UCB’s Post

The gene therapy industry is at a crossroads with the upcoming EU Joint Clinical Assessment (JCA). While it aims to streamline clinical assessments at a European level, a new article in STAT highlights how gene therapy developers are concerned that the evidence requirements for ATMPs in the draft guidelines are not fit for purpose. https://lnkd.in/eJc88bct What impact could this have? Paolo Morgese, ARM's Vice President of Public Affairs, Europe, is quoted saying, “We fear that this will have a negative impact on European competitiveness, so attracting developers, attracting investment, attracting research — it will be more difficult in Europe in years to come if this approach is not changed.” ARM is engaging with the JCA Coordination Group and other key stakeholders to ensure the process accommodates this transformative medicine. #Genetherapy #Europe

🌍🔬 Promising Developments in Cell and Gene Therapy in South and Central America - June 2024 Insights from the recently published regulatory Watchdog report: "A Roadmap for Accelerating Access to Life-Saving Therapies in South and Central America", from the ISCT, International Society for Cell & Gene Therapy. The report includes highlights from the recent IMPACT-ISCT Joint Session held in Santiago, underscoring the critical need for accessible treatments like #CAR-T cells. 🔍 Key highlights: . 96 CGT approvals worldwide, yet none accessible in many countries in the region including #Chile. IMPACT center aims to bridge this gap. . Brazil leads with 6 ATMP approvals, setting a regulatory precedent. . Strategic partnerships with regulatory bodies and Health advocacy groups such as Pro Salud Chile to ensure robust frameworks for therapy access. https://lnkd.in/dsXncmTS #CellTherapy #GeneTherapy #CAR-T #MedicalInnovation #RegulatoryFramework #HealthcareInnovation #IMPACTCenter #ISCT #GlobalHealthcare #ResearchAndDevelopment Maroun khoury

Cell and gene therapy in the Asia-Pacific region https://lnkd.in/etktvGiz Rethinking ecosystems to enable access. In this update, we bring you the key insights from our latest webinar on access for cell and gene therapies in the Asia-Pacific (APAC) region featuring a discussion between Jose Manuel Garnica, Independent Thought-Leader and Jeff Weisel, Senior Strategic Advisor at Avalere Health. This summary of the discussion includes insights for overcoming the challenges of affordability and navigating APAC healthcare systems, especially in resource-constrained markets. We also hear why public-private partnerships and policy-shaping are key in building a future with optimal access to transformative cell and gene therapies in the region. https://lnkd.in/etktvGiz #MarketAccess #HEOR #WeAreAvalereHealth

📢 CEO Reflects on Gene Therapy's Future 🔍 Frédéric Revah, CEO of Généthon, reflects on the past, present, and future of the organization in an interview. 🧬 Généthon was created in 1990 with a focus on gene therapy research for rare genetic diseases. 💡 Revah discusses the challenges of financing development for rare diseases and the need for equity in distribution. 🏭 Généthon is working on improving bioprocessing and reducing the cost of production for gene therapies. 🤝 The organization collaborates with partners in various fields to advance their research. ⚙️ Exciting developments include producing AAV in plants and using artificial intelligence to optimize bioreactors. ❌ The industry faces challenges such as improving the full-to-empty ratio of virus capsids. ❗️ Revah comments on the Pfizer incident and assures that Généthon is closely monitoring safety in their own clinical trial. #BioprocessUpdates #ASGCT2024 #FrédéricRevah #CEO #Généthon #genetherapy #raregeneticdiseases #equity #bioprocessing #costreduction #collaboration #AAV #artificialintelligence #bioreactors #viruscapsids #safety ▷ Read the full article here: 📎 https://lnkd.in/dnp5_PTk

Why is there uncertainty about the trajectory of the ever-growing cell and gene therapy sector? As IQVIA Institute’s latest report on the topic finds the combination of the health care systems’ infrastructure and the complexities of the patient journey pose significant barriers to the future success and sustainability of these treatments. Even the basic task of following the patient is a challenge in long-term follow-up studies, increasing the risk of patient loss to follow-up. Since it is unlikely that patients will be able or want to return to the treatment center for monitoring activities, the patient journey, and where patients engage will be important. For example, follow-up activities would be better integrated into their standard of care at the patient’s local primary care provider or specialist. To learn more about the elements to consider in long-term follow-up study design, go to page 51 of Strengthening Pathways for Cell and Gene Therapies: Current State and Future Scenarios. The report is available for download: https://bit.ly/49CxkUK #IQVIA #PharmaDeals #celltherapy #genetherapy #globalhealth #healthequity

Why is there uncertainty about the trajectory of the ever-growing cell and gene therapy sector? As IQVIA Institute’s latest report on the topic finds the combination of the health care systems’ infrastructure and the complexities of the patient journey pose significant barriers to the future success and sustainability of these treatments. Even the basic task of following the patient is a challenge in long-term follow-up studies, increasing the risk of patient loss to follow-up. Since it is unlikely that patients will be able or want to return to the treatment center for monitoring activities, the patient journey, and where patients engage will be important. For example, follow-up activities would be better integrated into their standard of care at the patient’s local primary care provider or specialist. To learn more about the elements to consider in long-term follow-up study design, go to page 51 of Strengthening Pathways for Cell and Gene Therapies: Current State and Future Scenarios. The report is available for download: https://bit.ly/499sL3P #celltherapy #genetherapy #globalhealth #healthequity

Significant uncertainty still exists about the future trajectory and sustainability of the cell and gene therapy sector, with a range of future scenarios possible across eight key indicators which will define the future through 2035 and beyond. Read the latest report from IQVIA Institute for Human Data Science:

China dominates CAR-T therapies. 55% of CAR-T trials are happening in China. How did this happen? Government initiatives. As part of Healthy China 2030 and other programs, China turned its focus towards advanced therapies. For every CAR-T developed by US companies, there are 1.5 times more CAR-T therapies being investigated by Chinese companies. The number of newly identified CAR-T therapies from Chinese developers has doubled every year since 2014. Lots happening there. We should keep our eyes on China. PS: What other country is innovating in cell and gene therapy? Source: GlobalData Healthcare #ATMP #CGT #CART

🧬 In 2023, 500+ gene therapy trials were conducted in North America alone, promising wider patient access and breakthrough treatments. New developments in manufacturing and supply chains are driving down costs and amplifying accessibility to these groundbreaking treatments. With 10 FDA-approved therapies available today, the market is beginning to convert the potential of science into commercial products that benefit patients.     At QHP, we're closely monitoring the trends shaping the future of gene editing therapies. We anticipate innovative advancements in manufacturing and supply chain processes to help lower costs and improve access to treatments.     Gene editing marks a new era in medicine! Watch the video below for your industry check-up: #lifesciences #privateequity #celltherapy #geneediting 

There exists a notable disparity between regions in terms of the population versus the number of gene therapy trials conducted. This striking contrast calls for the establishment of an advocacy group dedicated to promoting research on innovative medicines, bridging the gap between scientific communities, patients, and policymakers. Understanding these disparities is essential as we strive to ensure equitable access to cutting-edge therapies worldwide, as highlighted in recent research (Cornetta et al., 2022). We invite you to join IAAP-CGT group in this critical mission to drive change and make a difference. IAAP-CGT has a mission to unite professionals in the field of Cell and Gene Therapy (CGT) from India and the USA. Our primary goal is to foster collaboration, share knowledge, and advance research and development in the CGT domain.