Premier Research’s Post

Rich insights from Industry Giants - Rob Schulz and James Wabby. Take a moment to listen, truly worthwhile. Regulatory intelligence must consider cultural norms and political climate to drive a successful regulatory submission strategy. Find out how.

Following on from our post earlier this week giving details of BioPhorum Advanced Therapy Medicinal Products’ introductory paper on validation challenges, we would now like to highlight the paper in which our expert members discuss leveraging platform and process characterization data to accelerate CGT validation and commercialization. You can download the paper here: https://lnkd.in/euuCKFca   Or view the webinar here: https://lnkd.in/eJdXZDNn With the number of approved cell and gene therapies growing, there is a strong desire to create common approaches to manufacture novel therapies by leveraging past knowledge. Members of BioPhorum Advanced Therapy Medicinal Products evaluated and summarized opportunities to leverage process development and characterization knowledge across similar technology platforms to help accelerate subsequent process validation activities. To view BioPhorum Advanced Therapy Medicinal Products’ full portfolio of publications visit:  https://lnkd.in/exqf9Khu  #celltherapy #genetherapy #platform

💫 What a busy start today in London! 🇬🇧 There's still time to meet this afternoon, and if you don't catch us today, there's also an opportunity tomorrow. 👉🏼 We're ready for another productive day at booth #111! 💡 Discuss together how ReiThera’s capabilities can advance your gene therapy program or viral vaccine from preclinical to clinical and commercial production. #gmp #biotech #biotechnology #genetherapy #cellandgenetherapy #advancedtherapies #adtherapies24

To close the week, we would like to remind you of the final slide deck/presentation created by experts within BioPhorum Advanced Therapy Medicinal Products on validation challenges and method validation controls. You can download the slide deck/presentation here: https://lnkd.in/e6HKtXT8 This method validation controls content reviews the regulatory challenges faced in cell and gene therapy with a focus on identifying and using appropriate analytical controls when dealing with both limited regulatory guidance and limited materials - thus providing possible options to avoid potential issues and accelerate the validation process. The content focuses on challenges such as appropriate controls and standards, alternative materials when resources are scarce, and the relevance of reference standards when processes change. To view BioPhorum Advanced Therapy Medicinal Products’ full portfolio of publications visit: https://lnkd.in/exqf9Khu #ATMP #celltherapy #genetherapy #validation

🔔  Circular RNA news ! ⭕ Orna Therapeutics revealed the acquisition of ReNAgade Therapeutics ! ORNA is a main player in the field of circular RNA, developing circular RNA (oRNA®) therapeutics and ReNAgade unveiled new LNPs for delivering mRNA to extra-hepatic tissues. According to ORNA’s press release, this strategic move « unifies two industry-leading circles with the knowhow to significantly expand circular RNA engineering and delivery technologies and advance in vivo CAR and gene editing approaches for a range of diseases ».   💡 Get more information on ReNAgade Therapeutics ionizable lipids with KnowMade’s Insight https://lnkd.in/eGvc_-R5   💡  Get more information on circular RNA with KnowMade’s Insight https://lnkd.in/gns4SXhP 🔹 For an overview of KnowMade’s activities dedicated to mRNA therapeutics see the dedicated webpage 👇 https://lnkd.in/gney6ZhX   #mrna #circular #LNP

We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field. https://lnkd.in/exhRKaUz

Want a first look at our innovative platform for unlocking patient access to life-saving cell and gene therapies? Heading to ISCT, International Society for Cell & Gene Therapy's conference in Vancouver? Then book a live demo with the Ori Biotech team here - https://lnkd.in/eANBmhCP Not lucky enough to be joining, but still want to check it out? Sneaky peek below 👇

Working hard to efficiently develop safe, effective cell and gene therapies? Watch our latest video on overcoming limitations in CGT development and product characterization with #NGS-based assays! Genedata is your trusted partner to develop streamlined NGS-based assays for #CellTherapy or #GeneTherapy and bring them to production faster. Our platform supports your #biosafety, identity, and integrity testing through the entire development and manufacturing process with comprehensive, automated, yet configurable workflows. Unlock more time for insight generation! Discover more about #GenedataSelector here: https://buff.ly/3XhLZ30 #DigitalizingBiopharma #Bioinformatics #DataAnalysis

Biocom California recently invited Sapient to contribute an article for its Lifelines Magazine edition focused on innovations in #genetherapy and #celltherapy. https://bit.ly/3V6gqd4 While cell- and gene-based treatments are advancing rapidly, spurred by a landmark year of FDA approvals, their development is not without challenges. We discuss how #multiomics for gene and cell therapy can deliver the novel insights needed to realize the incredible therapeutic potential represented in these pipelines today: https://bit.ly/3V6gqd4

Over the next two weeks, we would like to reintroduce you to the great content our members have created to address the various challenges around validation within ATMPs.    You can download our validation challenges paper here: https://lnkd.in/e73dpJd8    Or watch the webinar summarising the paper’s content here: https://lnkd.in/eg5NMYSK    Within this paper, members of BioPhorum Advanced Therapy Medicinal Products highlight the complexity and challenges associated with validating cell and gene therapy products, as well as sharing potential options to help resolve them.    Some of the challenges showcased in the paper include having a limited number of batches available at commercial scale prior to and during PPQ, assay variability, and reference standards.    Having greater awareness of possible issues and addressing them as early as possible will help provide a clear and fast path for successful validation of process and analytical methods. We therefore hope you will find these resources of value.    For further information on BioPhorum Advanced Therapy Medicinal Products’ publications visit:    https://lnkd.in/exqf9Khu    #ATMP #celltherapy #genetherapy #validation