HGT Pipeline Enhanced with Protein Replacement Therapy Being Investigated for the Treatment of Dystrophic Epidermolysis Bullosa
Shire plc (LSE: SHP, NASDAQ: SHPG), announces that it has signed an agreement to acquire Lotus Tissue Repair, Inc. of Cambridge, MA, a privately held biotechnology company developing the first and only protein replacement therapy currently being investigated for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is a devastating orphan disease for which there is no currently approved treatment option other than palliative care. Subject to customary government approvals, Shire will purchase the company for an upfront payment and certain contingent payments based on the achievement of certain safety and development milestones.
Epidermolysis Bullosa (EB) is a set of rare, genetic diseases characterized by the presence of extremely fragile skin and recurrent blister formation resulting from minor mechanical friction or trauma. DEB is one of the more severe of the genetic disorders that comprise EB. Severe cases of DEB may also include internal blistering of the mouth, esophagus, lower GI tract, upper airway and GU tract.
Shire’s Human Genetic Therapies business will undertake the further development of Lotus Tissue Repair’s lead product candidate, a proprietary recombinant form of human collagen Type VII (rC7), an intravenous protein replacement therapy for the treatment of DEB. The product is in late pre-clinical development and has the potential to be a first-in-class systemic therapy for the treatment of DEB. This acquisition expands Shire’s commitment to finding treatments for EB, which also includes ABH001, Shire’s Regenerative Medicine product currently being investigated as a dermal substitute therapy for the treatment of non-healing wounds in patients with EB.
“DEB is one the most devastating orphan diseases, severely impacting the lives of patients and their families, many of whom have few or no treatment options other than palliative care,” said Dr. Philip J. Vickers, Global Head of Research and Development, Shire Human Genetic Therapies. “rC7 protein replacement therapy has the potential to provide a first-in-class disease-modifying treatment for these children. We plan to apply our proven ability to develop protein replacement therapies for rare genetic diseases to progress rC7as a possible groundbreaking treatment that offers hope to patients with DEB.”
"This acquisition of Lotus Tissue Repair by Shire further underscores the potential of this proprietary rC7 technology to dramatically change the treatment landscape for DEB patients and their families," said Dr. Mark de Souza, founding Chief Executive Officer of Lotus Tissue Repair. "We are thrilled that this program will become part of the innovative pipeline at Shire and the company's growing commitment to this patient community."
Lotus Tissue Repair is a private company launched in 2011 by a proven team of biotechnology entrepreneurs, world-leading experts in rC7 protein replacement therapy for DEB and top-tier life sciences investor, Third Rock Ventures.
About Type VII Collagen
Type VII collagen (C7) is a collagen found in the skin of humans and other animals. It is localized in the basement membrane zone (BMZ) between the epidermis and dermis in large structures called Anchoring Fibrils (AFs), which are composed of C7. The AFs hold the epidermis and dermis together. Purified human C7 can be extracted in very small quantities from human skin, human amnion, and skin cells such as keratinocytes or dermal fibroblasts. However, large amounts of C7 cannot be generated from these sources in this manner.
About Recombinant Collagen VII (rC7)
Dr. Mei Chen and Dr. David Woodley, co-founders of Lotus Tissue Repair Inc., are the co-inventors of recombinant collagen type VII (rC7) technology and leading experts on its use as protein replacement therapy. Lotus is developing its rC7 technology as the first and only protein replacement therapy for dystrophic epidermolysis bullosa (DEB). This approach directly addresses a primary driver of the condition: deficiency or dysfunction of C7. Pre-clinical findings to date are promising, showing in multiple pre-clinical models that rC7 as a protein replacement therapy is potent, long-lasting, and is specifically retained in the skin and other affected tissues after intravenous injection.
ABH001 is an engineered, human fibroblast-derived dermal substitute generated by culturing human neonatal dermal fibroblasts onto a bioresorbable polyglactin mesh scaffold. The PGLLA mesh which serves as the scaffolding onto which fibroblasts are grown, they secrete dermal collagen, other extracellular matrix proteins, growth factors, and cytokines, creating a three-dimensional human tissue containing metabolically active living cells. The final product consists of a well-developed dermal matrix and evenly dispersed neonatal dermal fibroblasts.
Epidermolysis bullosa (EB) is a set of rare, genetic diseases characterized by the presence of extremely fragile skin and recurrent blister formation resulting from minor mechanical friction or trauma. Dystrophic epidermolysis bullosa (DEB) is one of the more severe forms of the genetic disorders that comprise EB and is caused by a deficiency or dysfunctionality of collagen type VII. The recessive form of DEB is extremely painful and patients suffer from severe skin blistering, extremely fragile skin and mucosa of the mouth, esophagus, and anus, mutilating scarring of the hands and feet, joint contractures, and strictures of the esophagus. In the second or third decade of life, these patients develop aggressive squamous cell carcinomas in chronically wounded areas that often lead to metastasis and death. There are no adequate treatments available for this painful, debilitating and costly disease.
For further information please contact:
+1 781 482 0999
+44 1256 894157
Jessica Mann (Corporate)
+44 1256 894 280
Jessica Cotrone (Human Genetic Therapies)
+ 1 781 482 9538
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