Please find the speaker details below:

1. Professor Nick Lench, Executive Director, Nucleic Acids Therapy Accelerator
   “Precision Genetic Therapies for Rare Diseases”
   This presentation provides an introduction to antisense oligonucleotide RNA therapies and their use in the treatment of rare genetic diseases. ASOs can be used to modulate gene expression through silencing, knockdown, exon skipping and splice switching. The success of nusinersen (Spinraza®) for the treatment of spinal muscular atrophy (SMA) demonstrates how an ASO can restore functional protein and significantly improve clinical outcomes. Accelerated regulatory approval pathways are now available for N of 1 personalised therapies, particularly for early-onset, regressive, neurological disorders.
2. Dr Peter Oliver, Head of Research, Nucleic Acids Therapy Accelerator
   “Preclinical development of nucleic acid therapeutics for rare and ultra-rare CNS disorders”
   NATA has established a preclinical development pipeline for antisense oligonucleotide (ASO) and small interfering RNA (siRNA) therapies, including design, synthesis, conjugation chemistry and biological testing in vitro and in vivo. NATA has a particular interest in rare neurological and neurodevelopmental disorders, where some of the first ASOs have been approved for clinical use. This talk will provide exemplars of the genetic targets being developed by NATA, including aspects of the design and screening process and the 3-D cell models being applied for functional studies.