Treatment projects

The current research project, funded by DEBRA Research and led by Dr Yanling Liao at New York Medical College, seeks to compare the effects of different IL-1 blockers on survival as well as the cellular and molecular changes resulting from their administration in the hypomorphic mouse, a murine model of recessive dystrophic epidermolysis bullosa (RDEB). Recent work by Dr Yanling Liao and collaborators (DOI: 10.1016/j.jid.2025.01.010) has demonstrated that genetic ablation of IL-1R1, the primary signalling protein complex that mediates inflammatory and immune responses by binding IL-1 cytokines, attenuates inflammation and prolongs the survival of hypomorphic mice. In particular, the expression of IL-1alpha, a pro-inflammatory cytokine, was significantly elevated early in this model. Inhibiting signaling through this receptor might help reduce chronic inflammation, pro-fibrotic cytokines, and inflammatory pain, all clinical signs associated with RDEB.

DEBRA Research is funding a proof-of-concept project led by Professor John Marshall at Queen Mary University of London to develop targeted delivery strategies for epidermolysis bullosa (EB). The project focuses on integrin αvβ6, a wound-healing receptor that is minimally expressed in healthy skin but upregulated in EB blisters and EB-associated squamous cell carcinomas, making it a promising therapeutic target.

Using a high-affinity αvβ6-binding peptide, the team will systemically compare three different delivery systems side-by-side. This project aims to identify the most effective and selective approach for skin-directed gene therapy in EB, laying key groundwork for future translational development.

DEBRA Research is funding a research project to model recessive dystrophic epidermolysis bullosa (RDEB) using an advanced skin organoid-on-chip (SKO-chip) platform that closely reflects human skin structure and function. Led by Karl Köhler at Boston Children’s Hospital, the project supports the study of disease mechanisms and tissue repair in a controlled, physiologically relevant environment.

The SKO-chip enables live imaging, spatial control, and the integration of immune or therapeutic components, allowing researchers to monitor tissue responses to injury and treatment. A key objective is the development of a new open-top chip design, which improves optical access and enables direct tissue manipulation for topical drug testing, immune cell seeding, and wound modeling.

Nonsense mutations in key structural skin proteins can prevent proper protein production and cause several forms of EB. Among these, mutations in collagen VII cause the dystrophic forms of the disease.

ReadOn Therapeutics gGmbH, a non-profit subsidiary of DEBRA Research created in partnership with Nonsense Therapeutics Ltd., focuses on pre-competitive drug development of read-through therapies for EB. By advancing this drug class independently of commercial priorities, ReadOn works to bring the most promising therapeutic candidates closer to clinical translation and, ultimately, to people living with EB.

Learn more about our investment in ReadOn Therapeutics.

This clinical observation project, conducted in collaboration with EB House Austria, will explore the feasibility and potential benefit of non-invasive transcutaneous vagus nerve stimulation (tVNS) using a CE-certified device approved for the treatment of sleep disturbances. Sleep disturbances are a major and often overlooked burden for people living with Epidermolysis Bullosa (EB). Chronic pain, itching, and inflammation can severely impair sleep quality, limiting recovery and negatively affecting overall quality of life. The project aims to generate initial real-world evidence on whether tVNS could represent a safe and practical supportive approach to improving sleep in EB.

DEBRA Research has invested in Nova Anchora, a US-based biotech company developing protein replacement therapies for dystrophic EB (DEB). Its lead program, NvA-011, is a recombinant type VII collagen therapy designed to restore the defective protein in DEB. 

By supporting this program alongside other mission-aligned partners, DEBRA Research supports the advancement of a targeted, topical therapy aimed at directly addressing the underlying cause of DEB. By combining philanthropic and strategic investment, this collaboration helps accelerate the development of innovative treatments that bring hope for people affected by severe forms of EB.

Learn more about our investment in Nova Anchora.

DEBRA Research has entered a strategic collaboration and investment partnership with Dermaliq Therapeutics, a US-based clinical-stage company developing next-generation topical therapies using its proprietary hyliQ® technology platform. The partnership focuses on improving cutaneous drug delivery and advancing innovative, touchless topical treatments for people living with EB, targeting key symptoms such as wound healing, itch and prevention of new skin lesions. 

Dermaliq’s hyliQ® technology enables non-contact delivery of a wide range of therapeutic compounds directly to the skin and target cells. This approach could represent a major step forward in the delivery of future EB therapies. By supporting this, we help accelerate the development of new formulations and topical therapies that can improve both comfort and quality of life. 

Learn more about our investment in Dermaliq Therapeutics.

DEBRA Research has invested in Eliksa Therapeutics, a US-based biotechnology company developing ELK-003, a biological eye drop designed to treat ocular complications in people living with EB. Developed using a proprietary amniotic-fluid-derived formulation, ELK-003 aims to protect and repair the corneal surface. It is currently being evaluated in a clinical study conducted in collaboration with DEBRA Chile.

This joint investment, made with several EB-focused organizations, supports clinical development of a therapy targeting one of the most painful and underserved manifestations of EB.

Learn more about our investment in Eliksa Therapeutics.

Healome Therapeutics Ltd., a UK based therapeutic startup develops a proprietary eye drop matrix. Enhancing effectiveness of eye drops is achieved by improved retention time on the eye leading to improved drug delivery, protection, lubrication and healing. Healome’s technology provides for more effective and user-friendly eye drop formulations having the potential for a wide application ranging from healthy cornea to diseased / damaged corneal environments.

DEBRA Research’s investment supports Healome with the aim to develop technologies to effectively deliver drugs to the eye to provide for treatment solution for EB patients suffering from complications in the eye including corneal abrasions, one of the most severe manifestations of the disease.

DEBRA Research has invested in Mariposa Therapeutics Ltd., a UK-based biotechnology company developing a novel small-molecule therapy for EB Simplex (EBS). Mariposa’s lead candidate, MP5219, is a topical treatment designed to improve skin stability by activating protective keratin pathways and strengthening the structural resilience of keratinocytes, addressing the underlying disease mechanism in EBS 

Mariposa is currently in late preclinical development, working toward IND-enabling studies and first-in-human clinical evaluation. Through this investment, DEBRA Research supports the advancement of a first-in-class, mechanism-based approach with the potential to meaningfully improve daily disease burden for people living with EBS.