Pharmaron’s cell and gene therapy webinar series

Cell therapy development programs generate encouraging pre-clinical and clinical results with the promise that these tailored therapeutics can provide a curative rather than maintenance therapy. Therapies are being developed across the spectrum from academic institutions to small and medium biotechs/biopharma as well as large pharma companies which have all invested significantly in the cell and gene therapy (CGT) field. As additional CGT therapies gain approval, specifically in solid tumors and auto-immunity, the addressable patient populations will grow requiring scaled -out/-up commercial manufacturing. Although the CGT field is experiencing significant tailwinds, challenges remain. Cell sourcing has long been considered a pain-point which is potentially compounded with the necessity to develop robust GMP manufacturing processes and appropriate analytical methods.

Xdrop® is a microfluid droplet technology that enriches and amplifies long DNA fragments of largely unknown sequence from low sample input. Genomics researchers have coupled it with long and short-read sequencing (Oxford Nanopore, PacBio and Illumina) to gain high-resolution insights into genomic regions that have previously been inaccessible.  Examples of its use in unraveling plant genomes include closing gaps due to poor genome assemblies or low correspondence between reference genome and plant variety, resolving structural rearrangements like transposable elements, and examining gene clusters without performing whole genome sequencing. Xdrop can be applied in relation to QTL mapping and bulk segregant analysis by enabling targeted sequencing of the identified region of interest in several segregating plants with the aim of identifying the polymorphisms either linked to or causing the phenotype of interest. The long-range genomic information (100 kb) and single-molecule resolution achieved with Xdrop target enrichment simplifies working with large and structurally complex genomes with an easy capture design and workflow.

Discussion on this webinar was lively and highly informed. Watch the recording below to see the Foghorn Therapeutics collaborative scientific team of David Lahr (Head of Informatics), Joe Marchionna (Research Informatics), and Johannes Voigt (Computational Chemist), together with An2h Discovery CSO Jennifer Johnston, as they reveal cutting-edge informatics workflows with CDD Vault.

Gain an understanding of how to help maintain the proper ethical pain management of patients. We are practicing in the midst of a national crisis in which every day over 130 people in the US die of overdosing from narcotic prescriptions overdose, heroin, and other related drugs. The pain was described as the "5th vital sign" by some drug companies in the 1990s and stated that patients would not be addicted to pain killers and providers began to overprescribe them in order to adequately treat pain. Approximately one-third of the chronic opioid user for none malignant pains misuse them. The incidence of neonatal abstinence syndrome has been on the rise due to widespread opioid use. CDC has published new guidelines as for how to counteract this crisis and regulatory agencies including states medical boards and DEA has increased enforcement against irresponsible and fraudulent opioid prescription writers to include license revocations and jail terms.