October 8, 2020

Impact: Moderate

  • Krystal Biotech announced the presentation of preclinical data supporting the ongoing development of KB301. Data will be presented at the American Society for Dermatologic Surgery (ASDS) 2020 Virtual Meeting to be held October 9-11, 2020.
  • KB301 is an investigational therapy designed to restore collagen homeostasis locally via directed expression of full-length human type III collagen gene (COL3A1).
  • Poster titled, In Vitro and In Vivo Pharmacology of KB301, an HSV-1-Based Gene Therapy, for the Treatment of Superficial Skin Depressions highlights that:
    • In vitro, KB301 readily transduced primary aged male and female human dermal fibroblasts (HDFs) and induced full-length COL3 expression, proper maturation, and subsequent secretion.
    • In vivo, successful vector transduction and subsequent COL3 expression was observed in a dose-dependent manner at both the transcript and protein levels in young and aged immunocompetent mice.
    • Safety looks promising as high concentrations of KB301 were non-cytotoxic to the cells in vitro.
  • A phase 1 trial, PEARL-1, is currently recruiting and expects to enroll 22 patients. The study will evaluate the safety and tolerability and initial efficacy of 2 different dose levels of KB301 injections in healthy buttock skin (NCT04540900).

inThought Analysis

KB-301 is a gene therapy that uses Krystal Biotech’s Skin TARgeted Delivery platform (STAR-D) that consists of a modified herpes simplex virus-1 vector (HSV-1) vector and skin optimized gene transfer technology. HSV-1 is a replication-defective, non-integrating viral vector that can efficiently penetrate a broad range of skin cells, making this vector significantly better than others. It can be applied topically or through intradermal injections. Clinical data of Krystal Biotech’s other STAR-D based treatments has been positive in rare orphan skin diseases.

Source: Krystal Biotech Press Release