Beremagene geperpavec API Manufacturers

General Description:

Beremagene geperpavec, identified by CAS number 2241888-62-8, is a notable compound with significant therapeutic applications. Beremagene geperpavec is a live, replication-defective herpes simplex virus type 1 (HSV-1)-based vector therapy. Developed by Krystal Biotech, it was first approved by the FDA on May 19, 2023, for the treatment of wounds associated with dystrophic epidermolysis bullosa (DEB). DEB is caused by mutations in the _COL7A1_ gene that encodes collagen VII (COL7), a major component of the anchoring fibrils for dermal–epidermal cohesion. Beremagene geperpavec is genetically modified to deliver _COL7A1_, thereby restoring the levels of the COL7 protein.

Indications:

This drug is primarily indicated for: Beremagene geperpavec is indicated for the treatment of wounds in patients six months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. Its use in specific medical scenarios underscores its importance in the therapeutic landscape.

Metabolism:

Beremagene geperpavec undergoes metabolic processing primarily in: There is no information available. This metabolic pathway ensures efficient processing of the drug, helping to minimize potential toxicity and side effects.

Absorption:

The absorption characteristics of Beremagene geperpavec are crucial for its therapeutic efficacy: There is limited information available regarding the absorption of beremagene geperpavec. In an initial clinical study, viral vector DNA was detected in skin swab samples in all nine treated subjects, with a maximum level ranging from 5.1×10⁴ to 4.1×10⁸ vector genomes. The drug's ability to rapidly penetrate into cells ensures quick onset of action.

Half-life:

The half-life of Beremagene geperpavec is an important consideration for its dosing schedule: There is no information available. This determines the duration of action and helps in formulating effective dosing regimens.

Protein Binding:

Beremagene geperpavec exhibits a strong affinity for binding with plasma proteins: There is no information available. This property plays a key role in the drug's pharmacokinetics and distribution within the body.

Route of Elimination:

The elimination of Beremagene geperpavec from the body primarily occurs through: There is limited information available. In an initial clinical study involving subjects who received beremagene geperpavec via skin swabs, no viral vector DNA was detected in blood or urine. Understanding this pathway is essential for assessing potential drug accumulation and toxicity risks.

Volume of Distribution:

Beremagene geperpavec is distributed throughout the body with a volume of distribution of: There is no information available. This metric indicates how extensively the drug permeates into body tissues.

Clearance:

The clearance rate of Beremagene geperpavec is a critical factor in determining its safe and effective dosage: There is no information available. It reflects the efficiency with which the drug is removed from the systemic circulation.

Pharmacodynamics:

Beremagene geperpavec exerts its therapeutic effects through: Beremagene geperpavec is a gene therapy that restores the deficient levels of human type VII collagen (COL7) protein. In RDEB mice and human RDEB xenografts, beremagene geperpavec restored C7 expression in keratinocytes and fibroblasts. In a trial comprising patients with dystrophic epidermolysis bullosa, the topical administration of beremagene geperpavec was associated with greater wound healing at three and six months compared to placebo. The drug's ability to modulate various physiological processes underscores its efficacy in treating specific conditions.

Mechanism of Action:

Beremagene geperpavec functions by: Collagen VII (C7, COL7), encoded by the _COL7A1_ gene, is an anchoring fibril component that holds the epidermis and dermis together to maintain skin integrity. COL7 is synthesized by epidermal keratinocytes and dermal fibroblasts. Dystrophic epidermolysis bullosa (DEB) is an inherited disorder caused by _COL7A1_ gene mutations, leading to reduced or deficient levels of biologically active and functional COL7. Deficient levels of COL7 result in no functional anchoring fibrils and impaired dermal-epidermal adhesion. DEB is associated with blistering, wounding, and scarring of the skin and other organs starting at birth. Beremagene geperpavec is a herpes simplex virus type 1 (HSV-1)-based vector genetically modified to express COL7. Upon topical application to wounds, beremagene geperpavec enters both keratinocytes and fibroblasts and the vector genome is deposited in the nucleus. Once in the nucleus, transcription of the encoded human COL7A1 is initiated to produce and secrete COL7 by the cell in its mature form. COL7 molecules arrange themselves into long, thin bundles that form anchoring fibrils. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.

Toxicity:

Categories:

Beremagene geperpavec is categorized under the following therapeutic classes: Administration, Topical, Cellular and Gene Therapy. These classifications highlight the drug's diverse therapeutic applications and its importance in treating various conditions.