INSIGHTS ON CELL & GENE THERAPY DISCOVERY

• Drug Repurposing Trends And Strategic Approaches For Shortening Timelines

  As the complexity of drug development increases, so does the industry’s focus on strategies and solutions that can help bring advanced products to market as quickly as possible.

• Can Gene Therapy Treat Dominantly Inherited Disorders?

  Recent applications of adeno-associated virus (AAV) mediated gene therapy have mainly focused on correcting recessively inherited diseases. What about dominantly inherited disorders? AAV could be a delivery mechanism for treating those, too.

• Big Gains In FSHD Research: A Newly Published Model of FSHD And A Potential Gene Therapy To Improve Functional Outcomes

  Facioscapulohumeral muscular dystrophy (FSHD) is the most prevalent dominantly inherited muscular dystrophy in the world. The new model aims to provide the basis for future studies to bring therapeutic options to patients with FSHD.

• Preclinical Gene Therapy Study Shows Muscle Restoration In Charcot-Marie-Tooth Disease Model

  A gene known to repair damaged nerves also repairs damaged muscle, a team of investigators at Nationwide Children’s Hospital discovered.

• Preventing The Development Of Muscular Dystrophy Through Surrogate Gene Therapy

  Researchers have identified a key regulatory protein implicated in Galgt2 overexpression and began to elucidate its protective mechanism against muscular dystrophy.

• Krabbe Disease Successfully Treated With Gene Therapy In Preclinical Animal Model

  Krabbe disease is an aggressive, incurable pediatric neurodegenerative disease caused by mutations in the galactosylceramidase (GALC) gene. The standard of care for presymptomatic babies is hematopoietic stem cell transplantation (HSCT); however, the morbidity and mortality of HSCT is high. A recent study published in The Journal of Clinical Investigation reports the successful treatment of Krabbe disease in a preclinical animal model using gene therapy.

• Simplifying And Expediting Inflammatory Biomarker Research With Ella

  The Global Forum on Childhood Pneumonia estimates that 11 million pediatric lives will be lost by the year 2030 if more global action is not devoted to accelerating research efforts against this disease. Among the actions required, is a commitment to research partnerships and breakthrough innovations to both advance our understanding of the immune response during infection and identify earlier time points for therapeutic intervention. Read on to learn how a team of clinician-scientists at Penn State Health are working to address this challenge using Simple Plex assays on Ella.

• Characterizing CAR T Cell Therapy Biomarkers Through Multianalyte Analysis

  Identification and monitoring of biomarkers related to T cell activation and associated cytokine release syndrome (CRS) will be necessary to fully realize the immense potential of chimeric antigen receptor (CAR) T cell therapy. See how the Ella immunoassay platform with Simple Plex™ multianalyte assays enable fast and accurate quantitation of analytes of interest and allow for the detailed analysis of an individual’s response to T cell infusion.

• Vericheck ddPCR Mycoplasma Detection Kit

  The Vericheck ddPCR Mycoplasma Detection Kit is the first Droplet Digital PCR (ddPCR) based Mycoplasma testing solution. The probe-based sensitive and specific kit has been designed and validated to meet European, U.S., and Japanese pharmacopoeia requirements and can detect up to 122 Mycoplasma species.

• Transitioning Your Assay From Quantitative PCR To Droplet Digital PCR

  There are similarities and differences between quantitative PCR and Droplet Digital PCR. Here we will show data that provide guidance for migrating a qPCR assay that uses a standard curve to a ddPCR assay that directly quantifies a sequence without a standard curve. Learn about how transitioning your assay from quantitative PCR to Droplet Digital PCR can be simple and provide consistent results.