for recessive dystrophic epidermolysis bullosa (RDEB) Aug 10, 2020
- Amryt Pharma to Host Key Opinion Leader Call on Epidermolysis Bullosa Disease Landscape Jul 29, 2020
- Collagen degree: Bigger-wound treatment by Abeona key in RDEB? Jul 29, 2020
- Krystal Biotech Announces Initiation of Pivotal Phase 3 Study of Beremagene Geperpavec (B-VEC, previously KB103) in Patients with Dystrophic Epidermolysis Bullosa Jul 28, 2020
- Sneak Peek: EBconnect.org [Video] Jul 17, 2020
- BridgeBio Pharma affiliate to provide interim update on Phase 1/2 trial of PTR-01 (BBP-589), a collagen 7 protein replacement therapy, in patients with recessive dystrophic epidermolysis bullosa Jul 10, 2020
- Abeona Therapeutics Announces Two Presentations Related to Its Recessive Dystrophic Epidermolysis Bullosa (RDEB) Clinical Program at the SPD 45th Annual Meeting Jul 10, 2020
- IV Immunoglobulin May Help Treat Inflammation in Severe EBP, Case Report Suggests Jul 8, 2020
- Abeona Therapeutics Reinitiates Enrollment in EB-101 Pivotal Phase 3 VIITAL™ Study in RDEB after COVID-19 Related Pause Jul 8, 2020
- InMed announces dosing initiation in 2nd Phase 1 trial of INM-755, a topical CBN-based (cannabinol) cream to potentially treat epidermolysis bullosa Jul 7, 2020
- SD-101 Cream Lacks Wound Healing Superiority in Phase 3 Trial, Study Reports Jul 1, 2020
- Pipeline progress in epidermolysis bullosa Jun 24, 2020
- Small RNA molecules play a role in RDEB skin cancer development, study finds Jun 17, 2020
- BridgeBio Pharma’s Phoenix Tissue Repair Provides Updates to its Recessive Dystrophic Epidermolysis Bullosa (RDEB) Program (PTR-01/BBP-589) May 13, 2020
- Filsuvez® Confirmed as Global Brand Name for AP101 May 7, 2020
- Phase 3 Trial of Gene Therapy B-VEC for DEB Expected to Start by June May 6, 2020
- A less common CRISPR-mediated repair strategy shows promise for both, RDEB and DDEB patients May 4, 2020
- InMed Receives CTA Approval for Second Phase 1 Clinical Trial with INM-755, a topical therapy to treat epidermolysis bullosa Apr 30, 2020
- Spray-on Gene Therapy for Accelerated Wound Healing in RDEB with RenovaCare SkinGun™ in New Study Underway at King’s College London Mar 30, 2020
- Abeona’s phase 3 RDEB trial back in action Mar 17, 2020
- Abeona Therapeutics Announces First Patient Treated in Pivotal Phase III Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa Mar 17, 2020
- The Company Using Gene Therapy to Cure Rare Skin Conditions: Castle Creek Biosciences is developing treatments such as FCX-007 that may provide relief for “Butterfly Children” with RDEB Feb 24, 2020
- Stem Cell Therapy Considered Safe and Eases Symptoms in Adults With RDEB, Phase 1/2 Trial Shows Feb 19, 2020
- Aegle Therapeutics Announces $4M Financing to Fund Groundbreaking Stem Cell Exosome AGLE-102 Clinical Trial to Treat Orphan Disease Dystrophic Epidermolysis Bullosa Jan 7, 2020
- FDA Grants Orphan Drug Designation to APR Applied Pharma Research’s Investigational Drug APR-TD011 for the Treatment of Epidermolysis Bullosa Dec 4, 2019
- Epidermolysis bullosa: FDA seeks to help development of new treatments Jul 3, 2019
Research
- Efficacy of Allogeneic Cord Blood Platelet Gel on Wounds of Dystrophic Epidermolysis Bullosa Patients after Pseudosyndactyly Surgery Oct 3, 2020
- Learning to pop blisters in epidermolysis bullosa with a simple model Sep 20, 2020
- QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa: a preclinical study Sep 16, 2020
- Raloxifene and n-Acetylcysteine Ameliorate TGF-Signalling in Fibroblasts from Patients with Recessive Dominant Epidermolysis Bullosa Sep 16, 2020
- Efficacy of topical ropivacaine in children and young adults with hereditary Epidermolysis Bullosa Sep 16, 2020
- Cells from discarded dressings differentiate chronic from acute wounds in patients with Epidermolysis Bullosa Sep 15, 2020
- Apparent Missense Variant in COL7A1 Causes a Severe Form of Recessive Dystrophic Epidermolysis Bullosa via Effects on Splicing Sep 14, 2020
- Cellular therapy options for genetic skin disorders with a focus on recessive dystrophic epidermolysis bullosa Sep 4, 2020
- Slac2-b co-ordinates extracellular vesicle secretion to regulate keratinocyte adhesion and migration Sep 2, 2020
- Losartan as disease modulating therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Sep 2, 2020
- Immune tolerance of allogeneic hematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds Aug 31, 2020
- Prevalence, pathophysiology and management of itch in epidermolysis bullosa Aug 18, 2020
- The G Protein-Coupled Receptor (GPR) 15 Counteracts Antibody-Mediated Skin Inflammation Aug 14, 2020
- 12/15-Lipoxygenase choreographs the resolution of IgG-mediated skin inflammation Aug 4, 2020
- Disease-associated keratin mutations reduce traction forces and compromise adhesion and collective migration Jul 29, 2020
- The Role of the Renin-Angiotensin System (RAS) in Skin Physiology and Pathophysiology in Various Skin Diseases Such as Dystrophic Epidermolysis Bullosa Jul 22, 2020
- Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 SD-101 ESSENCE Study Jul 21, 2020
- Enteral iron absorption in patients with recessive dystrophic epidermolysis bullosa Jul 17, 2020
- Profiling trial burden and patients’ attitudes to improve clinical research in epidermolysis bullosa Jul 10, 2020
- Inherited epidermolysis bullosa: update on the clinical and genetic aspects Jul 8, 2020
- Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: not more effective than current standard of care Jun 23, 2020
- Novel biallelic variants in COL7A1 cause recessive dystrophic epidermolysis bullosa Jun 14, 2020
- Intravenous Injection of Muse Cells as a Potential Therapeutic Approach for Epidermolysis Bullosa Jun 12, 2020
- Traditional and advanced therapeutic modalities for wounds in the paediatric population: an evidence-based review Jun 12, 2020
- Epidermolysis Bullosa Pruriginosa successfully treated with combination of topical and systemic agents Jun 10, 2020
- Promising effect of intravenous immunoglobulin therapy for epidermolysis bullosa pruriginosa Jun 7, 2020
- Gastrointestinal involvement of primary skin diseases, including epidermolysis bullosa acquisita (EBA) May 26, 2020
- Propranolol is an effective topical and systemic treatment option for experimental epidermolysis bullosa acquisita May 22, 2020
- Exposed CendR Domain in Homing Peptide Yields Skin-Targeted Therapeutic in Epidermolysis Bullosa May 20, 2020
- Meeting Report: The First Global Congress on Epidermolysis Bullosa, EB2020 London – Toward Treatment and Cure May 15, 2020
- Platelet-rich-plasma injection and becaplermin gel as effective dressing adjuvants for treating chronic non-healing ulcers in patients with junctional epidermolysis bullosa May 11, 2020
- New Treatment of Wound Healing With Allogenic Acellular Human Skin Graft: Preclinical Assessment and In Vitro Study Apr 24, 2020
- Molecular Therapeutics in Development for Epidermolysis Bullosa: Update 2020 Apr 23, 2020
- One of the safer approaches to establish gene and cell therapies for future clinical use in hereditary skin disorders such as recessive dystrophic epidermolysis bullosa (RDEB) Apr 23, 2020
- Transplantation of a New Biological Product in Rare Diseases, Such as Epidermolysis Bullosa: Response and Clinical Outcome Apr 22, 2020
- Leading edge: emerging drug, cell, and gene therapies for junctional epidermolysis bullosa Mar 20, 2020
- Gentamicin Induces Laminin 332 and Improves Wound Healing in Junctional Epidermolysis Bullosa Patients with Nonsense Mutations Mar 17, 2020
- The photobiomodulation therapy together with the use of cord blood platelet gel could be safely suggested as primary treatment for oral lesions in patients with inherited epidermolysis bullosa Mar 16, 2020
- Predictable CRISPR/Cas9-Mediated COL7A1 Reframing for Dystrophic Epidermolysis Bullosa [PDF] Mar 3, 2020
- Collagen 17 (COL17) Processing and Blistering Skin Diseases Feb 12, 2020
- Consensus reclassification of inherited epidermolysis bullosa and other disorders with skin fragility Feb 4, 2020
- Treatment with anti-neonatal Fc receptor (FcRn) antibody ameliorates experimental epidermolysis bullosa acquisita in mice Jan 23, 2020
- Genomics‐based treatment in a patient with two overlapping heritable skin disorders: Epidermolysis bullosa and acrodermatitis enteropathica Jan 12, 2020
- Dressing material in children with epidermolysis bullosa: A qualitative study on experiences of parents and affected children Jan 8, 2020
- Toward treatment and cure of epidermolysis bullosa Dec 11, 2019
- CRISPR/Cas9-based targeted genome editing for correction of recessive dystrophic epidermolysis bullosa using iPS cells Dec 9, 2019
- Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa Nov 28, 2019
- Management of Recessive Dystrophic Epidermolysis Bullosa in a Newborn with Porcine-derived Extracellular Matrix Nov 12, 2019
- Thymosin β4: potential to treat epidermolysis bullosa and other severe dermal injuries Oct 1, 2019
- EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa Sep 26, 2019
- Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullosa Sep 19, 2019
Clinical Trials
- Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB [Completed] Aug 20, 2020
- A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (DEFI-RDEB) Aug 4, 2020
- A Phase 1/2 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Aug 3, 2020
- A Phase 2 Open-Label Study of PTR-01 in Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Aug 3, 2020
- Injections of Botulinic Toxin in Plantar Lesions of Localized Epidermolysis Bullosa Simplex Aug 3, 2020
- Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB Jul 31, 2020
- Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17) Jul 31, 2020
- A Phase 3 Double Blinded, Placebo-Controlled, Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, Previously “KB103”) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) Jul 29, 2020
- Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex [Completed] Jul 27, 2020
- Evaluation of the Efficacy of ROPIVACAINE in Children and Young Adults With Hereditary Epidermolysis Bullosa (EBROPI) Jul 9, 2020
- A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Jul 2, 2020
- Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa [Completed] Jun 16, 2020
- Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC May 28, 2020
- Phase 3 Efficacy and Safety Study of Oleogel-S10 in Patients with Inherited Epidermolysis Bullosa Apr 24, 2020
- Intravenous Gentamicin Therapy for Restoration of Full-Length Type VII Collagen in Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) Apr 7, 2020
- MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions Apr 2, 2020
- Rigosertib for RDEB-SCC Mar 24, 2020
- Safety Study of the Administration of MSC Extracellular Vesicles (AGLE-102) in the Treatment of Dystrophic Epidermolysis Bullosa Wounds Mar 24, 2020
- Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa (GEM-1) Mar 13, 2020
- Topical QR-313 in Dominant Dystrophic Epidermolysis Bullosa (DDEB) or Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene (WINGS) Feb 26, 2020
- Allogeneic ABCB5-positive Stem Cells (allo-APZ2-E) for Treatment of Epidermolysis Bullosa Jan 30, 2020
- Ex Vivo Gene Therapy Clinical Trial for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using Genetically Corrected Autologous Skin Equivalent Grafts (EBGraft) Jan 28, 2020
- Self-Assembled Skin Substitute for the Treatment of Epidermolysis Bullosa (SASS) Jan 27, 2020
- Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES) Sep 19, 2019
- Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa (CELEB) Aug 28, 2019