While the standard of care has improved for PwCF, efforts are now focused on introducing treatment for all PwCF and on limiting the side effects of current treatment options. In particular, a shift towards gene therapy is envisaged. The development of gene therapies also requires a range of bioanalytical assays to be developed and validated. Watch our recorded webinar, Best Practices in Clinical Gene Therapy Method Development, featuring an AAV Vector Shedding Assay: https://lnkd.in/gfi7Zxqe #CF #genetherapy
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We are quickly approaching an inflection point with respect to using cell therapies to treat #autoimmune disease. While we have increasing confidence that cell therapies will play a pivotal role in the treatment of autoimmune disorders, sponsors will have to overcome significant barriers to deliver on this promise: reducing manufacturing costs, expanding treatment infrastructure, improving the risk/benefit profile, to name just a few. Curious to learn more? Check out our blog post and presentation at #ISCT2024 by Health Advances Cell and Gene Therapy practice co-leader Ned Wydysh PhD, and Jamie Pierson of Parexel. Read our blog here: https://bit.ly/3yZzelC ISCT, International Society for Cell & Gene Therapy #CGT #CellandGene
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Haichen Yang, MD, MA, MBA, Vice President, Clinical Research, Amicus Therapeutics Inc.[NASDAQ: FOLD] Contributed an article "Challenges and opportunities in gene therapy clinical development" “The scarcity of clinical stage gene therapy assets is not just a temporary phenomenon. Rather, the transition of pre-clinical gene therapy assets into clinical stages turned out to be much harder, due to safety concerns, manufacture difficulties, and elevated complexities in clinical development” says Haichen Yang The article will also be featured in the forthcoming special print edition of PTO Public Companies 2024. Read More : https://lnkd.in/gM5ZR9FG #clinicaltrials #regulatoryhurdles #patientsafety #ethicalconsiderations #manufacturingscalability
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An interesting proposal to consider a manufacturer’s product with FDA marketing authorization in a “hold status” as active for NTAP eligibility purposes. Hospitals may be negatively impacted as the timeframe a hospital can obtain NTAP may essentially be shortened for that product.
CMS released the IPPS proposed rule Wednesday. A quick review revealed: ✅ CMS is continuing to slowly make certain SDOH codes CCs to promote increased utilization of those codes ✅ CMS is proposing some changes to NTAP specifically around gene therapies for sickle cell disease: 📌 Increase the NTAP percentage from 65% to 75% for a SCD gene therapy 📌 Proposing to use the start of the fiscal year, October 1, instead of April 1, to determine whether a technology is within its 2- to 3-year newness period. 📌 Beginning with applications for NTAP for FY 2026, CMS is proposing to no longer consider an FDA marketing authorization hold status to be an inactive status for the purpose of the NTAP application eligibility.
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It's almost here! Tomorrow the EMMA International team will be releasing a new White Paper on February 16th: "CRISPR Technology and its Impact on the Regulatory Landscape." As researchers explore the potential of CRISPR in the development of gene therapies, the regulatory landscape becomes a critical aspect of ensuring the safety and efficacy of these groundbreaking treatments. In this White Paper, we will delve into how the FDA navigates and regulates CRISPR technology in the context of gene therapy. Click the link here to explore our expansive library of White Papers: https://lnkd.in/eA7YDkDc
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Did you know that there are currently more than 4000 therapies in development? According to the American Society of Gene and Cell Therapies (ASGCT), there are currently over 4,000 #gene, #cell, and #RNA therapies in development. The ASGCT also noted significant increases in the number of therapies in all three phases of clinical pipeline development, with a notable 11% growth in Phase I programs. This significant development over the years shows promising cell and gene therapy trends. Want to know more about #cellandgenetherapy, join us at Advanced Therapies USA 👉 https://lnkd.in/eaBjYK3y #adtherapiesUSA #advancedtherapies
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Check out CTI's Podcast "Moving Medicine Forward," highlighting the work we conduct in Cell and Gene Therapy: 1. Bret Marshall discusses CTI's involvement in Cardiac gene therapy trials, exploring our impact, goals, and industry trends 2. Michael Schmidt and Nicole Sparshott discuss Chain of Custody best practices 3. Joana Lancastre discusses feasibility considerations for Cell and Gene Therapy Trials 4. Jaclyn Guillory shares CTI's experience with enrolling patients to trials with targeted therapies #CTIPodcast #MovingMedicineForward #CRO #ASGCT24 #CellTherapy #GeneTherapy
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Launching revolutionary cell and gene therapies (CGT) from lab to patient is a complex but rewarding challenge. 🧬💡 Understanding the BLA filing process and navigating clinical trials are pivotal steps in this quest. 🔍 Dive into the key considerations for BLA filing, ensuring your groundbreaking CGT advancements are set for success. It explains the complexities and breakthroughs in cell and gene therapy development. Ready to learn more? Click the link here ➡️ https://lnkd.in/dc-dqA-Q Have questions about navigating clinical trials? 🤔 LaSalle Group is here to help! #CellGeneTherapy #ClinicalTrials #BiotechInnovation #HealthcareProgress
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It's almost here! On Friday the EMMA International team will be releasing a new White Paper on February 16th: "CRISPR Technology and its Impact on the Regulatory Landscape." As researchers explore the potential of CRISPR in the development of gene therapies, the regulatory landscape becomes a critical aspect of ensuring the safety and efficacy of these groundbreaking treatments. In this White Paper, we will delve into how the FDA navigates and regulates CRISPR technology in the context of gene therapy. Click the link here to explore our expansive library of White Papers: https://lnkd.in/eA7YDkDc
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New policy update! 🏛️📜 ARM supports the U.S. Administration's proposal to strengthen programs that incentivize the use of cell and gene therapies. The proposal also seeks clarity and transparency on the process of establishing a regulatory framework in Medicare that aligns with the incoming pipeline of advanced therapies to expand patient access. Read more about ARM's views on the proposal and how it will impact the cell and gene therapy sector here: https://lnkd.in/eM-bEKvi
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Business Manager | Quality Control Solutions | Bioproduction | Sterile Pharmaceuticals | Cell & Gene Therapy | Personal Care
Microbiological examination of cell-based products is critical to ensure safety prior to patient infusion. 💉 The harmonized chapters of the compendial sterility test method are not currently adapted for cell and gene therapy products due to three major constraints: 1️⃣ Time To Result 2️⃣ Method Subjectivity 3️⃣ Matrices Compatibility 💡 Download bioMérieux's comprehensive white paper to learn how BACT/ALERT® 3D DUAL-T overcomes these challenges to assess the sterility of cell and gene therapies, making a positive impact on product release and patient health.
