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Krystal Biotech (NASDAQ:KRYS) has announced it has begun its Phase 1/2 study of KB105 in transglutaminase-1 (TGM1) deficient autosomal recessive congenital ichthyosis (ARCI). As quoted in the press release: The study, termed GEM-3 study, will examine KB105, an HSV-1 based gene therapy engineered to deliver a human TGM1 gene, in patients with ARCI. Up to …
Krystal Biotech (NASDAQ:KRYS) has announced it has begun its Phase 1/2 study of KB105 in transglutaminase-1 (TGM1) deficient autosomal recessive congenital ichthyosis (ARCI).
As quoted in the press release:
The study, termed GEM-3 study, will examine KB105, an HSV-1 based gene therapy engineered to deliver a human TGM1 gene, in patients with ARCI.
Up to six subjects are presently planned for the study. Pediatric patients will be enrolled in the study following establishment of safety in adult subjects. This study is an intra-patient comparison of KB105 and placebo-administered target areas. Patients will be evaluated for safety, and target areas will be assessed individually with the Investigator Global Assessment (IGA) and Visual Index of Ichthyosis Severity (VIIS) scales. Target areas will be imaged and evaluated for safety and efficacy and subjects will be on-trial for approximately 3.5 months.
TGM1-deficient ARCI is a debilitating rare skin disease characterized by excessive, thick scaling of the skin, causing multiple chronic health conditions. There are approximately 23,000 cases of TGM1-deficient ARCI worldwide and about 400 new cases per year globally. Krystal’s approach is to use a non-replicating, non-integrating engineered HSV-1 vector to deliver the TGM1 gene to keratinocyte skin cells, potentially allowing them to produce the TGM1 protein that is lacking in this patient population. KB105 is designed to be an off-the-shelf treatment for TGM1-deficient ARCI that can be applied topically, directly to a patient’s skin.
“TGM1-deficient ARCI is a debilitating condition that drastically affects patient’s lives and has no current treatment options beyond palliative care,” said Pooja Agarwal, vice president, product development of Krystal. “KB105, based on our proprietary topically-applied gene therapy platform technology, has the potential to be the first-ever treatment for people with TGM1-deficient ARCI.”