Cordula M Wolf

Despite strong foundational research, Germany faces challenges in integrating cell and gene therapies (CGT) into medical care, risking falling behind internationally. A national CGT strategy was presented yesterday.   Here’s how the strategy differs from the CGT strategy in the US and UK:   𝐂𝐞𝐧𝐭𝐫𝐚𝐥𝐢𝐳𝐚𝐭𝐢𝐨𝐧 𝐚𝐭 𝐏𝐚𝐮𝐥-𝐄𝐡𝐫𝐥𝐢𝐜𝐡-𝐈𝐧𝐬𝐭𝐢𝐭𝐮𝐭 (𝐏𝐄𝐈): Germany plans to centralize regulatory processes for GCT at the Paul-Ehrlich-Institut to streamline the regulatory environment. This involves defragmenting the regulatory landscape and introducing "sandbox" procedures as innovation spaces. ​In the US, multiple agencies such as the FDA and NIH handle different aspects of GCT regulation and development, while the UK has the MHRA and other bodies involved in the regulatory process. 𝐆𝐨𝐯𝐞𝐫𝐧𝐚𝐧𝐜𝐞 𝐚𝐧𝐝 𝐒𝐭𝐚𝐤𝐞𝐡𝐨𝐥𝐝𝐞𝐫 𝐈𝐧𝐯𝐨𝐥𝐯𝐞𝐦𝐞𝐧𝐭: Germany emphasizes a "bottom-up" approach involving diverse stakeholders directly in the governance structure. This includes establishing a governance structure with the participation of significant stakeholders and developing a GCT network map and annual progress reports​. This differs from the US and UK, where strategies tend to have more top-down, centralized approaches often led by major government agencies like the FDA in the US or the MHRA in the UK. 𝐅𝐢𝐧𝐚𝐧𝐜𝐢𝐚𝐥 𝐚𝐧𝐝 𝐒𝐭𝐫𝐮𝐜𝐭𝐮𝐫𝐚𝐥 𝐒𝐮𝐩𝐩𝐨𝐫𝐭 𝐟𝐨𝐫 𝐆𝐌𝐏: Germany’s strategy includes specific measures for financial and structural support in GMP production, such as establishing a central GCT-GMP committee and ensuring sufficient financial resources for GMP infrastructures​. This level of detailed infrastructure planning and financial commitment is not as prominently featured in the US and UK. 𝐄𝐦𝐩𝐡𝐚𝐬𝐢𝐬 𝐨𝐧 𝐏𝐨𝐥𝐢𝐭𝐢𝐜𝐚𝐥 𝐄𝐧𝐠𝐚𝐠𝐞𝐦𝐞𝐧𝐭: Germany’s approach includes continuous political engagement and annual progress reporting to ensure political support and adaptation of policies. This structured political integration is more pronounced than in the US and UK, where political support is sought but not as systematically integrated​. 𝐈𝐧𝐭𝐞𝐠𝐫𝐚𝐭𝐢𝐨𝐧 𝐨𝐟 𝐇𝐮𝐦𝐚𝐧𝐢𝐭𝐢𝐞𝐬 𝐚𝐧𝐝 𝐒𝐨𝐜𝐢𝐚𝐥 𝐒𝐜𝐢𝐞𝐧𝐜𝐞𝐬: The strategy highlights the importance of maintaining an open discourse involving humanities and social sciences, ensuring that ethical, legal, and social aspects (ELSA) are considered in GCT development​. This broader societal integration is less emphasized in the US and UK strategies, which focus more on the scientific and regulatory aspects. 𝐌𝐲 𝐭𝐡𝐨𝐮𝐠𝐡𝐭𝐬: I welcome the centralization at PEI. This offers the potential for rapid progress. I have mixed feelings about the other points, as they have the potential to slow things down. What's your thoughts on this?

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What can be learned from preclinical experiments? And what cannot be learned? What do preclinical researchers need to know about clinical applications? What do clinicians need to learn from preclinical researchers? Prof. Dr. Ken Herrmann (Universitätsmedizin Essen) and Prof. Dr. Katharina Lückerath (University Hospital Essen) will answer in "Theranostics: From Mice to Men and Back, a MOLECUBES Webinar" on Tuesday, 18 June 2024, 16:00 CEST (10:00 EDT). Register now: https://lnkd.in/dJSQ9CnE MOLECUBES NV #Theranostics #PreclinicalImaging #Imaging

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At #ESMOBreast24, we are happy to share our #LUZERNtrial The study focuses on ER+/HER2-, locally advanced, or metastatic breast cancer patients who harbor either germline BRCA (gBRCA) mutations or wild-type BRCA with homologous recombination deficiency (HRD). Patients were treated with Niraparib (PARP inhibitor) in combination with aromatase inhibitors to evaluate the efficacy and safety of this strategy (The LUZERN Strategy). 220-P <<Niraparib Plus Aromatase Inhibitors for Luminallike(HER2-,ER+) and gBRCA or HDR+ Metastatic Breast Cancer (LUZERN)>> Presented by Daniel Alcalá López PhD. Download the poster here: https://lnkd.in/dSRSfcMR #ESMObreast24

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Quantro and Boehringer Ingelheim Hit Milestone in #Cancer Treatment Development QUANTRO Therapeutics GmbH, which focuses on transcription factors targeting cancer treatments, announced that it has reached a key milestone in its joint R&D program with Boehringer Ingelheim. In successfully applying Quantro’s proprietary Quantroseq Transcriptional Fingerprint technology, all pre-set goals for technical proof-of-concept, cross-validation of HTS #screening technology, and identification of high-quality hits for a previously undruggable transcription factor were fully accomplished. Cancer, a leading global cause of death, often has limited treatment options. Transcription factors, key gene expression regulators often disrupted in cancer, are promising therapeutic targets. Despite being hard to target with drugs, the 2022 joint R&D program aims to develop innovative, first-in-class cancer treatments targeting these factors. Read more on #CMI online: https://lnkd.in/eVKrqmhv

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𝐑𝐨𝐜𝐡𝐞 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐬 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐞 𝐏𝐡𝐚𝐬𝐞 𝐈 𝐑𝐞𝐬𝐮𝐥𝐭𝐬 𝐟𝐨𝐫 𝐂𝐓-𝟑𝟖𝟖 𝐢𝐧 𝐎𝐛𝐞𝐬𝐢𝐭𝐲 𝐚𝐧𝐝 𝐓𝐲𝐩𝐞 𝟐 𝐃𝐢𝐚𝐛𝐞𝐭𝐞𝐬 Roche (SIX: RO, ROG; OTCQX: RHHBY) Roche announced positive results from the Phase I trial of CT-388, a dual GLP-1/GIP receptor agonist for obesity and type 2 diabetes. A once-weekly injection of CT-388 over 24 weeks resulted in significant weight loss in obese adults compared to placebo. The mean placebo-adjusted weight loss was 18.8% (p < 0.001). At week 24, 100% of CT-388 treated participants lost >5% of their weight, 85% lost >10%, 70% lost >15%, and 45% lost >20%. The treatment was well tolerated, with mild to moderate gastrointestinal-related adverse events. “We are very pleased with the significant and clinically meaningful weight loss seen with CT-388,” said Levi Garraway, M.D., Ph.D. Levi Garraway, Roche’s Chief Medical Officer. “These results are highly encouraging for further development of CT-388 for obesity and type 2 diabetes, highlighting its potential as a best-in-class therapy.” Obesity, affecting over four billion people globally, poses significant health challenges, including type 2 diabetes, cardiovascular diseases, and chronic kidney disease. CT-388, an incretin-based medicine, aims to regulate blood sugar and reduce appetite by activating GLP-1 and GIP receptors. An additional cohort from the Phase I trial will evaluate CT-388 in obese patients with type 2 diabetes over 12 weeks, with results expected in the second half of 2024. #ClinicalTrials #Obesity #Type2Diabetes #Biotechnology #Pharmaceuticals #MedicalResearch #GLP1 #GIPReceptor #IncretinTherapy #HealthcareInnovation #WeightLoss #DiabetesManagement

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𝗣𝗿𝗲𝗱𝗶𝗰𝘁𝗶𝗼𝗻 𝗮𝗻𝗱 𝗣𝗿𝗲𝘃𝗲𝗻𝘁𝗶𝗼𝗻 𝗮𝗿𝗲 𝗞𝗲𝘆 𝗳𝗼𝗿 𝗖𝗼𝘂𝗻𝘁𝗲𝗿𝗮𝗰𝘁𝗶𝗻𝗴 𝗧𝘆𝗽𝗲 𝟭 𝗗𝗶𝗮𝗯𝗲𝘁𝗲𝘀 🎙#Interview with 𝗣𝗿𝗼𝗳. 𝗔𝗻𝗲𝘁𝘁𝗲-𝗚𝗮𝗯𝗿𝗶𝗲𝗹𝗲 𝗭𝗶𝗲𝗴𝗹𝗲𝗿 about her contribution to novel therapies for type 1 diabetes. Our #type1diabetes expert and Director of the Institute of Diabetes Research at #HelmholtzMunich is driven to prevent the development of this chronic disease.   She says: "𝘞𝘪𝘵𝘩 𝘵𝘩𝘦 𝘩𝘦𝘭𝘱 𝘰𝘧 𝘵𝘺𝘱𝘦 1 𝘥𝘪𝘢𝘣𝘦𝘵𝘦𝘴 𝘴𝘤𝘳𝘦𝘦𝘯𝘪𝘯𝘨 𝘢𝘯𝘥 𝘱𝘳𝘦𝘷𝘦𝘯𝘵𝘪𝘰𝘯 𝘴𝘵𝘶𝘥𝘪𝘦𝘴, 𝘸𝘦 𝘸𝘢𝘯𝘵 𝘵𝘰 𝘴𝘵𝘰𝘱 𝘰𝘳 𝘥𝘦𝘭𝘢𝘺 𝘵𝘩𝘦 𝘤𝘭𝘪𝘯𝘪𝘤𝘢𝘭 𝘮𝘢𝘯𝘪𝘧𝘦𝘴𝘵𝘢𝘵𝘪𝘰𝘯 𝘰𝘧 𝘵𝘺𝘱𝘦 1 𝘥𝘪𝘢𝘣𝘦𝘵𝘦𝘴 𝘪𝘯 𝘤𝘩𝘪𝘭𝘥𝘳𝘦𝘯. 𝘖𝘶𝘳 𝘷𝘪𝘴𝘪𝘰𝘯 𝘪𝘴 𝘢 𝘸𝘰𝘳𝘭𝘥 𝘸𝘪𝘵𝘩𝘰𝘶𝘵 𝘵𝘺𝘱𝘦 1 𝘥𝘪𝘢𝘣𝘦𝘵𝘦𝘴."   👉Read the full interview: https://lnkd.in/d2qkVfHv   👇Get a glimpse in the video! 👇   #AWordWithout1 #AWW1 #GPPAD #diabetes #T1D #prevention #diagnosis #therapy #research

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CHANGE4RARE 2025 AGENDA PUBLISHED The #change4RARE 2025 agenda, developed in collaboration with key rare disease experts, proposes concrete measures in the political and governmental arenas to improve conditions for people with rare diseases. The 4 core messages included in the 2025 Agenda are summarized in the Pharma Fakten article below: 1.     Strengthen patient participation 2.     Collect and use data systematically 3.     Invest in infrastructure 4.     Ensure market access for orphan drugs   You can read and download the complete Agenda 2025 here: https://lnkd.in/enfUdav7

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The three-year evolution of Germany’s Digital Therapeutics reimbursement program and its path forward - npj Digital Medicine Germany's Digital Therapeutics Reimbursement Program Evolution #Introduction Germany has made significant progress in its Digital Therapeutics reimbursement program over the past three years. #Initial Challenges Initially, Germany faced challenges in implementing a reimbursement program for Digital Therapeutics due to regulatory and reimbursement barriers. #2019 Milestone In 2019, Germany achieved a milestone by introducing Digital Therapeutics into the national healthcare system. #2020 Progress In 2020, Germany continued to make progress by expanding coverage for Digital Therapeutics and increasing patient access. #2021 Developments In 2021, Germany further developed its reimbursement program by incorporating Digital Therapeutics into disease management programs. #Future Outlook The future of Germany's Digital Therapeutics reimbursement ai.mediformatica.com #digital #health #digitalhealth #application #this #germany #reimbursement #digitaltherapeutics #medicine #healthcare #trend #healthcaresystem #healthit #healthtech #healthcaretechnology @MediFormatica (https://buff.ly/44YkDTx)

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See you at health.tech Munich; 5-6 June 2024 Please DM if you like to meet talking about sequencing, genetic diagnostics, biobanking, external sample storage, lab-on-chip systems. Looking forward to discuss topic such as rare diseases, stratgetic cooperations, investments, clinical trials. #ht24 #biotechnologie #diagnostics

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„Nach dem Spiel ist vor dem Spiel“ (Sepp Herberger) This is not only true for soccer, but also for scientific meetings. After #Bio2024 in San Diego and #EACR in Rotterdam, we are at the Meeting of the Federation of European Neuroscience Societies #FENS2024 in Vienna. The ariadne.ai booth is ready and Tosin Oyewale and I will be there from tomorrow onwards, helping you with all questions regarding image analysis and spatial biology in neuroscience. #neurology #neuroscience #imageanalysis #imageprocessing #spatialtranscriptomics #spatialproteomics #spatialbiology

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Remarkable work by my colleagues Alexander Renner, Dominik Schmiedel and team from Fraunhofer IZI was recently published at the journal Gene Therapy. The publication discusses the development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer in non dividing cells. By achieving high transduction rates of freshly isolated immune cells before expansion, the approach enables a streamlined and cost-effective automated production of off-the-shelf cell therapeutics in less than 1 day, requiring fewer viral particles and less manufacturing steps. This holds the potential to significantly reduce the time and resources required for producing e.g. NK cell therapeutics. https://lnkd.in/dfWN6miH

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𝗕𝗶𝗼𝗡𝗧𝗲𝗰𝗵 𝗱𝗼𝘂𝗯𝗹𝗲𝘀 𝗱𝗼𝘄𝗻 𝗼𝗻 𝗔𝗗𝗖 𝗽𝗮𝗿𝘁𝗻𝗲𝗿 𝗠𝗲𝗱𝗶𝗟𝗶𝗻𝗸 𝗶𝗻 𝗻𝗲𝘄 $𝟭.𝟴𝗕 𝗯𝗶𝗼𝗯𝘂𝗰𝗸𝘀 𝗽𝗮𝗰𝘁 As BioNTech SE’s pipeline tilts back toward oncology, the German biotech is once again tapping a young Chinese partner in a bid for the next generation of the field’s hottest modality. It’s going back to MediLink Therapeutics for antibody-drug conjugates in a new deal that begins with $25 million and promises up to $1.8 billion down the road, plus tiered royalties should any of the ADCs make it to market. For that money, BioNTech snags the exclusive option for the global license to MediLink’s so-called TMALIN ADC platform for “several novel targets,” the companies said over the weekend. https://lnkd.in/d36Z2rXc

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⭐️ Spotlight Series – HUMAIN Award Winners ⭐️ 🎥 In this fireside chat series we give the stage to the five winners of the HUMAIN Award – a joint initiative of InSphero and the Physicians Committee for Responsible Medicine for enabling and supporting researchers advancing non-animal drug discovery. Meet Dr. Ute Wölfle from the University of Freiburg, with whom we discussed her project, titled “Influencing the interaction of fatty liver spheroids and psoriasis keratinocyted by liver- protecting herbal remedies”. ▶️ Watch the interview to learn: ✔️ What inspired Ute’s research and what is the link between MASH and psoriasis ✔️ How non-animal methods will advance her studies on the skin disease ✔️ How will InSphero’s assay-ready 3D InSight Human Liver Microtissues and MPS technology unlock the full potential of the winning project 🔜 Stay tuned for our next interview with Dr. Felicia Gerst from the University of Tübingen Learn more about our reliable and scalable solutions for Liver Disease: https://lnkd.in/dK8ygcQB Jan Lichtenberg Elizabeth Baker Janine McCarthy Lisa Hoelting Madhu Lal Nag, MBS PhD Olivier Frey Frauke Greve Francisco Verdeguer Radina Kostadinova, PhD Jesus Francisco Glaus Garzon Joel Zvick Rositsa Hadzhipetrova Tina Žel Wolfgang Moritz Paul Edgard Clémençon #TheModelofExcellence™ #NAMs #3dinvitro #drugdiscovery #researchgrant #partnership #academia #mash #nash #fibrosis

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Bringing a drug to market and ultimately to patient care can be a daunting undertaking, but consistent steps forward add up to major progress over time. Congrats to our colleagues at SciRhom for the approval of its first clinical trial application! Since its founding, SciRhom has worked closely with Professor Carl Blobel to develop first-in-class humanized antibodies against iRhom2. This recent approval by the Austrian regulatory authorities is another great step forward as part of SciRhom's collaborative efforts with Hospital for Special Surgery to drive better treatment for #autoimmune diseases such as #rheumatoidarthritis. Looking forward to more progress ahead! Jan Poth, Jens Ruhe, Matthias Schneider, Carl Blobel, Jeff Wang, Kevin Lastorino, Zachary Robert Goldberg. #innovation #biotech #therapeutics #pharma #autoimmune #arthritis 

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Yesterday 12 June, the National Strategy for Gene and Cell Therapies (GCT) was published in Berlin and presented at an official event to Bettina Stark-Watzinger, Federal Minister of Education and Research. SKC's founder and managing director, Univ.-Prof. Dr. Matthias P. Schönermark, was one of the 150 experts of the working group for a national strategy for GCT and contributed significantly to the development of the strategy. 💬 "It was a great pleasure and honor to be able to participate in Working Group VII, which dealt with improving market access for gene and cell therapies. We developed concrete ideas on how market access for ATMPs in Germany can be directly facilitated and made internationally competitive. Let us hope that politicians will now react in a targeted manner." (Prof. Schönermark) The national strategy for GCT strives for a holistic concept for the location Germany, which covers all parts of the value chain from basic research through to the provision of medical care. The primary goal is to ensure the well-being of patients, who are offered new perspectives through GCT focusing on serious diseases for which there are no effective treatment options available. The strategy is focused on therapeutic approaches with advanced therapy medicinal products (ATMPs), therapeutic approaches with novel biological products (e.g. mRNA) and other approaches of this kind in the context of gene- and cell-based therapies. It comprises eight fields of action with strategic goals and concrete proposals for measures including the areas of 1️⃣ networking and stakeholder support, 2️⃣ training and strengthening expertise, 3️⃣ technology transfer, 4️⃣ standards and regulatory frameworks, 5️⃣ expansion of quality and capacities in the area of good manufacturing practice production, 6️⃣ research and development, 7️⃣ market authorisation and transition to supply as well as 8️⃣ interactions with society. The measures are now to be implemented in collaboration with all stakeholders and will be subsidised by the federal and state governments over the next few years. As Market Access Special Forces, SKC has already been able to support the German market access of 8 ATMPs in recent years. We welcome the proposals of the national strategy for gene and cell therapies and will continue to contribute to ensuring that patients with diseases for which there are no or only limited treatment options have access to effective treatment.

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Roche's Alecensa Approved by FDA for Early-Stage ALK-Positive NSCLC The U.S. Food and Drug Administration (FDA) has approved Roche's Alecensa (alectinib) for the adjuvant treatment of patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) following tumor resection. This approval was based on the positive results from the Phase 3 ALINA study, which showed that Alecensa reduced the risk of disease recurrence or death by 76% compared to platinum-based chemotherapy. The FDA's decision marks a significant advancement in the treatment of early-stage ALK-positive NSCLC, providing patients with a targeted therapy option that can significantly improve their chances of a cure. Roche chief medical officer and global product development head Levi Garraway said: “With an unprecedented 76% reduction in the risk of disease recurrence or death versus chemotherapy, Alecensa significantly improves upon the standard of care for people with early-stage ALK-positive lung cancer. “At Roche, our goal is to give patients the best chance of cure by bringing effective, targeted treatments to early-stage disease before their cancer has spread. This approval brings us one step closer to achieving that mission.” This approval was facilitated through the Project Orbis initiative, enabling simultaneous review and submission of the application across international regulatory agencies. https://lnkd.in/e7zh8WJY Be sure to follow Practical Patient Care on LinkedIn for more industry news and insights. #PracticalPatientCare #LungCancer #ALKPositiveNSCLC #TargetedTherapy #FDAApproval

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Study by Rocio Sotillo and team published in Drug Resistance Updates unveils #NovelApproach to combat #Resistance in non-small cell lung cancer (#NSCLC), revealing the potential of #CombinedTherapy to delay resistance development. In NSCLC, a genetic anomaly known as ALK fusion drives cancer growth by over-activating the ALK protein. While ALK inhibitors initially show promise in halting tumor progression, cancer cells quickly develop resistance. This study demonstrates that combining ALK and SRC inhibitors enhances cancer cell response and delays resistance development. Congratulations to Alberto Diaz Jimenez, Maria Ferreira Ramos, Bárbara Helm, Sara Chocarro, Dario Lucas Frey, Shubham Agrawal, Kalman Somogyi, Ursula Klingmüller, Junyan Lu and Rocio Sotillo. DOI: 10.1016/j.drup.2024.101081 https://lnkd.in/dysi2KDk #CancerResearch #LungCancer #DrugResistance #CombinationTherapy #ResearchBreakthrough /cmp

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+++ 𝗤𝟭 𝗿𝗲𝘀𝘂𝗹𝘁𝘀 𝟮𝟬𝟮𝟰 𝗮𝗻𝗱 𝗰𝗼𝗿𝗽𝗼𝗿𝗮𝘁𝗲 𝘂𝗽𝗱𝗮𝘁𝗲 +++ We started the year with good progress across our oncology pipeline and our commercialization activities. Some key events: 🔬 𝗢𝗻𝗰𝗼𝗹𝗼𝗴𝘆: We and our collaboration partner Duality Biologics dosed the first patient in a Phase 3 clinical trial with our #ADC candidate BNT323/DB-1303 in breast cancer patients, advancing towards BioNTech’s goal of ten or more potentially registrational trials running by the end of 2024. 🩹 𝗖𝗢𝗩𝗜𝗗-𝟭𝟵: We are focusing on preparing for variant-strain adaptation of our COVID-19 vaccine to be ready for a commercial launch ahead of the upcoming 2024/2025 vaccination season, pending approvals. 🌎 𝗖𝗼𝗺𝗺𝗲𝗿𝗰𝗶𝗮𝗹 𝗿𝗲𝗮𝗱𝗶𝗻𝗲𝘀𝘀: We appointed our General Manager Fatma Ocak for the U.S. in line with our commercial preparations to drive our first expected global #oncology product launch. 𝗟𝗶𝗻𝗸 𝘁𝗼 𝗣𝗥 𝗮𝗻𝗱 𝗳𝗼𝗿𝘄𝗮𝗿𝗱-𝗹𝗼𝗼𝗸𝗶𝗻𝗴 𝘀𝘁𝗮𝘁𝗲𝗺𝗲𝗻𝘁𝘀: https://lnkd.in/ePDQiHbH  

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