Poster Hall Hours

Tuesday, May 10, 2016
3:00 pm - 6:30 pm

Wednesday, May 11, 2016
10:00 am - 6:30 pm

Thursday, May 12, 2016
10:30 am - 2:00 pm

30 Min Webinar + Q&A | 11:00AM EST | October 20th

Michael V. Templin, PhD DABT
Director, Scientific Advisory Services
Charles River

Nucleic Acid–based Therapies: Challenges and Opportunities during Nonclinical Development

Nucleic Acid-based therapies, also described as “oligonucleotides”, represent a distinct drug class. Antisense oligonucleotides (ASO), short interfering RNAs (siRNA), and microRNAs (agonists and mimics) are the most familiar “oligos”. These compounds are a chemically derived entity, and as such, encompass traditional approaches to safety assessment. However, oligos also resemble biologics with the potential for exaggerated pharmacology. The combination of a synthetic origin and concerns for biologic-like actions has resulted in the nonclinical development pathway incorporating small (i.e., two species) and large (i.e., pharmacology driven) molecule paradigms; and this has been successfully followed with several programs in clinical development or receiving regulatory approval. This webinar will address the challenges and opportunities for safety assessment of oligonucleotides based on established pathways, however, key takeaways and points for discussions will also include: 

1)Specialized routes of administration, e.g., Intrathecal administration, and how this can influence studies in rodents and non-rodents. 

2)Requirements for bioanalytical assays to include an expanded breadth and depth of analysis and involve more extensive molecular biology approaches and techniques. 

3)Gene modulation has moved the oligo field closer to “gene therapy”; however, the impact this will have on safety assessment has not been fully determined. 

Abstract

Speaker