# A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa

> **NIH FDA R01** · CASTLE CREEK BIOSCIENCES, LLC · 2021 · $500,000

## Abstract

7. PROJECT SUMMARY/ABSTRACT
Recessive dystrophic epidermolysis bullosa (RDEB), is an autosomal recessive, inherited skin
disease caused by mutations within the type VII collagen gene. The disease is characterized by
painful blisters and wounds on skin and mucous membranes due to lack of adhesion of the
epidermis to the inner dermal layers of the skin. The sequelae of wounds are often debilitating,
disfiguring, and sometimes fatal. RDEB patients have a reduced life expectancy with early death
resulting from infection, organ failure or squamous cell carcinoma (SCC). Current therapy for
RDEB is limited to palliative wound care as there are currently no approved drugs for RDEB.
Fibrocell Technologies, Inc. (FTI) is developing FCX-007, a suspension of live, ex-vivo,
autologous human dermal fibroblast cells that have been genetically modified using a lentiviral
vector (INXN-2004), which enables the expression of the human type VII (C7) protein. FTI has
an open Investigational New Drug Application (IND 016582) for FCX-007.
FTI proposes to continue its interventional, intra-patient randomized and controlled, open-label,
Phase 3 study to evaluate the efficacy, durability, and safety of FCX-007. FTI has worked
closely with the FDA in designing the Phase 3 clinical trial based on data gathered from the
Phase 1/2 trial (FI-EB-001). Clinical efficacy will be determined by blinded investigator
assessment of complete wound closure, durability of response, change in surface area of wound,
and a patient reported outcome of pain. Clinical safety will be assessed by testing for presence of
replication-competent lentivirus (RCL), type VII collagen autoantibody immune reactions,
neoplasms (squamous cell carcinoma (SCC)), as well as physical examinations and adverse
events (AEs). Efficacy and durability of FCX-007 will be assessed by the closure of wounds
from Week 12 through Week 48, which FTI believes represents a clinically meaningful benefit
to study subjects. FTI expects FCX-007 to be clinically safe given the FCX-007 Phase 1/2
clinical data (FI-EB-001), the preclinical safety data, and the autologous nature of the therapy.
FCX-007 has been granted Orphan, Rare Pediatric Disease, and Regenerative Medicine
Advanced Therapy (RMAT) designations by the FDA for the treatment of subjects with RDEB.
The grant funding will be used to execute the Phase 3 clinical trial (FI-EB-002) which may lead
to a potentially effective cell-based gene therapy for RDEB subjects. This grant proposal fulfills
the goal of FDA's Office Orphan Product Development grant program to support the clinical
development of products for use in rare diseases where no current therapy exists.

## Key facts

- **NIH application ID:** 10544379
- **Project number:** 6R01FD007289-02
- **Recipient organization:** CASTLE CREEK BIOSCIENCES, LLC
- **Principal Investigator:** Mary Spellman
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2021
- **Award amount:** $500,000
- **Award type:** 6
- **Project period:** 2021-09-10 → 2027-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10544379

## Citation

> US National Institutes of Health, RePORTER application 10544379, A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (6R01FD007289-02). Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/grant/nih/10544379. Licensed CC0.

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