GenomeWebinars

This webinar will discuss recent research on antisense oligonucleotide (ASO) therapies as an option for individualized care for rare pediatric brain disorders.

Antisense technology can be administered into the cerebral spinal fluid to modify gene expression – a promising option for so-called nano-rare mutations, which appear in only a handful of patients worldwide.

In this webinar, Joseph Gleeson of the University of California, San Diego, will discuss his team’s work modeling pediatric brain disease using patient-derived induced pluripotent stem-cell (hIPSC) neural models to assess the effectiveness of ASO candidate drugs on disease-relevant differential gene expression.

Gleeson and his colleagues have optimized protocols for hIPSC differentiation, ASO screening, and genetic assessment, which they hope will support future FDA Investigational New Drug applications for patients with rare disease.

In this webinar, Gleeson will discuss the following:

• Mutations leading to toxic gain-of-function or 'splice-gain' are amenable to ASO therapy.
• Antisense oligonucleotides are a drug class that can interact with the transcriptome to modulate gene expression.
• High-throughput methods to derive neural cells from hIPSCs can be achieved with direct differentiation protocols.

The COVID-19 pandemic underscored the importance of molecular diagnostics and compelled laboratories to quickly validate new methods and increase testing capacity through the expansion of resources and high-throughput automation. As SARS-CoV-2 infections reach an equilibrium, and the virus moves from a pandemic to an endemic disease, testing demand is decreasing, revealing the opportunity to address broader public health needs through the expansion of infectious disease diagnostics.

Aegis BioPharma Laboratory launched COVID-19 testing in April 2020 with a daily capacity of 3,000 tests and quickly scaled during that first year to over 100,000 tests per day. The lab has performed over 14.2 million COVID-19 PCR tests to date, servicing a broad network across the United States, Puerto Rico, and the US Virgin Islands. Aegis leveraged the partnerships, technologies, and infrastructure developed during the COVID-19 surge to build the infectious disease testing menu aimed at limiting the spread of health risks within the community.

Attendees of this webinar will learn how Aegis is:

• Utilizing extra resources and capacity to create opportunities for business development and laboratory growth, post-pandemic.
• Standardizing laboratory processes and procedures to efficiently add new workflows.
• Creating flexible and customizable infectious disease panels that provide actionable results to meet the ever-changing public health needs.

The evolutionarily conserved EZH2 enzyme is upregulated in most solid cancers. It mediates repressive histone H3-Lys-27-tri-methylation (H3K27me3), which leads to tumor suppressor gene silencing. EZH2-dependent transcriptional signatures predict prognosis in cancer patients.

EZH2 silencing blocks metastasis and causes selective cancer cell death. Several EZH2 inhibitors (EZH2i) are currently in clinical trials. However, while EZH2i monotherapy isn’t effective in most solid malignancies, synthetically lethal approaches — such as EZH2i tazemetostat in combination with doxorubicin and immunotherapy in advanced epithelioid sarcomas — have generated promising results.

In this session, Francesco Crea and Francesca Salani will provide an overview of EZH2 inhibitors in cancer therapy and the development of a non-invasive diagnostic tool to monitor target engagement and downregulation in patients exposed to EZH2 inhibitors.

Terry Kelly, chief innovation officer at Volition, will also outline how Volition works with organizations such as the Open University through its Nu.Q Discover program, which gives scientists and drug developers access to a range of state-of-the-art assays for rapid epigenetic profiling in disease, model development, preclinical testing, and clinical studies.

3D genomics is a powerful NGS approach that provides unparalleled access to the sequence, structure, and regulatory landscape of genomes. In this webinar, Anthony Schmitt, senior vice president of science at Arima Genomics, will share how 3D genomics allows researchers to study noncoding regions of the genome, explore structural variants driving disease, and identify new therapeutic targets.

In this webinar, you will:

1. Hear about exciting new developments in 3D genome technologies and workflows.
2. Learn how to use targeted 3D genome methods, including capture Hi-C with Agilent SureSelect probes, to characterize regulatory interactions, non-coding elements, and structural variants.
3. Have a chance to ask questions during a live Q&A following the presentation.

The rise of genomics has transformed life sciences research, providing powerful tools for exploring all manner of biological questions. More recently, advances in protein analysis have made it feasible to analyze the proteome with a throughput and depth of coverage compatible with genomic techniques. The ability to measure proteins at such scale makes it possible to add proteomics to genetic analyses, allowing researchers to extract new insights from genomic datasets and mine additional value from the immense investments made in the field.

Please join us to hear four experts discuss the present challenges and opportunities for using proteomics to power interpretation of genomic data: Melissa Miller, director, human genetics at Pfizer; Chris Whelan, director, neuroscience data science at Johnson & Johnson; Benjamin Sun, head of biomarker genetics at Biogen; and Anders Mälarstig, director of human genetics and computational biomedicine at Pfizer and an affiliate researcher at the Karolinska Institute.

The panelists will discuss topics including how adding circulating protein data to genomic analyses can yield new understanding of complex biology, including around mechanisms of action of drugs and drug pathways and in identifying new biomarkers for drug candidates and drug repurposing.