Eli Lilly Acquires Gene Therapy for Blindness from MeiraGTx

📢 𝗕𝗶𝗴 𝗡𝗲𝘄𝘀 𝗶𝗻 𝗕𝗶𝗼𝘁𝗲𝗰𝗵. 𝗘𝗹𝗶 𝗟𝗶𝗹𝗹𝘆 𝗺𝗮𝗸𝗲𝘀 𝗮 𝘀𝗶𝗴𝗻𝗶𝗳𝗶𝗰𝗮𝗻𝘁 𝘀𝘁𝗿𝗮𝘁𝗲𝗴𝗶𝗰 𝗺𝗼𝘃𝗲, 𝗮𝗰𝗾𝘂𝗶𝗿𝗶𝗻𝗴 𝗠𝗲𝗶𝗿𝗮𝗚𝗧𝘅'𝘀 𝗽𝗿𝗼𝗺𝗶𝘀𝗶𝗻𝗴 𝗿𝗲𝘁𝗶𝗻𝗮𝗹 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝘆. This deal, valued at up to $𝟰𝟳𝟱 𝗺𝗶𝗹𝗹𝗶𝗼𝗻 ($75M upfront + $400M+ in milestones), not only brings a key asset into Lilly's pipeline but also provides access to additional MeiraGTx assets currently under development. This underscores Lilly's expanding commitment to advanced therapeutic modalities. Strategic Implications. This acquisition positions Lilly firmly in the rapidly growing field of in vivo gene therapy, particularly for ocular diseases. This acquisition positions Eli Lilly and Company firmly in the rapidly growing field of 𝗶𝗻 𝘃𝗶𝘃𝗼 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝘆, particularly for ocular diseases. MeiraGTx's therapy, designed to produce therapeutic proteins directly within the eye, represents a cutting-edge approach with the potential for long-term impact on patient care. It’s a clear signal of Lilly's drive to diversify its pipeline with innovative, high-impact assets and leverage broader access to emerging technologies. #GeneTherapy #MergersAndAcquisitions #EliLilly #MeiraGTx #Ophthalmology https://lnkd.in/earn8qZr

A new paper in Cell Reports Medicine reports the preclinical research that led to AviadoBio’s ASPIRE-FTD trial. https://lnkd.in/gbtGtA_a Researchers evaluated the gene therapy AVB-101 across several models, including mice, sheep, and non-human primates, to study its effects and safety. The main disease work was done in Grn-/- mice, which develop many of the same brain changes seen in people with FTD-GRN. Delivering the GRN gene restored progranulin in affected brain regions, improved cell health, and led to better memory and behaviour in the treated mice. In sheep and non-human primates, progranulin levels increased in the cerebrospinal fluid (CSF), and the therapy was shown to be safely delivered and widely expressed in the brain — key steps before moving into clinical testing. These findings provided the foundation for ASPIRE-FTD, a Phase 1/2 study now enrolling people with FTD-GRN to evaluate safety, tolerability, and changes in progranulin levels in the brain and CSF. Two other gene-therapy programs are also underway — PROCLAIM ( Prevail Therapeutics / Eli Lilly and Company) and upliFT-D (Passage Bio) — reflecting growing momentum toward treatments that target the genetic cause of FTD-GRN. For families, this represents steady and meaningful progress: years of careful lab work are now translating into real therapies being tested in people. DM me if you want to chat more!

🧬 Eli Lilly and Company Acquires Rights to MeiraGTx's LCA4 Gene Therapy Lilly has acquired exclusive global rights to MeiraGTx’s AAV-AIPL1 gene therapy for Leber congenital amaurosis type 4 (LCA4). This follows Lilly’s recent move to acquire Adverum Biotechnologies and Ixo-vec for wet AMD, marking its accelerated push into ophthalmology. 🔗 Read more here: https://lnkd.in/dZuJEAzP #GeneTherapy #Ophthalmology #LCA4 #VisionLoss #Acquisition #Innovation #ClinicalTrials #Lilly #MeiraGTx #AMD #Retina

🎉 Exciting milestone in cell & gene therapy! 🎉 The launch of the inaugural Broermann Medical Innovation Award - a €1 million prize recognizing translational research that meaningfully advances patient care. Two pioneers in personalized immune-cell therapies: Carl June and Michel Sadelain whose work on engineered T-cells (#CAR-T cells) has transformed the treatment of leukemias, lymphomas and multiple myeloma. As someone immersed in the world of cell and gene therapies from early discovery through analytical development, technology transfer and manufacturing, this moment resonates deeply. What stands out: 🔬 From bench to bedside - This award emphasizes not just discovery but translation, foundational science plus real-world clinical impact. 💡 Risk, perseverance & vision - Dr. June and Dr. Sadelain weathered scepticism, funding scarcity and technical challenges to deliver a true medical innovation. 🌍 Global impact - The award’s mission is to support breakthroughs that benefit patients worldwide including those facing rare or neglected conditions. 🚀 Encouraging the next wave for those of us in biotech, CDMO, regulatory and translational science roles, this is a strong signal: complex, bold, patient-driven research does get recognition and should continue to thrive. Here’s to the innovators, the risk-takers, the patient-champions may this award inspire the next generation of breakthroughs in immunotherapy, cell therapy and beyond. 🚀 💼 At NEXIS BIOCONSULT with over 15 years of experience supporting cell/gene therapies, my drive remains firm to help turn scientific breakthroughs into therapies that transform lives. #CellTherapy #GeneTherapy #Immunotherapy #CART #TranslationalMedicine #Biotech #LifeSciences #Innovation #PatientFirst #HealthcareImpact #DrugDevelopment #Manufacturing #TechnologyTransfer #CDMO #Biopharma #NextGenTherapies Image credit: Nemes Laszlo/ Getty Images

🧬 The Next Big Debate: Who Will Benefit First from Gene & Cell Therapies? - https://lnkd.in/dcMXf6Aw I recently came across a thought-provoking review titled “Cell & Gene Therapy Accessibility” by Rayne H. Rouce and Matthew H. Porteus, published in Science (2024) and it highlights one of the biggest questions in modern medicine: 👉 As we stand on the brink of curing genetic and rare diseases who actually gets access first? Gene and cell therapies (CGTs) are reshaping healthcare by offering curative potential for conditions once deemed untreatable from spinal muscular atrophy and sickle cell disease to advanced cancers. But as innovation accelerates, accessibility is quickly becoming the next frontier of inequality. 💡 The Promise CGTs can replace, repair, or reprogram defective genes. One treatment can mean a lifetime cure a paradigm shift from chronic management to true healing. 🚧 The Challenge The review points out that availability ≠ access. These therapies often cost hundreds of thousands to millions of dollars per patient, and that’s before considering infrastructure, manufacturing, and follow-up care. Some of the biggest barriers include: 💰 High manufacturing & delivery costs 📜 Outdated reimbursement models 🏥 Limited healthcare infrastructure & regulation 🌍 Equity gaps between countries and patient groups This means that, right now, the first to benefit are typically those in wealthier regions, often treated for rare diseases where healthcare systems can justify high upfront costs. Meanwhile, patients in low- and middle-income countries or those with more common conditions remain on the sidelines. 🌍 What Can Change? The authors suggest several promising paths forward: Decentralized & allogeneic manufacturing to cut costs. Outcome-based reimbursement models pay for results, not just treatment. Global partnerships to ensure equitable access and regulatory alignment. Transparent prioritization defining who gets treated first and why. ⚖️ Why This Debate Matters If CGTs truly redefine medicine, equitable access must be part of the equation. Otherwise, the gap between innovation and inclusion will only widen. It’s a crucial reminder that curing diseases isn’t enough we must cure the system too. 🧠 Your thoughts: If you were designing a global rollout strategy for gene and cell therapies, what would you prioritize first cost reduction, infrastructure, or access policy? #GeneTherapy #CellTherapy #HealthcareInnovation #BiomedicalEngineering #HealthEquity #PrecisionMedicine #TranslationalResearch #Biotech #AccessToCare #FutureOfMedicine

Solid tumors remain one of the most complex frontiers in cell and gene therapy. We’ve been awarded an ARPA-H award to help develop an in vivo CAR T platform targeting gastrointestinal cancers – with the goal of simplifying manufacturing and improving accessibility. The anticipated collaboration brings together leading academic institutions and Danaher companies to explore: 👉 Non-viral, LNP-based delivery for in vivo CRISPR genome editing 👉 Direct engineering of CAR T cells inside the body 👉 A potential 10x reduction in manufacturing costs This work builds on technologies used in the world’s first personalized CRISPR therapy and aims to address key barriers in cell and gene therapy – from targeting solid tumors to scaling production. Read the full story and hear from our CTO Dr. Beate Mueller-Tiemann and Danaher’s CTO for life sciences omics solutions, Dr. Sadik Kassim: https://okt.to/NirReg

Solid tumors remain one of the most complex frontiers in cell and gene therapy. We’ve been awarded an ARPA-H award to help develop an in vivo CAR T platform targeting gastrointestinal cancers – with the goal of simplifying manufacturing and improving accessibility. The anticipated collaboration brings together leading academic institutions and Danaher companies to explore: 👉 Non-viral, LNP-based delivery for in vivo CRISPR genome editing 👉 Direct engineering of CAR T cells inside the body 👉 A potential 10x reduction in manufacturing costs This work builds on technologies used in the world’s first personalized CRISPR therapy and aims to address key barriers in cell and gene therapy – from targeting solid tumors to scaling production. Read the full story and hear from our CTO Dr. Beate Mueller-Tiemann and Danaher’s CTO for life sciences omics solutions, Dr. Sadik Kassim: https://okt.to/ijFpvM

Solid tumors remain one of the most complex frontiers in cell and gene therapy. We’ve been awarded an ARPA-H award to help develop an in vivo CAR T platform targeting gastrointestinal cancers – with the goal of simplifying manufacturing and improving accessibility. The anticipated collaboration brings together leading academic institutions and Danaher companies to explore: 👉 Non-viral, LNP-based delivery for in vivo CRISPR genome editing 👉 Direct engineering of CAR T cells inside the body 👉 A potential 10x reduction in manufacturing costs This work builds on technologies used in the world’s first personalized CRISPR therapy and aims to address key barriers in cell and gene therapy – from targeting solid tumors to scaling production. Read the full story and hear from our CTO Dr. Beate Mueller-Tiemann and Danaher’s CTO for life sciences omics solutions, Dr. Sadik Kassim: https://okt.to/S3JwID

Eli Lilly and Company Secures Gene Therapy Deal for Rare Eye Disease Eli Lilly has taken a major step into the field of ophthalmology and gene therapy by partnering with MeiraGTx. The deal gives Lilly exclusive global rights to MeiraGTx’s experimental gene therapy, AAV‑AIPL1, aimed at treating Leber congenital amaurosis 4 (LCA4), a rare inherited condition causing severe vision loss from birth. This collaboration highlights how big pharma is betting on innovative treatments for rare diseases. Key Highlights: ▪️ Upfront Investment: MeiraGTx receives $75 million upfront and can earn over $400 million in milestones. ▪️ Global Rights: Lilly gains worldwide exclusive rights to develop and commercialize the therapy. ▪️ Transformative Potential: The therapy could provide a one-time treatment for LCA4 patients, improving vision from birth. ▪️ Strategic Move: Strengthens Lilly’s presence in rare eye disorders and gene therapy innovations. Read More: https://lnkd.in/guKtVhKg #GeneTherapy #RareDiseases #Ophthalmology #EliLilly #Biotech #InnovationInHealthcare #VisionRestoration

Solid tumors remain one of the most complex frontiers in cell and gene therapy. We’ve been awarded an ARPA-H award to help develop an in vivo CAR T platform targeting gastrointestinal cancers – with the goal of simplifying manufacturing and improving accessibility. The anticipated collaboration brings together leading academic institutions and Danaher companies to explore: 👉 Non-viral, LNP-based delivery for in vivo CRISPR genome editing 👉 Direct engineering of CAR T cells inside the body 👉 A potential 10x reduction in manufacturing costs This work builds on technologies used in the world’s first personalized CRISPR therapy and aims to address key barriers in cell and gene therapy – from targeting solid tumors to scaling production. Read the full story and hear from our CTO Dr. Beate Mueller-Tiemann and Danaher’s CTO for life sciences omics solutions, Dr. Sadik Kassim: https://okt.to/m2HtiG