# Ph1/2 Study of FCX-007 for Treatment of RDEB IND 16582 Protocolv4.1 (11/23/2016)

> **NIH FDA R01** · FIBROCELL TECHNOLOGIES, INC. · 2020 · $350,000

## Abstract

Project Description (Project Summary/Abstract)
Epidermolysis bullosa (EB) is a group of inherited genetic blistering skin disorders. A severe EB
subtype caused by mutations within the type VII collagen gene (COL7A1), recessive dystrophic
epidermolysis bullosa (RDEB), is an autosomal recessive, inherited skin disease. People with this
disease have defective or lack normal type VII collagen, which facilitates adhesion of the
epidermis, or outer layer of the skin, to the inner dermal layers of the skin. The disease is
characterized by painful blisters and wounds on skin and mucous membranes. The sequelae of
blisters and 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). RDEB is acknowledged as an orphan and pediatric rare disease by the U.S.
Food and Drug Administration (FDA). Current therapy for RDEB is limited to palliative wound care
as there are currently no curative treatments and no approved drugs for RDEB. Fibrocell
Technologies, Inc. (Fibrocell) is developing FCX-007, a gene-modified ex-vivo autologous
fibroblast therapy that will deliver type VII collagen to the skin of RDEB subjects. Fibrocell has an
open Investigational New Drug Application (IND 016582) for FCX-007.
In this grant application, Fibrocell proposes to continue its interventional, open-label Phase 1/2
study to evaluate the safety, efficacy and duration of effect of FCX-007. The target indication for
FCX-007 is the treatment of skin-blistering lesions in patients with RDEB confirmed by genetic
testing. Reducing wound size and facilitating wound closure will be clinically meaningful by
preventing or decreasing the rate of infection, pain, scarring, deformity or squamous cell
carcinoma. Fibrocell expects FCX-007 to be clinically safe given the autologous nature of the
therapy, and based on preclinical long-term toxicity and tumorigenicity data in animals. Clinical
safety will be assessed by testing for presence of replication-competent lentivirus (RCL), type VII
collagen autoantibody analysis for immune reactions to type VII collagen as well as physical
examinations. Efficacy and durability of FCX-007 will be assessed by presence/increase in type
VII collagen protein correctly localized to the basement membrane zone and incorporated into
ultra-structurally normal anchoring fibrils. FCX-007 has been granted orphan designation,
pediatric rare disease designation and fast track designation by the FDA for the treatment of
subjects with RDEB. This grant will be used to assist in completing the Phase 1/2 clinical trial
which may lead to a potentially effective cell-based gene therapy for RDEB subjects. Fibrocell
expects to work closely with the FDA in designing the Phase 3 clinical trial while data from the
current Phase 1/2 trial is being gathered. This grant proposal fulfills the goal of FDA’s Orphan
Product Division grant program to su...

## Key facts

- **NIH application ID:** 10001343
- **Project number:** 5R01FD006113-03
- **Recipient organization:** FIBROCELL TECHNOLOGIES, INC.
- **Principal Investigator:** John Michael Maslowski
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $350,000
- **Award type:** 5
- **Project period:** 2018-09-20 → 2021-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10001343

## Citation

> US National Institutes of Health, RePORTER application 10001343, Ph1/2 Study of FCX-007 for Treatment of RDEB IND 16582 Protocolv4.1 (11/23/2016) (5R01FD006113-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10001343. Licensed CC0.

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