Genetic Engineering & Biotechnology News’ Post

Pharmaphorum has just published an article regarding the opportunity costs of #genetherapies and how #exosomes fit into the equation. The article explores gene therapy approvals, and discusses some of the novel approaches the industry is adopting to develop #sustainable, #cost-effective, and #lifesaving gene therapies. https://lnkd.in/dxGTzRju

Cell and Gene Therapies were received with well-deserved excitement, but as they've become more common, biopharmaceutical manufacturers will need to navigate emerging challenges: stricter utilization management, regulatory scrutinity, HTA reassessments, and the ever-present challenge of securing ample uptake. Read my collaboration with Trinity's C&GT experts Ismail Ismailoglu, PhD and Julia Pikus to find out more!

Trinity's perspective on 2024 CGT trends to watch!

Excellent overview piece on current CGT trends. Real-time precision PAT analytics are a vital component in ensuring treatment effectiveness and success, from the accurate measurement of cellular fitness and characterization of donor/starting materials, to the measurement of potency throughout the manufacturing process. Novel precision analytics such as LumaCyte Laser Force Cytology adds additional benefits in easing the challenges around comparability, a very challenging piece of the puzzle. Developers who are considering commercial reimbursement contracts tied to patient treatment success must do everything they can to ensure their product is manufactured at the highest level of effectiveness and consistency. The constant release of draft guidance and now final guidance from the FDA focused on advanced therapy manufacturing and potency marks a turning point for our industry to break through status quo processes and invest in advanced analytics and solutions. The demand ahead for these life changing products is high and to meet this need we all must be laser focused on innovation and investing now in novel technologies and next generation analytics and processes that will ensure these treatments are manufactured at consistent and effective scale.

According to Rare Disease Advisor, “2024 will be a breakout year in addressing key challenges to developing cell and gene therapies, especially for rare disorders.”   Sadly, science continues to outpace the commercialization of innovative therapies while the pharmaceutical industry remains slow to change. As a result, patients are getting lost in the shuffle. At MPATHIK, we’re tackling these shifts head-on with empathy and collaboration.   Learn more about what’s expected in 2024 for gene therapies: https://lnkd.in/eWPSz2Jk

New White Paper looks at the solutions and policy options of how to pay for high-cost gene therapies when there is still so much uncertainty about the outcomes and the durability of clinical benefits. https://lnkd.in/gSGhUpBV

When it comes to cell and gene therapy revenues, it’s feast or famine. The feast side is well covered in this A-List, which ranks the top 10 therapies according to sales figures from 2023. The famine side begins almost exactly where this A-List ends. Future A-Lists are bound to push famine farther into the background as additional gene therapies are approved. For example, according to the Alliance for Regenerative Medicine, up to 17 gene therapies could be approved this year alone (up from the 5 gene therapies that were approved in 2023). #CellandGeneTherapy #AdvancedTherapies #RegenerativeMedicine

Health plan sponsors: The era of gene therapies is here. Are you ready for the financial implications? In 2023, seven gene therapies were approved by the FDA, bringing the total to 36 ― with an additional 500 in the pipeline that hold promise for a wide range of diseases and conditions. But as with other novel medical treatments, gene therapies often come with a hefty price tag. In our latest quarterly insight for health plan sponsors, we explore four financing solutions that support sustainable reimbursement and patient access to these high-cost treatments. Get the insight: https://lnkd.in/eJRMpjZJ #genetherapy #healthplans #employeebenefits #costmanagement

Last week my colleagues and I summarized key takeaways from a recent FDA webinar that followed the publication of a final version of a 2022 draft guidance, “Human Gene Therapy Products Incorporating Human Genome Editing.” While the guidance outlines general recommendations that are broadly applicable, rather than specifics relevant to individual gene editing development programs, the webinar provided additional clarity on areas sponsors' may be able to propose alternative studies or methods, with adequate justification. Read our insights here. https://lnkd.in/dSSk9Mua #genetherapy #geneediting

Check out my blog - The rise of Cell & Gene Therapy (CGT) is a real success story for the UK. Project professionals are playing an important role in helping CGT companies upscale their R&D, manufacturing, and implement their real estate strategies at pace, to facilitate their rapidly growing needs etc. Are you a client looking to upscale your CGT R&D and Manufacturing? We can help you align your science plan with your real estate strategy. #celltherepy #genetherepy #lifesciences #pharma #realestate #projectmanagement #costmanagement #projectcontrols #JLL