Last updated September 17, 2026

Epithelica

Developing topical gene-editing therapies for inherited skin diseases

Gene TherapyRare DiseaseDermatology

Autosomal recessive congenital ichthyosis (ARCI) is a group of rare inherited disorders that weaken the skin barrier, causing severe scaling, water loss and infection risk. Current care is lifelong and symptom-focused, relying on frequent bathing, moisturisers and retinoids rather than correcting the underlying mutation.

Epithelica is developing topical gene-editing therapies designed to correct disease-causing mutations directly in skin cells. Its lead ARCI approach uses a to create temporary micropores, allowing lipid nanoparticles carrying a to reach the living epidermis.

Key Investor Signals

Published study restored ~30% of normal TGM1 activity

A 2026 study corrected the target TGM1 mutation in human ARCI skin models and restored ~30% of normal transglutaminase 1 enzyme activity.

Two PCT filings cover editing and skin delivery

Published PCT applications cover the TGM1 gene-editing method and lipid-nanoparticle skin delivery, giving the programme IP around both correction and local administration.

TGM1 program has FDA and EU orphan designations

The TGM1-targeted ARCI program received EU orphan designation in December 2024 and FDA in March 2025.

Other Positive Signals

  • Repeat-dose testing found no systemic distribution of the lipid nanoparticles or genetic cargo beyond the skin.
  • Comprehensive off-target analysis found only negligible unintended DNA editing outside the intended target.
  • Co-founder Sarah Hedtrich holds a in Human Disease Models at UBC.

Team

Friedrich Hedtrich

Co-Founder and Chief Executive Officer

  • Managed Deutsche Bahn's DB mindbox accelerator from 2016 to 2019, running real-world startup proof-of-concepts with business units.
  • Created and managed the innovation hub at Germany's Federal Criminal Police Office, working with internal and external innovators.
  • Spent four years as a senior management consultant, leading software and process-improvement projects across multiple industries.
  • Has mentored founders through entrepreneurship@UBC, the University of British Columbia's venture-creation programme, since 2019.

Sarah Hedtrich

Co-Founder and Chief Scientific Officer

  • Leads the Hedtrich Lab at UBC, developing human epithelial disease models and next-generation therapies for skin and lung disease.
  • Named inventor on PCT applications covering TGM1 base editing and lipid-nanoparticle skin delivery.
  • Tier 1 Canada Research Chair in Human Disease Models and Associate Professor at UBC's School of Biomedical Engineering.

Product

Epithelica's lead programme combines topical lipid nanoparticles with base editing and laser-created micropores in the skin. The approach is designed to correct disease-causing DNA locally in the epidermis without viral vectors or cell transplantation.

Laser-assisted topical delivery

A fractional dermatology laser creates temporary micropores through the skin barrier, allowing lipid nanoparticles to reach the living epidermis. The pores then close, creating a local delivery route using equipment already used in dermatology.

Edits DNA without double-strand cuts

The base editor rewrites the disease-causing DNA letter without cutting both strands. Delivered as mRNA with a , it is designed to restore the normal TGM1 sequence in skin cells.

Reaches skin-renewing basal cells

Single-cell analysis found about 40% of cells reached by the treatment were basal keratinocytes, which continually renew the epidermis. Reaching these cells supports the potential for durable local correction.

Market Overview

ARCI is a rare inherited skin disease that causes lifelong scaling and failure of the skin barrier. TGM1 mutations account for a large share of cases, while current management remains symptom-focused and requires continuous topical care rather than correcting the underlying genetic defect.

Topical genetic medicine for inherited skin disease already has commercial validation. is approved in the US, Europe and Japan, generated $389.1M in 2025 revenue, and had secured more than 660 US reimbursement approvals by year-end, providing a clear regulatory and reimbursement precedent.

Estimated ARCI prevalence
~1 in 100,000

Approximate prevalence of ARCI, the inherited skin-disease group targeted by Epithelica's lead programme.

ARCI cases linked to TGM1
32–68%

Published range for ARCI cases caused by TGM1 mutations, the gene targeted by Epithelica's lead programme.

Daily topical treatment use
87%

Share of patients with inherited ichthyosis applying topical treatment at least daily in a 2024 German study.

Incomplete topical coverage
81.9%

Share of patients whose health insurance did not fully cover topical treatment in a 2024 German study.

Comparable Outcomes

In vivo base editing

Verve Therapeutics

Acquired by Eli Lilly for up to $1.3B

developed in vivo base-editing medicines for cardiovascular disease, using lipid nanoparticles to deliver gene-editing cargo directly into the body. Eli Lilly acquired Verve in July 2025 for about $1.0 billion upfront plus a contingent value right, bringing total potential consideration to about $1.3 billion.

Topical skin gene therapy

Krystal Biotech

IPO at ~$98M market value

developed topical gene therapies for inherited skin disorders. VYJUVEK delivers a functional gene to skin wounds and became the first approved genetic medicine for . Krystal listed on Nasdaq in September 2017 at a fully diluted market value of about $98 million.

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Sources

This profile was built from public company materials, ecosystem sources, market references, and professional profiles.

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