New Therapeutic Modalities for Rare Diseases

Is there potential for a new protein or messenger RNA that will fit all mutations and generate a global solution for a disease? Case studies on this and other novel approaches for treating rare diseases

​Eran Blaugrund

VP R&D, ART BioScience Ltd
Rehovot, Israel

​Modified mRNA Therapeutics for Rare Diseases 

The field of rare diseases is turning out to be a lucrative area if breakthrough medications can be provided to patients. In addition, regulatory agencies are trying to support and facilitate the development process for these disorders. This has brought about a large number of novel strategies for Duchenne muscular dystrophy (DMD) drug development but it remains to be seen whether these innovations can really make a difference in patients` lives’. Eran Blaugrund will provide a critical view on some of these novel methodologies and focus on one of them, injection of dystrophin mRNA.

​Stuart Hughes
Director and Head of Pharmacology
at Vertex Pharmaceuticals
London, UK

Leveraging Known Causal Human Biology for Discovering New Medicines for Orphan Diseases

In many cases the underlying cause of orphan and rare diseases is known, often being genetic nature. However, that the trajectory of disease causing events and a validated therapeutic intervention point are also known does not always follow. I will discuss the key requirements and approaches for addressing diseases where the essential causative events are known as well outline how this should differ for diseases where this is not the case.

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