Rare Disease Report

BioPontis Alliance and Massachusetts General Hospital Partner for HD

FEBRUARY 02, 2018
Mathew Shanley
Yesterday, nonprofit organization BioPontis Alliance and Massachusetts General Hospital (MGH), the largest hospital-based research program in the United States, announced an agreement to target new treatments for Huntington’s disease (HD).

The strategic collaboration between the two sides intends to advance discovery research being conducted at MGH, Massachusetts Institute of Technology (MIT), and Northwestern University on sirtuin 2 deacetylase inhibitors to pre-clinical testing to develop a potential treatment for neurological disorders like HD.

HD is well-chronicled for the devastating physical, psychological and cognitive effects it can have on a patient. The genetic and neurodegenerative disorder progresses rapidly from dementia-like symptoms into a complete loss of muscle function.

The agreement creates a path for the joint development of basic research toward a treatment candidate. It combines the basic research and early stage translation resources at MGH with with the expertise of rare disease therapeutics development at BioPontis.

“As clinicians and scientists, we are committed to not only unravel the basic mechanisms of diseases like Huntington’s disease, but to see this basic research fuel discovery of new therapeutics,” said Anne Young, MD, PhD, and former Chief of Neurology at MGH in a press release. “This is a unique opportunity for us, to collaborate with BioPontis on one of the major long term aims of our research, namely translating ground-breaking basic findings into treatment opportunities for patients.”

BioPontis Alliance is continuing its work to discover and develop a portfolio of new candidate medicines for rare diseases where there is a clear unmet need.

“It is through collaboration with world-class research institutions, such as Massachusetts General Hospital and the Flemish Institute for Biotechnology (VIB) that we can reach our goal of bringing new and exciting basic research forward to the biopharmaceutical industry for clinical trials, manufacturing and distribution to rare disease patients,” said Richard A. Basile, CEO and Co-Founder of BioPontis Alliance.

Patient organizations working with HD are excited about the prospect of this development project between BioPontis and researchers at MGH.

“The European Huntington Association (EHA) strongly endorses this collaboration,” said Astri Arnesen, President EHA. “We recognize the need to support academic results to reach further towards the clinic and BioPontis’ effort to bridge this gap in rare diseases is highly appreciated. This early stage research combined with the expertise at BioPontis Alliance provides additional hope to our patients and their families that a therapy will be developed for their disease. The strategy of BioPontis to integrate the patients’ perspective as early as possible in the process, we believe, will benefit the projects and hopefully contribute to success.”

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