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Addressing chronic inflammatory and degenerative diseases
February 2016
by Lloyd Dunlap  |  Email the author
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EXTON, Pa. & GERMANTOWN, Md.—Fibrocell Science Inc., an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs, and Intrexon Corp., a leader in synthetic biology, have announced an exclusive channel collaboration for the development of genetically modified fibroblasts to treat chronic inflammatory and degenerative diseases of the joint, including arthritis and related conditions.
 
According to the U.S. Centers for Disease Control and Prevention, arthritis—characterized by joint inflammation, pain and decreased range of motion—is the United States’ most common cause of disability, affecting more than 52 million adults as well as 300,000 children at a cost exceeding $120 billion.
 
“Fibrocell’s approach to personalized biologics is distinctive based on our proprietary autologous fibroblast technology: targeting the underlying cause of disease by extracting cells from a patient’s own skin to create localized therapies—with or without genetic modification—that are compatible with the unique biology of each patient,” a company spokesperson states. “Fibroblasts uniquely target the localized environment of skin and connective tissue, which are typically difficult to treat with systemic drug therapies, due to limited blood flow to these areas. The localized administration is designed to provide long-lasting therapeutic effect.”
 
“In collaboration with Intrexon Corporation, a leader in synthetic biology, Fibrocell is developing gene therapies using gene-modified autologous fibroblasts to express targeted proteins. Intrexon’s technology is designed to facilitate the assembly and delivery of the necessary target gene constructs to autologous fibroblasts. Access to this platform allows Fibrocell a rapid method to screen and construct the best genetic therapeutic solutions.”
 
“The synergy of our versatile fibroblast platform with Intrexon’s genetic engineering is readily apparent through two product candidates, FCX-007 and FCX-013, which have generated encouraging preclinical data across difficult-to-treat indications,” said David Pernock, chairman and CEO of Fibrocell. “We are excited by the expansion of our relationship with Intrexon and believe the power of our combined platforms will allow us to vie for leadership in this major therapeutic category and bring hope to patients and their families.”
 
Through the collaboration, Fibrocell’s proprietary fibroblast platform will be combined with Intrexon’s cellular engineering capabilities to generate cell-based therapeutics that have been modified to express one or more proteins at sites of joint inflammation, helping overcome the limitations of existing treatment approaches for chronic inflammatory and degenerative diseases of the joint.
 
“With present standards of care focused on limiting symptoms and slowing progression, optimal therapy for inflammatory, degenerative diseases of the joints represents a major unmet medical need and a significant commercial opportunity,” stated Dr. Samuel Broder, senior vice president and head of Intrexon’s Health Sector. “With Intrexon’s Better DNA suite of proprietary technologies, our innovative engineered cell-based therapies may provide long-lasting effect while minimizing the repeated injections necessary with current treatments.”
 
Overall, autologous fibroblasts offer distinct advantages as the technology platform for creating personalized cell and gene therapies:
  • Localized administration, which avoids side effects typically associated with systemic therapy and targets sites of tissue injury or inflammation
  • Reduced rejection concerns—autologous fibroblasts are compatible with the unique biology of each patient
  • Focus on monogenic or single-gene disorders with a known mechanism of action, so that the missing or deficient target gene is known and a functional copy can be provided or the protein of interest can be up-regulated to potentially correct the cause of the disease.
Under the terms of the agreement, Intrexon will receive a technology access fee of $10 million in cash and reimbursement for all research and development costs. The agreement also provides for regulatory and commercial milestone payments to Intrexon for each collaboration product of up to $30 million and $22.5 million, respectively, as well as a low double-digit royalty based on the net sales from collaboration products.
 
Fibrocell’s most advanced product candidate, azficel-T, uses its proprietary autologous fibroblast technology and is in a Phase 2 clinical trial for the treatment of chronic dysphonia resulting from vocal cord scarring. In collaboration with Intrexon, Fibrocell is developing gene therapies for skin and joint diseases using gene-modified autologous fibroblasts as well. Fibrocell is also engaged in preclinical development of FCX-007, its orphan gene-therapy product candidate, for the treatment of recessive dystrophic epidermolysis bullosa, and in preclinical development of FCX-013, its gene-therapy product candidate, for the treatment of linear scleroderma. In addition, Fibrocell and Intrexon will be commencing preclinical development of a gene-therapy for the treatment of chronic inflammatory and degenerative diseases of the joint, including arthritis and related conditions.
 
Intrexon’s integrated technology suite provides its partners across diverse markets with industrial-scale design and development of complex biological systems said to deliver unprecedented control, quality, function and performance of living cells.
 
Code: E021611

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