Novel Approaches for Treating Genetic Diseases

Join us next Wednesday, Feb. 8, for a two-hour discussion about some of the latest therapeutic modalities during Novel Approaches for Treating Genetic Diseases.

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After an introduction from moderator Andrew Coles, PhD, this Insight Series event will kick off with a talk from Sarah Glass, PhD, the chief operating officer at n-Lorem Foundation. Dr. Glass will talk about the foundation’s work to provide experimental antisense oligonucleotide (ASO) medicines to patients who have nano-rare diseases, which affect 30 or fewer people worldwide.

Next, Board Member and UMass Chan Medical School Professor Anastasia Khvorova, PhD, will discuss new developments in the treatment of Huntington’s disease. Dr. Khvorova references a 2019 Nature Biotechnology paper she coauthored about a divalent siRNA chemical scaffold that may induce silencing in huntingtin, the causative gene of the disease.

The final speaker will be ASGCT Treasurer Paloma Giangrande, PhD, who will talk about RNA editing as a therapeutic modality, particularly a platform developed by Wave Life Sciences, where she is vice president of platform biology.

Remember to bring your questions for the panel discussion! We look forward to seeing you Feb. 8 for this exciting event.

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