Tony Cheng-Fu Chang

💡 CELL THERAPY: UPCOMING MILESTONES WE ARE TRACKING Earlier this week I posted an overview of the "state of play" across five cell therapy growth vectors (link in first comment). Today, here are the upcoming milestones over the next ~6 months that we are tracking to monitor the space: 1️⃣ CRISPR Tx CTX112 (allogeneic CD19) basket-trial update in autoimmune — mid-2025   🔸 Why it matters — "Off-the-shelf" CAR-T could validate allogeneic approaches and broaden the autoimmune cell therapy opportunity. Weaker data or safety concerns could indicate an autologous-focused outlook for autoimmune 2️⃣ KYV-101 interim Phase 2 data in myasthenia gravis — 2H 2025   🔸 Why it matters — Durable remission with clean safety could support accelerated approval via RMAT. Mixed results could push out timelines for key cell therapy launch in autoimmune  3️⃣ BlueRock bemdaneprocel first-patient-in, Phase 3 exPDite-2 (Parkinson’s) — H2 2025   🔸 Why it matters — Fast enrollment could indicate physician and patient appetite for surgical iPSC-derived neuron therapy and may signal KOL buy-in; slow enrollment could flag future adoption hurdles despite promising early efficacy  4️⃣ Amtagvi CMC & clinical updates (24-day process progress + NSCLC data) — Q4 2025   🔸 Why it matters — Manufacturing process improvement plus objective responses in lung cancer would support both the supply security and indication expansion story for solid-tumor TIL therapy  5️⃣ CARTITUDE-4 five-year overall-survival read-out at ASH 2025 — Dec 2025   🔸 Why it matters — Indicates whether Carvykti’s survival data can support access / reimbursement in earlier-line multiple myeloma. A strong OS signal could accelerate front-line expansion  6️⃣ Casgevy durability + uptake update — Dec 2025 (ASH 2025 data and 3Q results)   🔸 Why it matters — Three-year follow-up on severe vaso-occlusive crises (sVOC) and transfusion independence will be key measures of long-term benefit. Management’s Q3 revenue commentary will also signal the pace of commercial ramp after slow start  7️⃣ Zimislecel (VX-880) pre-BLA meeting with FDA — early 2026   🔸 Why it matters — FDA alignment could enable the first stem-cell-derived islet therapy to launch in 2026; a request for longer follow-up could postpone launch and open the door for early-stage pipeline competitors  8️⃣ BONUS — Capstan in vivo CAR-T Phase 1 first-in-human data — timing TBD   🔸 Why it matters — Early clinical evidence will be first indication whether AbbVie’s big bet on (non-viral) in vivo CAR-T could pay off. If successful, in vivo approaches could disrupt the current ex vivo paradigm  These milestones will help identify where the cell therapy space is converting scientific potential into success stories. What cell therapy milestones are you watching over the next six months? #CellTherapy #CART #TCR #TIL #Autoimmune #RegenerativeMedicine #GeneTherapy #Biotech #LifeSciences

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Big news for European biotech: the Dutch 🇳🇱 government just released an ambitious vision to position the Netherlands as a global biotech leader by 2040. Three things that caught my attention in this vision: 1) Tools-focused approach. Rather than just funding basic research, they're investing in infrastructure, research facilities, and accessible data platforms. This is smart - tools transform industries, not just ideas. When architects moved from paper to computers, we saw entirely new types of buildings emerge. The same transformation happens when scientists get better tools. 2) The focus on commercialization is refreshing. Too many European innovation initiatives get stuck in the lab. The Dutch plan explicitly targets growth markets like Advanced Therapy Medicinal Products and alternative proteins, with clear pathways to turn research into businesses. 3) They're tackling regulation pragmatically. The vision calls for "regulatory sandboxes" to test innovations safely without killing them with red tape. This balance of innovation and safety is exactly what Europe needs. I've long believed Europe has unique advantages in biotech - world-class universities, deep technical talent, and strong public-private cooperation. But we've lacked the cohesive vision and funding to compete globally. This Dutch initiative could be the template for a broader European biotech renaissance. My experience at Google taught me that infrastructure investments pay exponential dividends. The same is true in biotech - the most valuable innovations aren't flashy new molecules but the tools that let scientists design and test thousands of them rapidly. Europe has a real opportunity to lead here. While the US focuses on foundation models and raw compute, we could focus on practical tools that transform how scientists work. What do you think? Is the Dutch vision ambitious enough? And what would you prioritize to position Europe as a biotech leader?

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Everyone in pharma is racing to launch generative AI tools, even the FDA. However, some things in life take time to flourish… 📈⚕️ As journalist Natalia Mesa outlines in her recent take for BioSpace the FDA’s recent rollout of Elsa, a large language model designed to help with clinical protocol reviews and scientific evaluations, raised some red flags ⚠️. According to Mesa’s reporting, FDA insiders described the implementation as rushed, lacking proper oversight, and prone to incorrect or partially accurate outputs. Some said Elsa wasn’t ready. Others questioned how decisions made with its help could hold up under legal or scientific scrutiny. I don’t know for sure, won’t speculate on FDA’s moves. But I can say one thing clearly: it took a decade for Insilico Medicine, to get to the most advanced and well-validated AI platform in the industry, PharmaAI. Steadily building, testing, and validating…it takes time to grow organically 🌿. For those curious, here is a brief historic note: 🧬 2014 – Founded to bring AI into aging and medicine. 📄 2016–2019 – Published foundational work; GENTRL, their early generative model, created viable molecules in under a month. 🧪 2020–2021 – Launched PharmaAI, combining tools for target discovery (PandaOmics), molecule generation (Chemistry42), and trial prediction (InClinico). 💊 2022 – AI-designed anti-fibrotic drug ISM001-055 entered Phase I trials. 📈 2023–2025 – That same drug, now named Rentosertib, reached Phase II trials—with results published in Nature Medicine in June 2025 confirming safety and initial efficacy in patients. 🚀 Over just a span of several years, PharmaAI platform enabled discovery and design of more than 30 preclinical and clinical drug candidates, and multiple new targets (see image). The lesson here is this: Insilico didn’t skip steps. It published results and AI platform mechanics relentlessly, validated with its own pipeline and client’s projects, and iterated, it took time and patience. Their tools evolved from basic algorithms to a trusted enterprise platform used by major pharma players across the globe… I think Alex Zhavoronkov and Alex Aliper could one day write a book about this roller coaster ride! Meanwhile, I feel like the FDA’s experiment with Elsa shows what happens when there is a push for a quick win in a complex area like medical AI, something Mesa’s reporting hints to… I hope they figure things out fast, the FDA is one of the most important organizations in the world, IMO. We all need them to be doing great, because the health and lives of many-many people depend on that 🙏🩺 Image credit: Insilico Medicine

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👋 Recombinant DNA 🧬, the genetic ‘scissors’ ✂️ called Crispr Cas9 and, more recently, AI 🌐represented breakthrough solutions with massive impact in biotechnology to design proteins, peptides and antibodies for innovative vaccines, crop protection solutions and alternative proteins.   Will the biosensor from Enduro be the next big thing 💪 in bioengineering? That’s what we believe at Supernova Invest!!   👉 Its universal genetic plug-in addresses the fundamental problem of declining cell productivity in biomanufacturing. Enduro’s solution could act as a critical enabler to break the cost barrier of biosolutions for a massive application into the AgriFood systems 🚀🚀.   📢 📢 Enduro has secured a €12M Series A funding led by Supernova Invest with the strong support of NOON Ventures and Sandwater. 👉 Peter Rugbjerg, Christian Munch 👏   #deeptech #transition  #AgriFoodTech   Theis Malmborg, Katherine Mellis, Thomas Videbæk

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FUTURE TRENDS IN CELL AND GENE THERAPY (2026–2030): CRISIS, OPPORTUNITY OR BOTH?/ After a turbulent 2024–25, I kept getting the same question: where does cell & gene therapy go next? This position paper tries to summarize a pragmatic roadmap for founders and investors. Our perspective is informed by recent experience, including our exit from Esobiotec (acquired by AstraZeneca). Thesis: The next five years will be shaped by four forces — in vivo delivery, cost curves (CMC), access policy, and China — with AI powering design, manufacturing, and clinical execution. From 2026 onward, expect fewer but “shocking” wins that reset expectations. What’s changing: ● From hype to hard problems. CAR‑T and gene therapy delivered real cures, now the frontier is solid tumors, larger genes, and scalable delivery. ● In vivo moves center stage. Recent landmark deals signal that editing or programming cells inside the body will define the next platform wave. ● Cost curves bend. Rapid CAR‑T (7–10 days), point‑of‑care manufacturing, and automated QC turn CMC into a core moat. ● Access vs. innovation finds rules. Hospital exemptions and right‑to‑try expansions will force clearer coexistence between academic access and commercial pathways. ● China changes the price and pace. Massive trial volume and lower costs put global pressure on CGT pricing, and open new cross‑border development routes. ● AI becomes non‑optional. From target and capsid design to bioreactor control and trial ops, AI is now a competitive baseline. Investor playbook 1️⃣ Back delivery (in vivo & non‑viral): the platforms that scale beyond bespoke products. 2️⃣ Build CMC + data moats: speed, reliability, and AI‑driven manufacturing as first‑order strategy. 3️⃣ Globalize early, design for access: partner with leading hospitals, plan China/EU routes, and align incentives (outcomes‑based models). Inside the paper you’ll find a 1‑page landscape map (p.1), a deal & signal table (p.9), and concrete takeaways for teams navigating the next cycle. 👉 Download/print the full paper: https://lnkd.in/dqzZX7ZX #celltherapy #genetherapy #biotech #venturecapital #AIinBiotech #CMC

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Thinking about launching a biotech company? In this second in our three part series, we looked at how equity division differed among the investors and CEOs, in two models of launched companies: investors launched model versus entrepreneur launched model. Reach out with questions or comments. Carlo Rizzuto Jigar Raythatha Robert Ang Andras Forgacs Artavazd Arumov Ehsan S. Yazdi, PhD, MPH Dov Shamir, Ph.D. Ilya Strebulaev Inna Khartchenko, MS, MBA Lisa Mueller

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