Jennie Crabbe’s Post

Retina gene therapy clinical trial news from Market Scope: 4D Molecular Therapeutics reported Feb. 3 that it had seen positive topline results from the Phase II PRISM trial evaluating 4D-150, an intravitreal gene therapy candidate, in wet age-related macular degeneration (AMD) patients with severe disease activity and a high treatment burden. At 24 weeks, in the high-dose group, 84 percent of patients received none or one supplemental #aflibercept injection—representing an 89 percent reduction in the annualized anti-VEGF injection rate. Visual acuity and central subfield thickness were both stable vs. aflibercept. #retina #clinicaltrial #genetherapy

Cell & Gene Therapy: Market Access & Regulatory Challenges can facilitate these transformative therapies being available for patients with devastating diseases, but not addressing these challenges transparently or head-on can prove fatal for drug development programs. EOS Intelligence is one of the leading experts in GCT Market Access, Regulatory, BD&L and CI insight and intelligence - if you wish to know more about this report and the data behind it, DM me and I will be happy to provide further insight. #cellandgenetherapy

My colleague Stuart Wagner, MBA at EOS Intelligence has recently published some excellent insights on the Market Access & Regulatory Challenges facing the exciting Cell & Gene Therapy space. If you're interested in staying up to date on the latest developments in this field, I highly recommend giving his work a read. Be sure to share your thoughts and join the conversation!

(Benefit-Risk Management / Cell and Gene Therapy / Managed Webpage) Besides the general FDA guidance search database, there are also specially managed webpages that aggregate topics such as CGT products. Recommend to bookmark this page if you're in this space. Look on the left margin for other product groups. Certainly an excellent primer for those "starting" in this space. Enjoy. #fda #cellandgenetherapy #celltherapy #genetherapy #biologics #guidance

The landscape of gene therapy is burgeoning with possibilities, offering promising advancements to patients with genetic disorders. In a revealing conversation with European Pharmaceutical Review Magazine, Roger Palframan, head of U.S. research at UCB, sheds light on the current trends, key developments, and future disruptors in gene therapy. His insights offer a glimpse into the potential of gene therapies. You can read the full interview 👉https://lnkd.in/exhVR3kp

As many of you are aware, in late December the FDA announced the availability of a new draft guidance for advanced therapies, which provides recommendations for developing a science- and risk-based strategy to help assure the potency of human cellular therapy or gene therapy products. A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through manufacturing process design, manufacturing process control, material control, in-process testing, and potency lot release assays. Understanding a product’s potency from the start is incredibly valuable… ensuring patients receive the full extent of a product's intended therapeutic effect. Laser Force Cytology analytics are a very important piece of an advanced manufacturing strategy due to the technology’s ability to deliver real-time insights, highly precise and reproducible data and all without the need for labels or florescents. Real-time PAT data insights hold the key to improved production and product consistency… it is great to see the increased focus on the need for precision analytics earlier in process development so that through a matrix approach, these data can allow for better understanding and rapid optimization throughout the manufacturing process. #CellTherapy #GeneTherapy #FDA #CGT

As cell and gene therapies (CGTs) rapidly advance, so does the need for a robust #digitalecosystem. Discover how embracing a digital ecosystem can revolutionize CGT companies to: • Enhance supply chain visibility • Reduce operational costs • Improve #patientcare experiences Our latest #whitepaper provides a detailed roadmap for #CGT stakeholders to navigate the complexities of #digitaltransformation effectively. Read it here: https://bit.ly/4aEblgQ

Potency Assurance for Cellular and Gene Therapy Products "A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through manufacturing process design, manufacturing process control, material control, in-process testing, and potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect." #FDA #celltherapy #cellandgenetherapy #manufacturing

It’s difficult to navigate the balance of phase-appropriate needs with commercial readiness during cell and gene therapy development. Partnering with a trusted and experienced viral vector #CDMO can help unlock unique insights and provide guidance to achieve the right balance for your therapeutic. Here are 4 recommendations to keep in mind during preclinical and clinical development:

🌟 The UK's #Cellandgene therapy sector is more than research – it's about transforming lives 🧬 In 2022, companies like Autolus Therapeutics and Orchard Therapeutics, rooted in UK brilliance, turned groundbreaking work into tangible results. Discover how the UK leads in patient-centric innovations, rewriting healthcare boundaries 🌐 https://ow.ly/4RfO50Qs8Es #CellandGeneUK