Drug Development and Industrial Pharmacy’s Post

Over 90% of CRISPR off-target effects detected using ResolveDNA® were missed by a prediction algorithm. This reveals a gap in methods used to detect and characterize unintended edits that occur with CRISPR/Cas9 activity editing, with potential implications for validation of cell or gene therapy candidates. Watch now, to learn how the primary template-directed amplification (PTA) chemistry that drives ResolveDNA® can be used to detect and characterize genetic changes following CRISPR/Cas9 gene editing. https://lnkd.in/eDnRzUSQ #genome #transcriptome #multiomics #CellTherapy #GeneTherapy

Prediction algorithms can tell you where they predict off target edits to take place. ResolveDNA will tell you where they actually take place and, how many cells you sequence have those off target edits.

CRISPR Alternative ✨ Have you heard about Applied StemCell's TARGATT site-specific knock-in technology? Our TARGATT system facilitates single copy insertion of large DNA fragments at a preselected safe harbor locus in iPSCs, HEK293 cells, and CHO cells. - Site-specific knock-in at a preselected safe harbor locus (no random integration) - Single-copy gene insertion - Large transgene knock-in (up to 20 kb) - Uninform expression - High integration efficiency & high-level expression of the transgene With TARGATT, you can avoid the limitations of standard gene editing technologies and create stable, high-expression engineered cells for your research. Start by exploring ASC's TARGATT technology today. It may be what you need to expand your research! Learn more @ https://lnkd.in/dxWDcEC Or contact us at [email protected] We also invite you to visit our new TARGATT technology page: 🔗 https://lnkd.in/gdGSnQ2z #TARGATT #geneediting #iPSCs #cellandgenetherapy

We had an engaging few days at the Next Generation Gene Therapy Vectors Summit! Viral vectors continue to predominate and evolve as a delivery modality. Our expert, Zeeshan Shaikh, highlights the standout conference insights: ✔ Significant efforts to optimize vector design for safety and tissue tropism. For example, improving liver targeting and tropism.   ✔ Most new capsid discovery approaches involve directed evolution and rational design. Innovative approaches are using bio-conjugates that enhance capsid properties.   ✔ Improvements in vector payload capabilities. E.g., EG 427 's non-replicating HSV vector can deliver 26 Kb payloads, ideal for delivering gene editing constructs with ease. Carbon Biosciences parvovirus vector allows payloads up to 6.3 Kb.   Scaling new and innovative vectors with unique capsid properties remains a challenge making it imperative to focus on manufacturing efforts. Any further insights? Drop it in the comments below! #biomanufacturing #innovation #viralvectors

🤔 What are the main features of Lipid Nanoparticles (LNPs), the most promising nanoformulations in the clinic? And why have they surpassed liposomes? Our latest blog dives into their unique structures, efficiencies, and versatile applications. Discover how DIVERSA's innovation is contributing to the field and the development of advanced gene therapies with complex molecules such as mRNAs. Check out the blog for a detailed comparison and insights into current lipid nanocarriers for gene delivery. 👇🏼 https://lnkd.in/gduh3fx8 Biocat, BioRegion of Catalonia CELTA INGENIEROS SL Labclinics, S.A LubioScience PROGEN Tebubio ZAGENO Inc. #drugdelivery #nanoparticle #deliveryvehicles #nanotechnology #nanomedicine #liposomes #lipidnanoparticles

Don't forget to join us today at 3pm GMT / 4pm CET /10am EST for the first webinar in our 3 part series on CRISPR and Gene Editing ONLINE. If you can't make it you can still register to catch up on-demand: https://lnkd.in/dA9KycdX   Today, we'll be discussing advances in gene editing systems. We'll be highlighting their applications and benefits and discussing the future of gene editing technologies, both from a technological standpoint and in relation to future therapeutics. Talk 1: Precise Transcript Targeting by CRISPR-Csm Complexes David Colognori, Post-Doctoral Associate, Doudna Lab, UC Berkeley    Talk 2: PRCISR CRISPR: Vivlion’s CRISPR-enabled PCR-free discovery platform   Martin Wegner, Head of R&D, Vivlion GmbH    Talk 3: Precise and Versatile Genome Engineering with Prime Editing  Andrew Anzalone, Head of the Prime Editing Platform and Scientific Co-Founder, Prime Medicine, Inc. #CRISPR #geneediting

As if Stunner was not already the BEST characterization tool - it just got better! Check out this link to watch the recording :) https://lnkd.in/dgq6A3qu

Prime editing, a novel way to edit genes that offers more functionality than CRISPR, is one step closer to human use. In a presentation at the annual European Society of Cell & Gene Therapy Congress in Brussels, Prime Medicine, Inc. announced that its prime editing technology was successfully used in primates to correct a rare liver gene mutation. The team said that although they will have to see clinical data before they can determine the clinical benefit in humans, “high-level prime editing in NHP is likely to support that we can achieve high-level editing in humans.” Learn more about this promising research here: https://hubs.li/Q02dHB9R0 #primemedicine #cellandgenetherapy #geneediting #liverdisease #biotech #cellandgenetherapy #CRISPR #liverdisease

#AAVdownstream challenges: expert insights #AAVvectors are a primary delivery mechanism for #genetherapies due to low immunogenicity, long lasting gene expression, and the ability to target specific tissues. The field of #AAV manufacturing is rapidly expanding, but there are challenges in the downstream processing of these #viralvectors. Next month, an expert panel including Nathalie Clément (VP, Vector Development for Translational Gene Therapies, Siren Biotechnology), Matt Roach (Director of AAV Production, BridgeBio), Nicolas Laroudie (Senior Field Application Scientist, Thermo Fisher Scientific), and William Kish (Principal Scientist - Downstream Process Sciences Engineering, Taysha Gene Therapies) will provide an opportunity to learn more about the latest trends and issues in #AAVmanufacturing, and specifically, how to address the most common downstream processing challenges Register for this #webinar below:

Today is National DNA Day, a day to celebrate the groundbreaking discovery of the double helix in 1953 and the successful completion of the Human Genome Project in 2003. On days like today, we are reminded of the important research and discovery that have resulted in two of the greatest scientific advancements of all time, both of which have accelerated advancements in #GeneTherapy and remain a significant driver of progress and #innovation to date. At Purespring, we have built on these powerful discoveries and are proud to be the first company to treat kidney diseases by directly targeting the podocyte with AAV gene therapy. Learn more about our novel approach here: https://lnkd.in/d8_G__CR #KidneyDisease #DNADay #Biotech #CellAndGeneTherapy