Commercial Partners

Wave Life Sciences

Chandra Vargeese PhD (Chief Technology Officer), Chris Francis PhD (Senior Vice President, Head of Emerging Areas), Paul Bolno MD (President and CEO) 

Wave Life Sciences is a biotechnology company focussed on delivering transformational therapies for patients with serious, genetically defined diseases.  They have played a leading role in advancing nucleic acid therapeutics to enable rational drug design and development, in particular through their development of stereopure oligonucleotides, which demonstrate important improvements in efficacy, safety and delivery. 

Through the precise control of stereochemistry, Wave has been able to design molecules capable of distinguishing between mRNA targets which differ by a single nucleic polymorphism, enabling allele specific targeting in the case of Huntington’s disease. Wave will bring synthesis capability that has been scaled to include GMP manufacturing and experience supporting clinical development programs (e.g. acquired through partnerships with major pharmaceutical companies), including global clinical trial operations (their head of clinical operations is based in the UK), and regulatory affairs personnel. 

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Robert McDowell, PhD (CSO, former SVP Drug Discovery), Tassos Gianakakos (President, CEO, Director) 

MyoKardia (now part of Bristol Myers Squibb) is a leader in the discovery and development of small molecule drugs that target the sarcomere to address cardiomyopathies. Through the development of precision medicine platforms, the company defines and engineers small molecules to selectively target distinct biophysical properties of the sarcomere that are disrupted by human mutations. The company has established diverse pre-clinical models (stem cell-derived cardiomyocytes to small and large mammals) with human cardiomyopathy mutations. MyoKardia founded and supports SHaRE . The Cureheart Project will harness MyoKardia’s precision assays, drug discovery and design platforms, pre-clinical models, GMP processing, and regulatory expertise and financial support to enable fundamental and first-in-man gene-therapy studies.

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