Alliance for Regenerative Medicine’s Post

Gene Therapy in Brazil

"We’re trying to administer 21st-century medicines within a 20th-century payment model,” said Jay Newman, SVP of Commercial Development, Pricing & Reimbursement at Spark Therapeutics, Inc., during the 2024 BIO International Convention held from June 3 to 6 in San Diego, California. The annual event brought together #caregivers, #patients, #hospitals, #policymakers, #manufacturers, and #innovators to discuss the potential of gene therapy and address issues related to #access. Panelists emphasized the need to shift discussions from the #cost of these drugs to their #value and highlighted how the clinical journey of cell and gene therapy creates new #patientneeds. They also discussed how states must develop #health plans that can cover the cost of these drugs without bankrupting patients while allowing states to balance their budgets. Michelle Campbell, mother of a daughter with β-#thalassaemia major, provided the #patientperspective during the panel “Pioneering Gene Therapy in the Real World.” https://lnkd.in/dnT_QwsH #BIO2024 #Biotechnology #Innovation #AdvancedTherapies #CGTs

🇬🇧 🧪 🏆 The #UK leads Europe in cell and gene therapy! Thanks to BioIndustry Association (BIA) for the new report, which highlights why there is no mistaking that the UK is cementing position as a global leader in #cellandgenetherapy: 🧬 UK companies secured 55% of Europe's cell and gene therapy VC funding in 2023 🧬 Clinical trial leader: the UK surpasses all other European countries with 84 drugs in clinical development 🧬 23 cell and gene therapies are already approved for patient use in the UK, demonstrating the sector's ability to deliver tangible benefits. Well done to the incredible UK businesses pioneering innovation in this sector: Autolus Therapeutics, Purespring Therapeutics, Orchard Therapeutics and Rinri Therapeutics 👏 👏 👏 Link to the press release and report: https://lnkd.in/dSnEaENS

This month, the sector has seen some notable developments in Brazil. The country granted marketing authorization to Upstaza, a gene therapy developed by PTC Therapeutics, Inc. to treat AADC deficiency. This is the third gene therapy product approved in Brazil, with Novartis’ Zolgensma and Spark Therapeutics, Inc.'s Luxturna both approved in 2020. https://lnkd.in/eE-kvhfE Additionally, GEMMA Biotherapeutics announced last week that it reached a $100 million agreement with Brazil’s Fiocruz, a public health research institute, to develop and manufacture cell and gene therapies for rare diseases. https://lnkd.in/eMH2ynNJ These are encouraging developments for advancing global access to cell and gene therapies beyond regions like North America, Europe, and East Asia.

𝐋𝐍𝐏 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 𝐥𝐚𝐧𝐝𝐬𝐜𝐚𝐩𝐞 🧬 Lipid Nanoparticles (LNPs) are 𝘷𝘦𝘳𝘴𝘢𝘵𝘪𝘭𝘦 𝘥𝘦𝘭𝘪𝘷𝘦𝘳𝘺 𝘸𝘪𝘻𝘢𝘳𝘥𝘴 in the world of biopharma and healthcare. Their main mission? To serve as the ultimate couriers for therapeutic molecules, with a special focus on mRNA-based therapies, vaccines, and gene therapy. 𝘍𝘪𝘷𝘦 𝘓𝘕𝘗 𝘤𝘰𝘮𝘱𝘢𝘯𝘪𝘦𝘴 𝘵𝘰 𝘬𝘦𝘦𝘱 𝘺𝘰𝘶𝘳 𝘦𝘺𝘦 𝘰𝘯 👇 - Hopewell Therapeutics, Inc. - METiS Therapeutics - Nanite - NanoVation Therapeutics™ - SiSaf Ltd LNPs are expertly designed 𝘮𝘪𝘤𝘳𝘰𝘴𝘤𝘰𝘱𝘪𝘤 𝘥𝘦𝘭𝘪𝘷𝘦𝘳𝘺 𝘷𝘦𝘩𝘪𝘤𝘭𝘦𝘴 with a paramount mission to bolster stability, facilitate cellular uptake, and orchestrate the precise delivery of therapeutic molecules. 𝐋𝐍𝐏𝐬 𝐟𝐮𝐧𝐜𝐭𝐢𝐨𝐧 𝐚𝐬 𝐯𝐢𝐠𝐢𝐥𝐚𝐧𝐭 𝐠𝐮𝐚𝐫𝐝𝐢𝐚𝐧𝐬, shielding delicate mRNA from the clutches of degradation, ensuring its secure voyage to particular cells or tissues within the body. Proficiently navigating through biological obstacles (Source: Benjamin McLeod, MBIOT and Springer Nature Group). ➡️ Tackling the world of LNPs brings forth a 𝐟𝐨𝐫𝐦𝐢𝐝𝐚𝐛𝐥𝐞 𝐬𝐞𝐭 𝐨𝐟 𝐜𝐡𝐚𝐥𝐥𝐞𝐧𝐠𝐞𝐬. These include potential concerns related to the intricate process of fine-tuning LNP formulations, achieving precise cell targeting, ensuring stability during storage and transportation, scaling up production, surmounting biological barriers, adhering to stringent storage requirements. Also, navigating the ever-present complexities of regulatory approval processes and managing heavy production costs isn't an easy one... P.S. - Want to stay ahead of the biotech revolution? Sign up for FREE to this week's 𝐂𝐆𝐓𝐰𝐞𝐞𝐤𝐥𝐲 newsletter and discover the newest companies, clinical updates and breakthroughs, here: https://lnkd.in/etZK5kzZ #biotechnology #CGTweekly #mrna #genetherapy

Are currently facing regulatory challenges in your cell or gene therapies❓   Now's your chance to achieve seamless approvals in your first regulatory submissions with support from the 𝗖𝗲𝗹𝗹 & 𝗚𝗲𝗻𝗲 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗔𝗳𝗳𝗮𝗶𝗿𝘀 𝗦𝘂𝗺𝗺𝗶𝘁.   📚𝗥𝗲𝗮𝗱 𝘁𝗵𝗲 𝗙𝘂𝗹𝗹 𝗔𝗴𝗲𝗻𝗱𝗮 - https://ter.li/t7h8j0   Take this opportunity to learn from top regulatory experts at AstraZeneca, Bristol Myers Squibb, CytoImmune Therapeutics, and more as they delve into every aspect of cell and gene therapy regulation; from pre-IND to post-BLA, benchmarking best practices, and preparing for every critical milestone.   🔍𝗙𝗶𝗻𝗱 𝗼𝘂𝘁 𝗠𝗼𝗿𝗲 - https://ter.li/t7h8j0   We hope you'll join us in October, to understand guidance, streamline submissions, and obtain approval first-time.   #CellandGeneTherapyRegulatoryAffairsSummit 

Gene therapy is a pivotal sector within the biopharma industry, where the demand for new technologies and products is ever-present. Despite its promise, there remains bottlenecks due to cost, quality, and speed. The Triangle region is one the preeminent areas for the manufacture and development of these products, and is on the forefront of pushing this science forward. Even with the greater issues effecting this curative and life changing science, this is a segment that needs to be advanced.

Is your employer health plan equipped to handle the cost (but also the life-saving ability!) of emerging cell & gene therapies? If you attended our HUB I HORAN 2024 Strategic Horizons Benefit Summit last week, you heard Mikal Jeffries, CEBS break this down for us.... 💡 While these are in the realm of drug treatments, gene therapies occur on the MEDICAL side of the plan 💡 These are typically one-time treatments developed for very rare conditions 💡 Average cost thus far appears to be in the $1MM - $2MM range What does this mean for your health plan? ➡ If fully-insured: These likely are covered on your plan, but if a claim hits would probably result in a large increase at renewal ➡ If self-funded/ASO: Check with your stop loss vendor to confirm if covered. If not, there are options we can help you evaluate Berkley Accident and Health (a Berkley Company) has developed a great overview of current and near-future state. Click below to read! #employeebenefits #pharmacy #genetherapy HUB International https://lnkd.in/gJ6YANEV

Exciting News for Cell & Gene Therapies! The FDA recently unveiled a new platform technology process to accelerate the approval process for cell and gene therapies. The FDA hopes to bring these transformative treatments to patients more efficiently by unveiling this groundbreaking approach. This process is poised to revolutionize drug development, allowing companies to streamline clinical trials, reduce costs, and expedite access to promising therapies. The implications for patients are significant - faster access to potentially life-saving treatments. The FDA has heard the pleas of patients and researchers for improved pathways to innovative treatments. This platform technology process will serve as a much-anticipated solution, advancing the frontier of medical possibilities. Read the full article online here: https://lnkd.in/dhGicHjz #FDA #cellandgenetherapy #drugdevelopment #RegulatoryUpdate #FightCancer #Oncology #MakeADifference #Pharma #Biotech

2024 might just be the year for multiple gene therapy breakthroughs! Did you know that 18% of healthcare industry professionals who took part in a survey by GlobalData, named CGT as the most impactful trend in the #pharmaceutical industry in 2024? Or that oncology is the top area for major CGT developments, with 44% of the CGT market by 2029 followed by immunology and genetic disorders? Up to 17 new gene therapy developments can be approved by European and American regulators in 2024. As I see it, the global #healthcare industry is ready to make a huge leap in the treatment of diseases that seemed uncurable for the longest time. And while there are opponents to gene therapy claiming it may be too dangerous or unethical, we can’t really deny the benefits that GT and gene editing can bring to the table. Which side are you on?