Transforming the Treatment of Rare Genetic Skin Diseases
Rinascera Therapeutics is a clinical-stage biotechnology company developing transformative therapies for patients living with rare genetic skin diseases, starting with RIN-001, a topical cancer interception therapy for Gorlin Syndrome, and RIN-002, a systemic collagen VII replacement therapy for Dystrophic Epidermolysis Bullosa (DEB).
Our mission is to give patients and their families a genuine second chance at life.
A Name That Means Renewal
Inspired by the Italian verb rinascere ("to be reborn") and the Latin root renasci ("to be born again"), our name embodies our commitment to developing transformative therapies that restore hope, function and possibility for patients and families facing lifelong disease.
Our Mission
Our mission is to give patients and their families a genuine second chance at life.
Our Guiding Principles
Scientific Rigor
Every decision is grounded in deep understanding of biology and clinical evidence.
Patient-First Timelines
We move with urgency while never compromising on safety or quality.
Collaborative Spirit
Partnering with leading researchers, clinicians, and patient advocates.
Renewal Mindset
True to our name—embracing innovation to transform patient outcomes.
Two Modalities
RIN-001 is a topical cancer interception therapy being developed for Gorlin Syndrome and high-frequency sporadic basal cell carcinoma populations, while RIN-002 is a systemic recombinant collagen VII replacement therapy being developed for Dystrophic Epidermolysis Bullosa (DEB). Both programs are grounded in well-defined disease biology and designed to address substantial unmet medical needs.
Genetically Defined Targets, Modality-Specific Delivery
RIN-001 (Gorlin Syndrome): a novel topical kinase inhibitor being developed as a cancer interception therapy that inhibits multiple downstream signaling pathways essential for the survival and growth of emerging BCC cells. Rather than targeting a single upstream driver, RIN-001 is designed to intercept cancer at its earliest stages—eliminating subclinical BCCs while they remain confined to superficial skin layers, before they develop into tumors requiring surgery. This shifts care from surgically removing tumors after they appear to treating them before they progress to visible tumors in the first place, and is particularly well suited for Gorlin Syndrome and high-frequency sporadic BCC populations, who continuously develop new tumors and often undergo numerous surgical procedures.
RIN-002 (DEB): An intravenous recombinant collagen VII replacement therapy designed to address the underlying loss of functional collagen VII systemically. Unlike localized approaches intended to treat individual wounds, Rinascera's strategy for RIN-002 is to advance an intravenous protein replacement therapy capable of targeting the cutaneous and systemic manifestations of DEB.
Strategic Rationale: Each program's route of administration is matched to its disease biology—topical cancer interception for localized Gorlin BCCs, and systemic IV replacement for DEB, a systemic collagen VII deficiency that demands a systemic therapy.
Cancer Interception (Gorlin Syndrome)
RIN-001 is a first-in-class topical kinase inhibitor being developed as a cancer interception therapy that inhibits multiple downstream signaling pathways essential for the survival and growth of emerging BCC cells in Gorlin Syndrome.
COL7A1 / Collagen VII (DEB)
An intravenous recombinant collagen VII replacement therapy designed to address the underlying loss of functional collagen VII systemically, targeting the cutaneous and systemic manifestations of Dystrophic Epidermolysis Bullosa (DEB).
Clinical Development Programs
Two rare disease programs with clinical proof-of-concept data generated and Phase 3 trials planned—targeting Gorlin Syndrome and Dystrophic Epidermolysis Bullosa (DEB).
| Program | Indication | Discovery | Preclinical | Phase 2 | Phase 3 | Approval |
|---|---|---|---|---|---|---|
| RIN-001 | Gorlin Syndrome | |||||
| RIN-002 | Dystrophic Epidermolysis Bullosa (DEB) |
Gorlin Syndrome
A novel topical kinase inhibitor being developed as a cancer interception therapy that inhibits multiple downstream signaling pathways essential for the survival and growth of emerging BCC cells.
- Clinical proof-of-concept data generated, Phase 3 trial planned
- Rather than targeting a single upstream driver, designed to intercept cancer at its earliest stages—eliminating subclinical lesions while confined to superficial skin layers, before they develop into tumors requiring surgery
- Shifts care from surgically removing tumors after they appear to treating them before they progress to visible tumors in the first place
- Particularly well suited for Gorlin Syndrome and high-frequency sporadic BCC populations, who continuously develop new tumors and often undergo numerous surgical procedures
Dystrophic Epidermolysis Bullosa (DEB)
An intravenous recombinant collagen VII replacement therapy designed to address the underlying loss of functional collagen VII systemically. Unlike localized approaches intended to treat individual wounds, Rinascera's strategy for RIN-002 is to advance an intravenous protein replacement therapy capable of targeting the cutaneous and systemic manifestations of DEB.
- Clinical proof-of-concept data generated, Phase 3 trial planned
- Uniquely systemic recombinant collagen VII replacement delivered via intravenous infusion
- Designed to treat DEB as a systemic disease—addressing collagen VII deficiency across skin and internal epithelial tissues, not individual wounds
- Acquired from BridgeBio; builds on substantial prior development work
Pipeline status reflects current development plans and is subject to change based on clinical data and regulatory interactions.
The Human Impact of Rare Skin Diseases
Gorlin Syndrome patients undergo an average of 10+ surgeries per year by their 40s. DEB patients spend hours daily in wound care, face a dramatically shortened lifespan, and carry a 90% lifetime risk of aggressive squamous cell carcinoma.
"People living with diseases like Gorlin Syndrome and DEB are not asking for marginal improvements. They are asking for lives with less pain, fewer surgeries, fewer wounds, fewer limitations and more possibility."— Nichola Eliovits, Co-founder & CEO
Gorlin Syndrome · RIN-001
RIN-001 is designed to intercept BCCs before they require surgical removal—reducing the need for repeated operations in Gorlin syndrome patients.
A topical formulation designed for convenient at-home use, empowering patients to manage their own care.
DEB · RIN-002
RIN-002 is uniquely systemic—addressing the underlying collagen VII deficiency across skin and internal epithelial tissues, treating DEB as the systemic disease it is rather than individual wounds.
Committed to therapies that reduce chronic wound burden and improve quality of life across the patient journey.
Gorlin Syndrome
Estimated US patients
DEB
Estimated US patients
Let's Connect
We welcome inquiries from investors, potential partners, researchers, and patient advocacy organizations.
Headquarters
Boston, MA
United States