Rare-disease foundation backs regenerative research – OutSourcing-Pharma.com

CureDuchenne is a global nonprofit honed in on discovering a cure for Duchenne muscular dystrophy (DMD). Its funding arm, CureDuchenne Ventures, is investing in regenerative medicine firm Mesentech, as part of a joint funding collaboration with the Charles H Hood Foundation (which seeks to advance early-stage research for pediatric conditions).

CureDuchenne Ventures, the funding arm of CureDuchenne (a nonprofit aimed at discovering a cure for Duchenne muscular dystrophy) is investing in Mesentech Inc., a regenerative medicine company with a prodrug platform that selectively delivers therapeutics to bone. The project is part of a new joint funding collaboration with the Charles H. Hood Foundation (CHF), which seeks to advance early-stage research for pediatric conditions.

Lianna Orlando, CureDuchennes senior director of research, told Outsourcing-Pharma that bone fractures and quality of life are the top two main outcome measures identified by people with DMD.

Individuals with Duchenne are at a high risk for osteoporosis for multiple reasons that include reduced weight-bearing activity (which normally drives development of strong and dense bones), as well as a side-effect of glucocorticoid therapy, and from the effect of the chronic inflammatory response seen in dystrophin-deficient muscles, she said. Fractures in the vertebral bones of the spine are seen in up to 30% of boys with Duchenne, and in addition to being very painful may lead to spine deformity; fractures in the long bones in the arm or the leg are common, and in many cases lead to permanent loss of ambulation.

Whats more, she said, bone fractures can lead to additional complications.

Long bone fractures can release fat emboli into the bloodstream, which can obstruct blood flow in critical places, including the lungsmost often the lungs. Although rare, fat embolism syndrome is a particularly serious complication of bone fractures in Duchenne; in the most severe cases, fat embolisms can lead to death, Orlando told OSP.

The CureDuchenne investment supports Mesentechs lead program, MES-1007, into clinical development and its evaluation in DMD. There are currently no approved therapies for bone wastage for individuals affected by DMD.

Debra Miller, founder and CEO of CureDuchenne, said the investment and collaboration hold the potential to increase the groups impact.

Addressing bone morbidities that contribute to the loss of ambulation could significantly add quality of life to everyone affected by Duchenne, said Miller.

In addition to providing Mesentech with funding, CureDuchenne intends to play an active role in advancing the prodrug technology platform for DMD. Additionally, CureDuchenne chief scientific officer Michael Kelly will join Mesentechs scientific advisory board.

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