Niloofar Davoodi, PharmD, PhD

𝗧𝗮𝗸𝗲𝗱𝗮 𝗲𝘅𝗶𝘁𝘀 𝗰𝗲𝗹𝗹 𝘁𝗵𝗲𝗿𝗮𝗽𝘆—𝗯𝘂𝘁 𝘁𝗵𝗶𝘀 𝗶𝘀𝗻’𝘁 𝘁𝗵𝗲 𝗲𝗻𝗱 𝗼𝗳 𝘁𝗵𝗲 𝘀𝘁𝗼𝗿𝘆 Takeda just announced they’re ending all ex vivo cell therapy work as part of a strategic portfolio shift. It’s easy to read this as a death knell for the modality. But history tells a different story. Promising modalities don’t die—they hibernate. Builders keep iterating through the quiet periods. Then, when the science catches up, pharma rushes back in. We’ve seen this pattern before:  • 𝗔𝗗𝗖𝘀 — Largely abandoned after early toxicity failures. Revived through better linkers, novel payloads, and smarter patient selection. Now a cornerstone of oncology.  • 𝗥𝗡𝗔 𝘁𝗵𝗲𝗿𝗮𝗽𝗲𝘂𝘁𝗶𝗰𝘀 — Stalled for years due to delivery challenges. Rescued by breakthroughs in siRNA and antisense chemistry. Now foundational to drug discovery.  • 𝗺𝗥𝗡𝗔 — Dismissed as too unstable and impractical. COVID vaccines changed everything overnight. Now every major pharma has an mRNA program. Ex vivo cell therapies might be in their “winter” phase. The challenges—complex manufacturing, hostile tumor microenvironments, limited persistence—are real but not insurmountable. The next wave is already forming: automated manufacturing platforms, multiplex genome editing, novel conditioning regimens, off-the-shelf products, and TME-resistant designs. If you’re building in cell therapy right now, you’re exactly where the winners were during past troughs. When the breakthrough arrives—and it will—pharma will flood back in, just like they always do. The question isn’t 𝘸𝘩𝘦𝘵𝘩𝘦𝘳 cell therapy will rebound. It’s 𝘸𝘩𝘰 𝘸𝘪𝘭𝘭 𝘴𝘵𝘪𝘭𝘭 𝘣𝘦 𝘣𝘶𝘪𝘭𝘥𝘪𝘯𝘨 when it does. https://lnkd.in/eKy6hMpx

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EU Grants Approval to Zemcelpro, a Cord Blood–Derived Stem Cell Therapy for Blood Cancer Patients - https://lnkd.in/ggEfsTXJ In August 2025, the European Commission (EC) granted conditional marketing authorization (CMA) to Cordex Biologics, a subsidiary of ExCellThera, for Zemcelpro® (UM171 Cell Therapy), a personalized stem cell therapy derived from a single cord blood unit for blood cancer patients who lack access to a suitable donor for a traditional transplant. ExCellThera announced the news on August 27, 2025. Clinicians emphasize the therapy’s transformative role. Dr. Fabio Ciceri, Professor of Hematology at Vita-Salute San Raffaele University in Milan, explained: “The curative potential of allogeneic stem cell transplantation is still limited by access to a suitable donor. UM171 Cell Therapy offers an option timely available for patients in need.” David Millette, CEO of ExCellThera and Cordex Biologics, framed it as a turning point: “As the first and only cell therapy approved for patients without access to suitable donor cells, Zemcelpro offers a vital new stem cell transplant option—and renewed hope—for those who have been left without one.” Read more at: https://lnkd.in/ggEfsTXJ #cordblood #stemcell #celltherapy

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𝗕𝗥𝗘𝗔𝗞𝗜𝗡𝗚 𝗡𝗘𝗪𝗦: Two approvals for MASH in 5 days 👇 Over the past week, Madrigal Pharmaceuticals and Novo Nordisk have both achieved landmark approvals in liver disease: ✅ Rezdiffra™ (resmetirom) – Madrigal The European Commission last night granted conditional approval for Rezdiffra in adults with noncirrhotic MASH and moderate to advanced liver fibrosis (F2–F3). It becomes the first and only approved MASH therapy in Europe. ✅ Wegovy® (semaglutide 2.4 mg) – Novo Nordisk The U.S. FDA has granted accelerated approval for Wegovy in adults with MASH and F2–F3 fibrosis. This marks the first GLP-1 agonist approved for MASH, adding to Wegovy’s expanding portfolio beyond obesity. 𝗞𝗲𝘆 𝗛𝗶𝗴𝗵𝗹𝗶𝗴𝗵𝘁𝘀: 🧬 Rezdiffra (Madrigal) - First-in-class THR-β agonist, once-daily oral therapy - MAESTRO-NASH Phase 3 trial: achieved both fibrosis reduction & MASH resolution - No biopsy required in EU → a precedent-setting regulatory decision - First European launch planned in Germany, Q4 2025 💉 Wegovy (Novo Nordisk) - GLP-1 agonist already approved for obesity & CV risk reduction - ESSENCE Phase 3 trial: 63% achieved steatohepatitis resolution, 37% fibrosis improvement 👏 Congratulations to both Madrigal and Novo Nordisk - a transformative week for patients. #MASH #biotech #liverdisease #pharma #metabolic

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In the biotech world, the early phase determines success or failure But the figures are alarming: 2023 biotech start-ups in Germany received just 203 million euros in early-stage funding. This is the lowest figure in the last six years - and well below the average of 325 million euros. → 24 er Zahlen For comparison: In the USA, several billion US dollars are invested in biotech start-ups in the seed and Series A phase every year. In 2023 alone, the volume was over 14 billion US dollars. What does that mean in concrete terms? Many start-ups do not fail because of the science. They fail because they run out of money before they can develop their product to market maturity. Early-stage financing is the bottleneck. Because this is precisely where it is decided whether we promote innovation in Europe OR whether it migrates. ➡️ What do I think is needed? Specialized early-stage funds for biotech. The biotech sector has different dynamics to traditional tech start-ups. Research takes longer, is more capital-intensive and riskier. Investors who understand these mechanisms are needed. Public co-investment programs must be a must-have. The state must act as an anchor investor to mobilize private capital. Tax incentives for early-stage investors are also essential. Those who invest in high-risk innovations should be rewarded with tax incentives. It is important to note that early-stage financing is not a subsidy. It is the prerequisite for turning cutting-edge European research into commercial success. If we do not close this funding gap, we in Europe will have to continue to watch our innovations grow in the USA.

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I’m energized by the compelling data emerging around BioNTech SE BNT113 in head and neck cancer treatment. Coupled with recent investor conversations, it’s clear there’s real momentum behind the shift toward in vivo immunotherapy. By programming the immune system directly within the body, this approach promises to streamline production and logistics—potentially delivering transformative care at a dramatically lower cost than traditional cell and gene therapies. To put things in perspective: developing a typical cell or gene therapy today can cost upwards of US $1.9 billion from R&D through clinical trials. And that’s just R&D, Cost of goods/manufacturing alone for a gene therapy can be between $500,000 and $1 million. In contrast, true in vivo approaches (which bypass ex-vivo cell manipulation and personalized manufacturing) could dramatically reduce costs and broaden accessibility. BNT113 exemplifies this potential—a solution that could be cheaper, scalable, and faster to deploy. I’m excited to see how this innovation reshapes the therapeutic landscape—and thrilled to be part of this conversation in making immunotherapy more efficient and accessible. References: https://lnkd.in/exCJK4t8 https://www.genengnews.com

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Significant regulatory shift in UK that may impact 𝗖𝗔𝗥-𝗧 delivery: At-patient manufacturing!   With summer holidays and other industry happening, this news may have passed you by (it did me!): As of 23rd July 2025, the MHRA (the UK regulator) legally allows flexible, innovative manufacturing approaches such as Point-of-Care and decentralised/distributed modular manufacturing for ATMPs including CAR-Ts.   𝗪𝗵𝗮𝘁’𝘀 𝘁𝗵𝗲 𝗯𝗮𝗰𝗸𝗴𝗿𝗼𝘂𝗻𝗱 𝗳𝗼𝗿 𝘁𝗵𝗶𝘀: 🔹 MHRA innovation office has been receiving enquiries around this kind of manufacturing since 2014 – but no regulatory framework was in place to allow for PoC or modular manufacturing. 🔹 As technology advances – particularly ATMPs – new manufacturing modalities are required that can take place in hospitals, clinics and even the patients own home! 💭 𝗜𝗺𝗮𝗴𝗶𝗻𝗲 𝘁𝗵𝗮𝘁, 𝗵𝗮𝘃𝗶𝗻𝗴 𝗮𝗻 𝗮𝘂𝘁𝗼𝗹𝗼𝗴𝗼𝘂𝘀 𝗖𝗔𝗥-𝗧 𝗺𝗮𝗻𝘂𝗳𝗮𝗰𝘁𝘂𝗿𝗲𝗱 𝗮𝗻𝗱 𝗱𝗲𝗹𝗶𝘃𝗲𝗿𝗲𝗱 𝘄𝗶𝘁𝗵𝗶𝗻 𝘆𝗼𝘂𝗿 𝗼𝘄𝗻 𝗵𝗼𝗺𝗲. 𝗛𝗼𝘄 𝘄𝗶𝗹𝗹 𝗶𝘁 𝘄𝗼𝗿𝗸? 🔹  The technical solutions are still in development. Notably, Spain has made some great progress on establishing localised CAR-T production centres in Barcelona and Navarra. 🔹 A centralised “Hub” control site will be responsible for the decentralised “Spoke” sites; being responsible for documentation, quality, release and inspections. 🔹 A Decentralised Manufacturing Master File (DM MF) is required to be managed by the control site with annual reporting to MHRA 🔹 GMP and Pharmacovigilance requirements are maintained and must be have appropriate management plans prior to approval   𝗜𝗻 𝘀𝘂𝗺𝗺𝗮𝗿𝘆: This legislation marks a significant regulatory shift, accommodating disruptive innovation in medicine manufacture and delivery. MHRA has adopted a flexible, guidance-led approach supported by stakeholder feedback, with a strong emphasis on collaboration and quality assurance. Links to the MHRA webinar and the guidance document in the comments 👇 𝗧𝗵𝗶𝘀 𝗵𝗮𝘀 𝗯𝗲𝗲𝗻 𝗵𝗮𝗶𝗹𝗲𝗱 𝗮𝘀 𝗮 𝗳𝗶𝗿𝘀𝘁 𝗮𝗺𝗼𝗻𝗴 𝗿𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘀 – but do you think this puts the UK in a strong position for systems developers?  💬 As always, let me know your thoughts in the comments below.

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