Experimental Protein Replacement Therapy for Congenital Muscular Dystrophy Type 1A (MDC1A)
MILFORD, MA and RENO, NV, USA I February 13, 2014 I Prothelia Incorporated (Prothelia) and University of Nevada, Reno announce that they have entered into strategic agreements with Alexion for the development of Laminin-111, a patented experimental protein replacement therapy for merosin-deficient congenital muscular dystrophy (MDC1A), a life-threatening, ultra-rare disease caused by a genetic deficiency of the Laminin-211 protein.
The three parties have entered into a binding agreement wherein Alexion has an exclusive option to acquire privately-held Prothelia and license Laminin-111 directly from the University of Nevada, Reno upon the achievement of specified research and development milestones. In addition, the University of Nevada, Reno and Alexion have entered into a sponsored research agreement to accelerate further research on the investigational therapy conditioned on the outcome of certain development research to be performed by Alexion.
“This partnership with Alexion represents a significant step toward further investigating Laminin-111 as a potential treatment for MDC1A,” said Richard Cloud, CEO at Prothelia. “We are confident that Alexion is the right partner, given their commitment to patients suffering from devastating, ultra-rare diseases like MDC1A.”
Laminins are a group of high molecular weight glycoproteins that help with the formation of the basal lamina (or basement membrane), which is a type of extracellular matrix.1 MDC1A is a life-threatening, ultra-rare disease caused by a genetic deficiency of the laminin-211 protein. Laminin-211 provides the necessary structural integrity to muscles, and patients with MDC1A present with respiratory insufficiency, poor muscle tone, muscle atrophy, delayed or absent motor milestones, feeding difficulties, scoliosis and joint contractures. Respiratory infection is a common cause of morbidity and early death in MDC1A patients.2 Currently, there are no approved therapies for MDC1A. Laminin-111 is a form of laminin found in embryonic skeletal muscle and has been shown in early animal studies to substitute for the loss of laminin-211.3
“MDC1A is a disease that aligns very well with our passion to develop and deliver first-in-class drug candidates that have the potential to transform the lives of people suffering with devastating disorders that currently have no treatment options,” said Martin Mackay, Ph.D., Executive Vice President, Head of Global Research & Development at Alexion. “Laminin protein replacement is a promising experimental therapy and addition to our research initiatives as we seek to further expand our product portfolio. We look forward to a productive collaboration with Prothelia and the University of Nevada.”
The patented Laminin-111 therapy is based on work by University of Nevada School of Medicine Professor, Dr. Dean Burkin, and was licensed to Prothelia in 2008.
“Dr. Burkin has devoted much of his research to the treatment of muscular dystrophy, and this partnership and continued investigational research builds on his work,” said Mridul Gautam, the University’s vice president of research and innovation. “This partnership exemplifies the powerful nexus of university-based discovery and industry collaboration which creates the possibility of important advancements being brought to the marketplace and ultimately bettering lives.”
Life Sciences Advisory, LLC and Foley Hoag LLP served as advisors to Prothelia.
About Prothelia
Prothelia Incorporated is a privately held biotechnology company focusing on protein replacement therapies for the muscular dystrophies and has received support from the National Institutes of Health and advocacy groups, including Cure CMD, Struggle against Muscular Dystrophy, Parent Project Muscular Dystrophy and Hope for Gus. Dr. Bradley Hodges founded Prothelia in 2007 and serves as Chief Scientific Officer. For more information, visit www.prothelia.com.
About University of Nevada, Reno
Founded in 1874 as Nevada’s land-grant university, the University of Nevada, Reno ranks in the top tier of “best national universities.” With nearly 19,000 students, the University is driven to contribute a culture of student success, world-improving research and outreach that enhances communities and business. Part of the Nevada System of Higher Education, the University has the system’s largest research program and is home to the state’s medical school, the University of Nevada School of Medicine. For more information, visit www.unr.edu.
References:
- Durbeej, M. (2010). Laminins. Cell and Tissue Research, 339(1): 259-268.
- Sparks SE, Escolar DM. “Congenital muscular dystrophies.” Handb Clin Neurol. 2011;101:47-79.
- Rooney JE., et al. “Laminin-111 protein therapy reduces muscle pathology and improves viability of a mouse model of merosin-deficient congenital muscular dystrophy.” Am J Pathol 2012, 180:1593–1602.
SOURCE: Prothelia
Post Views: 332
Experimental Protein Replacement Therapy for Congenital Muscular Dystrophy Type 1A (MDC1A)
MILFORD, MA and RENO, NV, USA I February 13, 2014 I Prothelia Incorporated (Prothelia) and University of Nevada, Reno announce that they have entered into strategic agreements with Alexion for the development of Laminin-111, a patented experimental protein replacement therapy for merosin-deficient congenital muscular dystrophy (MDC1A), a life-threatening, ultra-rare disease caused by a genetic deficiency of the Laminin-211 protein.
The three parties have entered into a binding agreement wherein Alexion has an exclusive option to acquire privately-held Prothelia and license Laminin-111 directly from the University of Nevada, Reno upon the achievement of specified research and development milestones. In addition, the University of Nevada, Reno and Alexion have entered into a sponsored research agreement to accelerate further research on the investigational therapy conditioned on the outcome of certain development research to be performed by Alexion.
“This partnership with Alexion represents a significant step toward further investigating Laminin-111 as a potential treatment for MDC1A,” said Richard Cloud, CEO at Prothelia. “We are confident that Alexion is the right partner, given their commitment to patients suffering from devastating, ultra-rare diseases like MDC1A.”
Laminins are a group of high molecular weight glycoproteins that help with the formation of the basal lamina (or basement membrane), which is a type of extracellular matrix.1 MDC1A is a life-threatening, ultra-rare disease caused by a genetic deficiency of the laminin-211 protein. Laminin-211 provides the necessary structural integrity to muscles, and patients with MDC1A present with respiratory insufficiency, poor muscle tone, muscle atrophy, delayed or absent motor milestones, feeding difficulties, scoliosis and joint contractures. Respiratory infection is a common cause of morbidity and early death in MDC1A patients.2 Currently, there are no approved therapies for MDC1A. Laminin-111 is a form of laminin found in embryonic skeletal muscle and has been shown in early animal studies to substitute for the loss of laminin-211.3
“MDC1A is a disease that aligns very well with our passion to develop and deliver first-in-class drug candidates that have the potential to transform the lives of people suffering with devastating disorders that currently have no treatment options,” said Martin Mackay, Ph.D., Executive Vice President, Head of Global Research & Development at Alexion. “Laminin protein replacement is a promising experimental therapy and addition to our research initiatives as we seek to further expand our product portfolio. We look forward to a productive collaboration with Prothelia and the University of Nevada.”
The patented Laminin-111 therapy is based on work by University of Nevada School of Medicine Professor, Dr. Dean Burkin, and was licensed to Prothelia in 2008.
“Dr. Burkin has devoted much of his research to the treatment of muscular dystrophy, and this partnership and continued investigational research builds on his work,” said Mridul Gautam, the University’s vice president of research and innovation. “This partnership exemplifies the powerful nexus of university-based discovery and industry collaboration which creates the possibility of important advancements being brought to the marketplace and ultimately bettering lives.”
Life Sciences Advisory, LLC and Foley Hoag LLP served as advisors to Prothelia.
About Prothelia
Prothelia Incorporated is a privately held biotechnology company focusing on protein replacement therapies for the muscular dystrophies and has received support from the National Institutes of Health and advocacy groups, including Cure CMD, Struggle against Muscular Dystrophy, Parent Project Muscular Dystrophy and Hope for Gus. Dr. Bradley Hodges founded Prothelia in 2007 and serves as Chief Scientific Officer. For more information, visit www.prothelia.com.
About University of Nevada, Reno
Founded in 1874 as Nevada’s land-grant university, the University of Nevada, Reno ranks in the top tier of “best national universities.” With nearly 19,000 students, the University is driven to contribute a culture of student success, world-improving research and outreach that enhances communities and business. Part of the Nevada System of Higher Education, the University has the system’s largest research program and is home to the state’s medical school, the University of Nevada School of Medicine. For more information, visit www.unr.edu.
References:
- Durbeej, M. (2010). Laminins. Cell and Tissue Research, 339(1): 259-268.
- Sparks SE, Escolar DM. “Congenital muscular dystrophies.” Handb Clin Neurol. 2011;101:47-79.
- Rooney JE., et al. “Laminin-111 protein therapy reduces muscle pathology and improves viability of a mouse model of merosin-deficient congenital muscular dystrophy.” Am J Pathol 2012, 180:1593–1602.
SOURCE: Prothelia
Post Views: 332
