Speaker: Dr. Jinyi Ma
Title: Preclinical Mouse Models Advancing Gene Therapy and Small Nucleic Acid Therapeutics
Webinar Description:
Small nucleic acid drugs—also known as oligonucleotide therapeutics—are synthetic nucleotide sequences, typically up to 30 bases in length, and can be either single- or double-stranded. These innovative therapeutics precisely regulate gene expression by targeting specific mRNA sequences, offering a highly versatile platform for treating a wide range of diseases. To date, small nucleic acid-based therapies have shown remarkable promise in addressing chronic metabolic disorders, rare genetic conditions, ocular diseases, and neurological disorders, making them a transformative tool in precision medicine.
In this webinar, we will introduce the mechanisms of action and preclinical applications of small nucleic acid therapeutics and gene therapies across multiple disease areas, such as obesity, hyperlipidemia, neurological disorders, and rare diseases. We will also highlight Biocytogen’s portfolio of humanized mouse models and disease models, designed to accelerate the development of these cutting-edge therapies.
Featured case studies will include:
Evaluation of INHBE-targeting siRNA in obesity models
LPA- and APOC3-targeting siRNAs for hyperlipidemia
Anti-TFR1 antibody-oligonucleotide conjugates (AOCs) for central nervous system (CNS) diseases
