The US Food and Drug Administration (FDA) has given approval for Fibrocell Science to start a Phase I/II clinical trial of its orphan gene-therapy product candidate, FCX-007, to treat recessive dystrophic epidermolysis bullosa (RDEB) in adults.
FCX-007 is being co-developed by Fibrocell and synthetic biology firm Intrexon.
RDEB is the most severe form of dystrophic epidermolysis bullosa (DEB), a congenital, progressive, painful and genetic disorder caused by the deficiency of the protein type VII collagen (COL7).
The company expects to begin the open label Phase I/II clinical trial in the second quarter of this year.
The trial's main objective is to evaluate the safety of FCX-007 in RDEB subjects.
In addition, the trial will assess type VII collagen expression and the presence of anchoring fibrils resulting from FCX-007, as well evidence of wound healing.
Six adult subjects are expected to be treated with FCX-007 in Phase I of the trial and six pediatric subjects in Phase II.
Before undertaking studies on pediatric subjects, Fibrocell has to get allowance from FDA and submit evidence of FCX-007 activity in adult subjects, with final data from its ongoing toxicology study.
Fibrocell chairman and chief executive officer David Pernock said: "Achieving this important milestone advances FCX-007 one-step closer to becoming the first therapy to treat the underlying cause of RDEB, offering hope to the patients and families who suffer from this devastating and painful disease."
The company along with Intrexon is also developing gene therapies for diseases affecting the skin, connective tissue and joints using genetically modified autologous fibroblasts.
Intrexon health sector senior vice-president and head Samuel Broder said: "Through FCX-007 gene therapy, we believe there is significant potential to address the unmet needs of patients with RDEB and greatly improve their quality of life.
"The promise of FCX-007 goes beyond palliative care, and we look forward to working with Fibrocell in moving this treatment into the clinic."