Abeona Therapeutics | EB-101 | Autologous ex-vivo COL7A1 gene-corrected cell therapy (transplant) | RDEB large non-healing chronic wounds | Surgical graft | BLA |
Castle Creek (Fibrocell) | FCX-007 (D-Fi) | Ex-vivo autologous cell-based lentiviral vector COL7A1 gene therapy | RDEB | Intradermal injection | III |
RHEACELL GmbH & Co. KG | AMESANAR | Allogeneic ABCB5+ MSCs | RDEB | Intravenous | III |
InMed Pharmaceuticals | INM-755 | Cannabinol formulation | EB, all types | Topical | II |
Relief Therapeutics | RLF-TD011 (Nexodyn) | Hypochlorous acid formulation/Antibacterial | EB, all types | Spray | II |
Quoin Pharmaceuticals | QRX004 | Read-through of nonsense mutations | RDEB | Topical | II |
Phoenicis Therapeutics | PTW-002 | Anti-sense oligonucleotide exon skipping | DDEB and RDEB | Topical | II |
Shionogi | Redasemtide | Peptide analogue of HMGB1, stem cell modulation | DEB | Intravenous | II |
Aegle Therapeutics | AGLE 102 | Allogeneic extracellular vesicles from MSCs | DEB | Topical | I/II |
Eloxx Pharmaceuticals | ZKN-013 | Ribosome modulating agent for non-sense readthrough | RDEB, JEB | Oral | IND-C |
Amryt Pharma | AP103 | Corrected collagen VII gene delivered using HPAE as non-viral vector | DEB | Topical | Preclin |
Constant Therapeutics | TXA127 | Angiotensin-(1-7) | EB | Oral | Preclin |
Branca Bunus | BrB-101 | CRISPR-Cas9 to remove COL7A1 mutations in exon80 | RDEB exon 80 | - | Preclin |
Telaria (Replay Bio) | - | synHSV (HSV-1 vector) | DEB | - | Disc? |