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| Stem definition | Drug id | CAS RN |
|---|---|---|
| 5855 | 2241888-62-8 |
| Molecule | Description |
|---|---|
|
Synonyms:
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Beremagene geperpavec is a gene therapy based on an engineered, replication-defective herpes simplex virus 1 (HSV-1) encoded with COL7A1 gene, addressing the underlying genetic cause of dystrophic epidermolysis bullosa. The HSV-1 vector belongs to the human herpes virus (HHV) family of double-stranded DNA viruses. Upon cutaneous application to the wounds, beremagene geperpavec can transduce both keratinocytes and fibroblasts. Following entry of beremagene geperpavec into the cells, the vector genome is deposited in the nucleus without integrating into, or otherwise disrupting, host cell DNA. Once in the nucleus, transcription of the encoded human COL7A1 is initiated. The resulting transcripts allow for production and secretion of COL7 by the cell in its mature form. These COL7 molecules arrange themselves into long, thin bundles that form anchoring fibrils. The anchoring fibrils hold the epidermis and dermis together and are essential for maintaining the integrity of the skin.
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| April 23, 2025 | EMA | Krystal Biotech Netherlands B.V. |
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| Source | Code | Description |
|---|---|---|
| ATC | D03AX16 | DERMATOLOGICALS PREPARATIONS FOR TREATMENT OF WOUNDS AND ULCERS CICATRIZANTS Other cicatrizants |
| CHEBI has role | CHEBI:49201 | anti-ulcer drug |
| CHEBI has role | CHEBI:73336 | vulnerary |
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Dystrophic epidermolysis bullosa | indication | 61003004 | DOID:2730 |
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| Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
|---|---|---|---|---|---|---|---|---|---|
| Collagen alpha-1(VII) chain | Adhesion | OTHER | UNKNOWN | DRUG LABEL |
| ID | Source |
|---|---|
| CHEMBL4594328 | ChEMBL_ID |
| C000731813 | MESH_SUPPLEMENTAL_RECORD_UI |
| 4042305 | VANDF |
| C5420879 | UMLSCUI |
| CHEBI:757826 | CHEBI |
| 11345 | INN_ID |
| AQN7K24KQU | UNII |
| 2637758 | RXNORM |
| D12632 | KEGG_DRUG |
| 370288 | MMSL |
| 41894 | MMSL |
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