Polyplus now part of Sartorius’ Post

In November 2023, the United States Pharmacopeia issued draft General Chapter <1040> Quality Considerations for Plasmid DNA as a Starting Material for Cell and Gene Therapies. The draft updates previous guidelines released on the production of plasmid DNA both as a therapeutic product and as a critical starting material. To better understand these draft guidelines, I posed several questions to USP's Rebecca Potts, Ph.D., who is the lead expert on the panel that developed the general chapter. I was particularly interested in how standards vary for GMP- and non-GMP-grade plasmids and the thinking behind these new guidelines - and this is hopefully captured the article below. https://lnkd.in/eENCqjMB

Preclinical data suggests that our placenta-derived unmodified natural killer (NK) cells (CYNK-001) and genetically modified NK cells (CYNK-201) may serve as potent and selective senolytic agents for use in addressing age-related diseases, and we're excited to present the data at the ASGCT Annual Meeting. #nkcells #cellularsenescence #asgct #celltherapy

Charles River Laboratories and AAVantgarde Bio have entered a CDMO agreement to produce GMP plasmid DNA. AAVantgarde Bio, with its innovative AAV vector platforms for large gene delivery, will use Charles River Laboratories' expertise to advance treatments for inherited retinal diseases, beginning with Stargardt's disease. Stargardt's, the most common inherited macular dystrophy, affects 1 in 6,500 people and currently has no therapies. This partnership leverages Charles River Laboratories’ GMP plasmid DNA CDMO center in Keele, UK, to support AAVantgarde Bio's Stargardt's disease program. Read more below about this agreement. https://lnkd.in/eV23ymd9 #CharlesRiver #AAVantgarde #GeneTherapy #CDMO #HealthcareInnovation

SparingVision Selects Second Target in Strategic Collaboration with Intellia Therapeutics “Combining SparingVision’s unparalleled knowledge of retinal disease with Intellia’s leading clinical-stage CRISPR-based technologies, the collaboration’s shared goal is to radically change the treatment of blinding ocular diseases by leveraging the power of gene editing. Under the terms of the partnership initiated in October 2021, Intellia granted SparingVision exclusive rights to its proprietary in vivo CRISPR-based technologies for up to three ocular targets addressing diseases with significant unmet medical need.” https://lnkd.in/enGG448u

Nice study using mRNA and AAV to mediate simultaneous gene editing and precise transgene knock-in using massively parallel homology-directed repair, producing a pool of stably integrated mutant variants each with targeted gene editing. https://lnkd.in/gxTUtqff

Phase I/II trials for Ocugen’s gene therapies, OCU410 and OCU410ST, are planned for initiation by the end of 2023 in geographic atrophy and Stargardt disease. https://lnkd.in/duT3ugcX #regulatoryaffairs #regulatory #regulatorycompliance #regulatoryintelligence

What can you learn from 34,865 host cell proteins gathered from across 500 projects?   At Alphalyse, we have curated a comprehensive database with data on 34,865 uniquely quantified Host Cell Proteins (HCPs) from 500+ HCP projects. It includes mAbs, recombinant proteins, vaccines, and gene therapy products – originating from 45 different host cell organisms and 49 distinct strains and viral vectors, such as CHO, E. coli, HEK, Sf9, AAV, and Lentivirus.   We have done this by leveraging data-independent and data-dependent mass spectrometry techniques and using intact proteins as internal standards for normalizing the data.   This robust workflow ensures reliable and reproducible HCP analyses – and enables comparison of HCP levels between projects.   At this year’s BEBPA HCP Conference, bioinformatics specialist Victor Chrone will share our insights about problematic HCPs across drug types and essential method parameters for assay quality control.   Don’t miss your chance to learn how LC-MS can enhance your understanding of your product’s HCP profile and empower informed decision-making for HCP clearance and monitoring.   Don’t delay – sign up today! Your last chance to register for the BEBPA HCP Conference is May 7: https://lnkd.in/eZVm7vB7   We look forward to seeing you there!

🤫 We’re excited to reveal a few details about this year’s program MSS2024’s program will be divided into 2 main sections: 🦠 Viruses, VLPs & nanoparticles: featuring vaccines, gene therapy vectors, phage therapies, LNPs & EVs 🧬 Nucleic acids: featuring pDNA & mRNA Both of these sections cover a wide spectrum of topics, allowing you to gain theoretical knowledge from lectures 👨🏫 and practical insights from wet-lab workshops. 🧪 Lectures and workshops for both sections will take place interchangeably, Monday and Tuesday will feature oral lectures on nucleic acids and workshops on viruses VLPs & nanoparticles, and the other way around on Thursday and Friday. Wednesday will stand out from the other days as oral lectures will focus on the Practical aspects of monolith technology and hand-on workshops will showcase PATfix, an at-line analytical system and a new automated in-line lysis system that’s going to be launched during MSS2024! 🤫 For a sneak peek at our attendee list for MSS2024 be sure to look at the attached graphic 🤫   📄 Discover our speakers and read their abstracts: https://lnkd.in/e6yDrxec 💡 -30% Early Bird registration offer until March 31st 📅 Abstract submission is open until February 29 MSS2024 | June 3-7 | Portoroz, Slovenia or virtually MSS2024 will bring together renowned experts from industry and academia to explore the latest innovations and best practices in purification and analytical solutions for emerging advanced therapies and vaccines. Whether you're a researcher, scientist, or technology provider, this event is your chance to gain valuable insights, network with industry leaders, and expand your knowledge base. 👉 Register here: https://lnkd.in/dgEqqV7v 👉 Discover more: https://lnkd.in/ecRfj6SQ #MSS2024 #GeneTherapy #Chromatography #Vaccines #Biotechnology #BioPharma

Balancing qPCR and ddPCR in Therapeutic Development Over the decades, several techniques for measuring RNA expression, including microarrays, PCR, and RNA sequencing (RNA-Seq), have facilitated the discovery and optimization of several therapeutics. In this article, we’ll focus on two of the most common and sensitive techniques for studying gene expression, reverse transcription quantitative PCR (RT-qPCR) and Droplet Digital™ PCR (ddPCR™), and how each can be used at different stages of therapeutic development. #ddpcr #research #medicine #lifesciences #rna #rnaseq #rnatherapeutics

Assessment of lentiviral titer is an essential component of ensuring the quality and safety of cell and gene therapies created with lentiviral vectors. In this paper, researchers describe a universal ddPCR-based assay for determining viral titer and vector copy number in modified cells, demonstrating the value of Droplet Digital PCR technology for sensitive and accurate quality control testing in cell and gene therapy manufacturing. Read it here: https://ow.ly/XaUa50RhEb3 #ViralTiter #CellTherapy #GeneTherapy #ddPCR