Shire acquires US rare genetic disorder biotech

Hannah Blake

pharmaphorum

Shire is to acquire the privately held biotechnology company, Lotus Tissue Repair, Inc. of Massachusetts, US. Lotus Tissue Repair is developing the first and only protein replacement therapy, which is currently being investigated for the treatment of dystrophic epidermolysis bullosa (DEB).

DEB is an orphan disease for which there is no currently approved treatment option other than palliative care. 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.

“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. 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.”

Dr. Philip J. Vickers, Global Head of Research and Development, Shire Human Genetic Therapies.

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

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.

&nbsp,

pharmaphorum-twitter

&nbsp,

Related news:

Shire adds to genetic disorder drugs with U.S. deal (Reuters)

Reference links:

Shire press release

&nbsp,

Don't miss your daily pharmaphorum news.
SUBSCRIBE free here.