Galenea and CHDI Foundation announce collaboration to identify key synaptic dysfunction in Huntington’s disease
Cambridge, MA and New York, NY, Galenea Corp. and CHDI Foundation, Inc. have announced a collaboration to identify synaptic dysfunction linked to >Huntington’s disease (HD) utilizing Galenea’s proprietary synaptic transmission drug discovery platform. Mounting evidence indicates that mutant huntingtin protein, the molecular defect in HD, disrupts normal synaptic function, contributing to the behavioral, cognitive, and motor symptoms of this devastating neurodegenerative disorder. Galenea’s innovative platform will provide new insights into the disease mechanism and, ultimately, offer a different approach to the discovery of therapies for HD.
“The application of Galenea’s high throughput platform to investigate synaptic transmission dysfunction in HD will be very informative to developing drugs that target, in a very precise way, the molecular underpinnings of cognitive and behavioral defects in HD”, said Ramee Lee, PhD, Director, Early Discovery Initiative at CHDI. “We are excited to work with Galenea to determine whether EEG signatures in rodent HD models are reflective of the synaptic dysfunction seen in HD patients. Such information could significantly facilitate HD preclinical drug discovery efforts,” added George Yohrling, PhD, Director, Target Assessment at CHDI.
Dysfunctions in synaptic transmission, the fundamental process by which neurons communicate, play a critical role in many central nervous system diseases, including HD. Galenea’s platform examines synaptic transmission on multiple levels. Galenea’s MANTRA ™ (Multiwell Automated Neuro TRansmission Assay) system, a revolutionary high throughput screening assay, monitors synaptic events at the cellular level using primary neuronal cultures. At the network level, Galenea has developed a state-of-the-art system for establishing in vivo electroencephalography (EEG) measures of behaviors in rodent disease models by monitoring brain and behavioral activities in parallel. The collaboration will employ this two-pronged approach to identify and characterize the synaptic defects that occur in HD at both the neuronal and network levels.
“We are pleased to be collaborating with CHDI who bring deep expertise and resources to the collaboration. Our vision is to translate initial discoveries that emerge from this critical first step into novel drug candidates that positively impact the lives of Huntington’s disease patients,” said Mark Benjamin, DSc, Galenea’s President and CEO. David Gerber, PhD, VP of CNS Research added, “The opportunity to apply Galenea’s platform to the discovery of synaptic-based therapeutics for HD is ideal. I am confident that this collaboration will yield new insights into the underlying pathology of this disease and ultimately new therapeutic candidates.”
About Huntington’s disease
Huntington’s disease is an inherited neurodegenerative disorder caused by a mutation in the huntingtin gene. The defect causes a DNA sequence called a CAG repeat to occur many more times than normal. Each child of a parent with a mutation in the huntingtin gene has a 50% chance of inheriting the mutation. As a result of carrying the mutation, an individual’s brain cells degenerate leading to behavioral, cognitive, and motor impairments that, over the course of the disease, significantly reduce the individual’s quality of life and ultimately cause death within 15 to 25 years of overt symptom onset. There are currently no therapeutics approved that slow the progression of Huntington’s disease. It is estimated that the disorder affects about 30,000 people in the United States and at least 150,000 others have a 50% risk of developing Huntington’s disease at some point.
About CHDI Foundation, Inc.
CHDI Foundation, Inc. is a privately-funded, not-for-profit, biomedical research organization exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington’s disease. As a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as quickly as possible. Our scientists work closely with a network of more than 600 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing strategic scientific direction to ensure that our common goals remain in focus. More information about CHDI can be found at www.chdifoundation.org
Galenea is a leader in the rapidly emerging field of synaptic transmission (ST), the process by which neurons communicate with each other. Dysfunctions in ST are now widely believed to play a central role in many psychiatric, neurological and neurodegenerative diseases, and modulators of ST therefore have the potential to yield breakthrough treatments. Galenea has developed an innovative ST drug discovery platform that integrates three components: MANTRA™, a high throughput, proprietary screening technology to identify a new generation of small molecule modulators of synaptic transmission; in vivo models using integrated EEG measures of animal behavior to more reliably determine CNS drug efficacy; and human EEG biomarkers, developed in tandem with and informed by our animal EEG data to greatly enhance CNS drug development. The company is advancing a novel pro-cognitive program derived from the platform and the approach can be extended to address multiple CNS disorders. Based in Cambridge, MA, Galenea has assembled a compelling scientific team, balancing academic aptitude with industry experience and entrepreneurship. For more information about Galenea, please visit the company’s website atwww.galenea.com
|Galenea Corp.||CHDI Foundation, Inc.|
|Lisa Paborsky, PhD||Simon Noble, PhD|