KB105 / Krystal Biotech - LARVOL DELTA

Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI) (clinicaltrials.gov) - P2 | N=15 | Not yet recruiting | Sponsor: Krystal Biotech, Inc.

New P2 trial • Atopic Dermatitis • Dermatology • Immunology • Vitiligo

Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI) (clinicaltrials.gov) - P1/2; N=6; Active, not recruiting; Sponsor: Krystal Biotech, Inc.; Recruiting ➔ Active, not recruiting; Trial primary completion date: Mar 2020 ➔ Oct 2022

Clinical • Enrollment closed • Trial primary completion date • Atopic Dermatitis • Dermatology • Gene Therapies • Immunology • Vitiligo

[VIRTUAL] Engineered Herpes Simplex Virus Type 1 (HSV-1)-Based Vectors as a Platform for Localized Delivery of Therapeutic Antibodies in the Treatment of Skin Disorders (ASGCT 2021) - P1/2, P3 | "(Krystal) has leveraged its HSV-1-based STAR-D (Skin-Targeted Delivery) platform to deliver therapeutic transgenes to patients with debilitating diseases, including vectors beremagene geperpavec (“B-VEC”) - encoding human COL7A1 for the treatment of dystrophic epidermolysis bullosa (ClinicalTrials.gov Identifier: NCT04491604), and KB105 - encoding human TGM1 for the treatment of autosomal recessive congenital ichthyosis (Clinicaltrials.gov Identifier: NCT04047732)...To evaluate the ability of a vector-encoded antibody to reduce an AD-like phenotype on a macro-scale, KB502 was applied topically in an MC903-induced murine model of AD...At this time, Krystal has expanded its STAR-D-based antibody portfolio to at least 12 unique products, including vectors expressing anti-CD20, anti-IL-17, and anti-CCR4 antibodies. The studies described herein warrant the further investigation of STAR-D as a platform for localized delivery of therapeutic antibodies, including for..."

IO biomarker • Atopic Dermatitis • Dermatitis • Dermatology • Gene Therapies • Herpes Simplex • Immune Modulation • Immunology • Inflammation • Vitiligo • CCR4 • IL10 • IL17A

Preclinical evaluation of a modified HSV-1 vector encoding human TGM1 for the treatment of autosomal recessive congenital ichthyosis (ARCI). (PubMed, J Invest Dermatol) - "Toxicity and biodistribution assessments upon repeat-dosing indicated that KB105 was well tolerated and restricted to the dose site. Overall, our results demonstrate that rescuing TGM1 deficiency in ARCI patients via topical KB105 application represents a promising strategy for safely and non-invasively treating this debilitating disease."

Journal • Preclinical • Atopic Dermatitis • Dermatology • Fibrosis • Gene Therapies • Immunology • Inflammation • Vitiligo

[VIRTUAL] First in human use of a novel in vivo gene therapy for the treatment of autosomal recessive congenital ichthyosis: Results of a phase I/II placebo controlled trial (SID 2020) - P1/2 | "With safety and preliminary efficacy established, the next phase of the study will evaluate KB105 safety and efficacy in larger target areas and expand enrollment to pediatric subjects in 1H 2020. A multi-center pivotal Phase 3 study is planned following completion of Phase I/II study."

P1/2 data • Atopic Dermatitis • Dermatology • Dermatopathology • Immunology • Pediatrics • Vitiligo