Asklepios BioPharmaceutical, Inc. (AskBio)’s Post

Was a fascinating Ad Com yesterday @FDA for the first-ever therapy to use CRISPR/Cas9 gene editing (Vertex's very impressive exa-cel gene therapy for sickle cell disease)- "Off target" gene editing, genomic heterogeneity, genome sequencing of edits, and the long term biological consequences of undesired edits was up for discussion. Take homes are its really important to analyse the genetic changes after editing and to hold samples back for long-term follow up (maybe a new guidance coming soon). The potential approval of this product next month is an important step for the CRISPR/Cas9 gene editing field of which there are dozens of other therapies in development.

Droplet digital PCR (ddPCR) is quickly becoming a mainstay of gene therapy. But scientists are struggling with data handling. Discover how the Tetra Scientific Data and AI Cloud™ streamlines ddPCR workflows, improving scientific outcomes in gene therapy.  https://lnkd.in/gzWS6W3E #genetherapy #ddPCR #ScientificDataAndAICloud #ScientificOutcomes

Droplet digital PCR (ddPCR) is quickly becoming a mainstay of gene therapy. But scientists are struggling with data handling. Discover how the Tetra Scientific Data and AI Cloud™ streamlines ddPCR workflows, improving scientific outcomes in gene therapy. https://lnkd.in/ep9NFZTC #genetherapy #ddPCR #ScientificDataAndAICloud #ScientificOutcomes

We are working in partnership with Bio-Works Technologies to develop new Optimer-based resins to improve the manufacture of viral vectors to support the continued rise in gene therapies. There is a clear need in the gene therapy market for affinity resins based on a technology other than camelid-antibodies. The current offerings for the affinity purification of viral vectors offer low binding capacity and are not cleanable using standard clean-in-place (CIP) protocols. As viral vector production continues to scale to support the gene therapy market, more modern solutions are required. Optimer offers tunable affinity with gentle elution for fragile targets, and manufacture that can be simply scaled for commercialisation.

Join Dipika Gurnani for an in-depth discussion on cell and gene therapy safety and purity testing, the challenges involved for manufacturers and how the latest innovations in ddPCR technology can help you: - Test for Replication Competent Virus (RCV) - Bring your gene therapies to market faster #RCV #ddPCR #purity

We are proud to be recognised in the BioIndustry Association (BIA) Cell and Gene Therapy report, which outlines the UK’s pivotal role in advancing life-changing cell and gene therapy research to deliver transformative outcomes for patients living with debilitating or life-shortening conditions. To date, there has been a lack of innovation in treatment options for chronic kidney disease, despite a patient population of over 840 million people worldwide. Purespring’s novel  gene therapy platform, which delivers a disease-modifying gene directly to the podocyte, has the potential to make a real difference to the lives of patients around the world. For more information on the UK’s thriving #CellandGeneTherapy ecosystem, read the full report here: https://lnkd.in/dZJ_4H4Y #GeneTherapy #CellandGeneUK #Biotech #KidneyDiseases

Join Form Bio & PacBio for this upcoming webinar on exploring the future of gene therapy, November 15, 2023 at 10am PT / 1pm ET. Gene therapy is at a pivotal juncture, where precision is absolutely crucial. Complete AAV genome sequencing doesn't just refine vector design; it also unveils concealed packaging challenges. HiFi sequencing data empowers researchers to: 🧬 Identify fragmentation 🧬 Confirm desired transcript expression 🧬 Detect truncations and impurities Register today: https://hubs.li/Q025TDqn0

Discover how the Tetra Scientific Data and AI Cloud™ streamlines ddPCR workflows, improving scientific outcomes in gene therapy. https://lnkd.in/e2AT3HRu #genetherapy #ddPCR #ScientificDataAndAICloud #ScientificOutcomes