Mission Bio Unveils Single-Cell CRISPR T-Cell Data

CREATE Medicines' $122M Series B: Advancing the Future of In Vivo CAR Therapy CREATE Medicines has secured $122M in Series B financing to accelerate its in vivo CAR pipeline, targeting both autoimmune diseases and oncology. This significant investment underscores the growing confidence in RNA-based immune programming as a transformative approach to immunotherapy. Unlike traditional ex vivo CAR-T therapies, CREATE's proprietary mRNA-LNP platform programs immune cells directly inside the body, enabling scalable, repeat-dose, off-the-shelf treatments. With the largest clinical dataset in the field—over 50 patients dosed—CREATE is demonstrating that in vivo CAR therapy can be delivered tolerably and repeatedly, which could fundamentally change how we treat cancer and autoimmune conditions. https://lnkd.in/eCiJ6DCE We're excited to announce that Robert Hofmeister, Chief Scientific Officer at CREATE Medicines, will be delivering a keynote presentation at our upcoming Cell and Gene Therapy International 2026 on Programming Immunity In Vivo: Targeted RNA/LNP Platforms for Precision CAR Engineering. This is your chance to hear directly from the scientific leadership behind this breakthrough platform and explore the latest advancements in in vivo immune programming and precision CAR engineering. If you're attending Cell and Gene Therapy International 2026, this keynote is not to be missed. 📅 Event Details: Cell and Gene Therapy International 2026 🗓️ Date: September 22-25th 2026 📍 Location: Boston, MA 🔗 Learn more and register here: https://lnkd.in/e4n3vHtK Join us to connect with global leaders and innovators shaping the future of cell and gene therapy. #CellTherapy #GeneTherapy #CARTherapy #InVivoCAR #Biotech #Innovation #PrecisionMedicine #LifeSciences #CGTI #InformaConnect

🧬 Gene expression analysis is only as powerful as the platform behind it. Microarrays help researchers measure thousands of genes at once, revealing which genes are active, inactive, or changing under specific biological conditions. But when the goal is reliable insight across hundreds or thousands of samples, precision matters. Our latest application note explores how high-performance microarray development can support: ✅ reproducible gene expression analysis ✅ biomarker discovery and validation ✅ scalable translational research ✅ diagnostics, oncology, infectious disease, and drug discovery applications From surface chemistry and spot uniformity to scalable manufacturing and quality control, SCHOTT® Microarray solutions are engineered to help bring complex biology into clearer focus. Download the application note here 👉 https://lnkd.in/gMkAykub No matter your microarray application, we’ve likely encountered something similar and can help guide your journey from concept to clinical impact. Ask the experts to find out more 👉 https://lnkd.in/gKNVfZJB

Benitec Biopharma Inc. Shares Promising BB-301 Clinical Results at ASGCT 2026 We came across an important clinical update from Benitec Biopharma Inc. that highlights meaningful progress in rare disease treatment. The company announced interim Phase 1b/2a results for its gene therapy candidate BB-301, targeting Oculopharyngeal Muscular Dystrophy (OPMD), a condition with no approved therapies and affecting nearly 15,000 patients globally. The therapy is specifically addressing severe dysphagia, a life-threatening complication impacting 97% of patients. Key highlights from the study include: • 12-month follow-up results from the first 4 Cohort 1 patients • 24-month durability data from the first patient • Early positive signals from Cohort 2 BB-301 stands out as the only clinical-stage therapy currently in development for OPMD-related dysphagia. Built on a novel “Silence and Replace” platform, it combines RNA interference with gene therapy to silence the mutated gene while restoring functional protein expression. The data will be presented at the American Society of Gene and Cell Therapy Annual Meeting 2026, where CEO Jerel A. Banks, M.D., Ph.D. will share insights on long-term safety and durable efficacy; signaling growing confidence in this innovative approach. With Orphan Drug and Fast Track designations from the FDA and EMA, BB-301 is positioning itself as a potential breakthrough in rare genetic disease treatment. We believe advancements like these are redefining the future of gene therapy and bringing new hope to underserved patient populations. Follow us for more real-time healthcare and biotech updates shaping the future. Submit your latest company news/updates/announcements/events, and we’ll feature it on our official LinkedIn page (Towards Healthcare Research & Consulting) free of charge. DM us or Contact us for further process: https://lnkd.in/dNNTvgwa #HealthcareResearch #BiotechNews #GeneTherapy #RareDiseases #ClinicalTrials #BenitecBiopharma #ASGCT2026 #OPMD #DrugDevelopment #Biopharma #InnovationInHealthcare

The FDA approved Otarmeni on April 23. On the surface this looks like a small rare disease approval. Around 50 babies are born each year in the US with OTOF-related congenital hearing loss. Small patient population. Accelerated approval. Regeneron is even offering the therapy for free. But this is not a small approval. Otarmeni is the first gene therapy to restore a neurosensory function to normal levels. Not improve. Not partially correct. Normal. In the CHORD trial, 42% of treated patients achieved normal hearing including the ability to hear whispers. 80% met the primary endpoint. Zero serious adverse events in the entire trial. That last number is the one the industry should sit with. Zero serious adverse events in a gene therapy trial. That has never happened before. For anyone working in gene therapy, CNS, or rare neurological conditions, this approval changes the benchmark conversation. The question for BD teams at Biogen, Roche, Sanofi, and every company with a neurological or sensory gene therapy program is no longer whether in vivo gene therapy can achieve normal function restoration. Regeneron just proved it can. The question is now whether your delivery platform, your promoter specificity, and your vector design can get to the same safety profile. Three things worth tracking from here. Regeneron will need to submit confirmatory data as a condition of the accelerated approval. What that data looks like will determine whether the accelerated pathway becomes a full approval and what the real-world durability looks like beyond the 48-week trial follow-up. The CNPV priority voucher that came with this approval is worth hundreds of millions of dollars on the open market. Watch for Regeneron to either sell it or use it strategically on a future program. And every gene therapy company with a sensory or CNS program just got a new floor for what investors and BD partners will expect from their preclinical and clinical packages. What is your read on what this means for the gene therapy space? #GeneTherapy #Pharma #RareDisease #FDAApproval #LifeSciences #PharmaBD

QIMR Berghofer is proud to announce a new strategic partnership with TFBS Bioscience, Inc. through a Memorandum of Understanding signed at BIO KOREA 2026 this week. The Institute and its subsidiary Q‑Gen Cell Therapeutics aim to work with TFBS Bioscience Inc to enable an integrated, end‑to‑end cell and gene therapy (CGT) platform that connects viral vector manufacturing, cell therapy production and translational research. TFBS Bioscience Inc is a Taiwanese business offering viral vector manufacturing for cell and gene therapy products, and biosafety testing and product release testing for cell and gene therapy products, vaccines and biologic products. This collaboration will strengthen both organisations’ presence across the Asia‑Pacific CGT market, combine TFBS Bioscience Inc’s viral vector expertise with Q‑Gen’s cell therapy and translational capabilities, support global clients from preclinical development through to clinical translation and enable joint Contract Development and Manufacturing Organisations contracts, co‑marketing and long‑term research partnerships We look forward to working with TFBS Bioscience Inc to accelerate the global translation of next‑generation therapies and expand opportunities for patients worldwide. #CellTherapy #GeneTherapy #CGT #Biomanufacturing #BioKorea2026 Stephen Weller Robert McLachlan Balaji Somasundaram Patrick Silvey Gordon Chua, PhD Shao-Hua Lo Tatung Yuan man-shiow Jiang TFBS Wilson Liao

Some moments in science and in company building stand apart. This is one of them. Publishing our work in Nature is something I don’t take lightly. It’s rare, hard-earned, and especially meaningful for a young company like Akribion Therapeutics, still early in its journey since December 2024. As a co-author and part of the team shaping this company, I feel immense pride, not only in the outcome, but in what it signifies. We are now sharing our core platform, including first in vivo data, with the scientific community. At its core is a powerful idea: identifying diseased cells through their unique RNA signatures and eliminating them with precision. Turning this vision into reality requires persistence, clarity, and an exceptional team. It’s particularly meaningful that our Head of R&D is first author; a reflection of the scientific strength we are building, even at seed stage. We are just getting started. While initially focused on HPV-positive head and neck cancer, we are expanding into broader applications across oncology, autoimmune, fibrotic, and infectious diseases. I’m deeply grateful to our team, collaborators, investors, and supporters who make this journey possible. #Biotech #GeneEditing #CRISPR #Oncology #Nature #DeepTech #TranslationalScience #PrecisionMedicine #Akribion

Proud to see Akribion Therapeutics’ core technology and first in-vivo data published in Nature. Congratulations to our team and our collaborators involved in this work! This is a major milestone for us and reflects our progress in harnessing RNA signatures to selectively eliminate pathogenic cells. We are advancing this technology toward novel therapeutic approaches with potential impact across oncology, fibrotic, autoimmune, infectious and other diseases. Stay tuned for what is still to come!  

A paper in Nature is often seen as the end of a scientific journey. For us, it feels much more like the beginning. When we started Akribion, the idea of using programmable RNA signatures to selectively eliminate cells was still highly conceptual. Over the past years, this concept has turned into a technology – and now into published data, forming the foundation for a new class of therapeutics and the path towards first-in-human studies. What makes this moment particularly meaningful to me is not just the publication itself, but the path to get here. There were many moments along the way initially building the company where things looked promising – and then suddenly didn’t. Experiments failed, timelines slipped, and the external environment was far from easy. But the team kept going and step by step, the pieces came together. This paper is the result of that persistence, scientific rigor and a lot of collaboration across institutions and disciplines. Very proud of what the team has achieved – and excited about what lies ahead as we move towards first-in-human studies. We’re currently in many conversations with investors and partners and will be at several upcoming conferences – always happy to connect. Onwards and upwards 🔬 🚀 #Biotech #Entrepreneurship #Startups #Innovation #LifeScience #BuildingInPublic #OnwardsAndUpwards

Excited for ASGCT next week! I just listened to the ASGCT 2026 Abstract Highlights podcast, which offered a great preview of the science and themes shaping this year’s meeting.   1️⃣ Big genes: SCN1A and ABCA4 AAV strategies Abstract #1 – Encoded (ETX‑101, Dravet): AAV9 delivers a transcription factor to upregulate SCN1A. Interim Phase 1/2 efficacy data in 21 patients, with outcomes potentially impacted by sirolimus use. Abstract #142 – Stoke: ASO‑based approach to upregulate SCN1A in Dravet syndrome. Abstract #115 – Ascidian (ACDN‑01, Stargardt disease, FIH): ABCA4 (6.8 kb). AAV8‑delivered RNA exon editing to reduce mutant ABCA4 RNA and restore full‑length functional protein. Abstract #3362 – HuidaGene: Investigator‑initiated study in China using a dual‑AAV intein approach to reconstitute full‑length ABCA4 in Stargardt disease.   2️⃣ In vivo gene editing and in vivo cell therapy Abstract #13 – Tessera: In vivo HSC gene editing for sickle cell disease using LNPs, with meaningful editing shown in non‑human primates. Abstract #24 – DFCI: In vivo CAR‑T generation using a T‑cell‑targeted alpha‑retroviral vector, showing CAR‑T expansion, tumor reduction, and good tolerability in a mouse neuroblastoma model.   3️⃣ Durability, safety, and real‑world questions Abstract #2384 – CHOP: No caregiver seroconversion after systemic AAV administration. Abstract #76 – Ultragenyx: Data on dosing AAV gene therapies in the presence of pre‑existing anti‑AAV antibodies. Abstract #145 – Telethon Institute: Long‑term follow‑up in beta‑thalassemia shows variability in durability and outcomes. Abstract #1357 – Sanofi: Liver‑directed AAV for PKU showed limited efficacy despite dose escalation; program discontinued, but data shared. Abstract #3378 – HRI/Hera: Sibling comparison in GM1 highlights challenges in natural history and outcome interpretation.   Which abstracts are you most excited about? I will be at the Lonza booth #1449. Come chat about in vivo gene editing CMC toolboxes (viral and non‑viral) and IIT translation. Podcast link: https://lnkd.in/eZ4FvBAv

Looking forward to sharing some of our most recent work at the American Society of Gene & Cell Therapy meeting in Boston next week. Hope to see you there! ⏩Discovery of dexamethasone-inducible transgene regulatory elements (TREs) via high-throughput in vivo screening with AAV vectors. Oral Presentation ID120. (Ellie Guilfoyle) Tuesday, May 12, 03:30 PM - 03:45 PM ET. Session: Reprogramming the AAV vector genome. ⏩Combinatorial screening of regulatory elements distinguishes transgene expression between cardiac and skeletal muscle in vivo. Oral Presentation ID123. (Carson Key Taylor) Tuesday, May 12, 04:15 PM - 04:30 PM ET. Session: Reprogramming the AAV vector genome. ⏩Whole genome methylation analysis in exhausted T cells reveals targets for CRISPR epigenetic editors. Oral Presentation ID402. (Rachel Conover) Thursday, May 14, 04:30 PM - 04:45 PM ET. Session: Programmable epigenetic and RNA editing for precision regulation. ⏩A high-throughput and modular screening platform to optimize CAR designs for oncology and autoimmunity. Poster Presentation ID3250. (Sean McCutcheon & Josh Black) Thursday, May 14, 05:00 PM - 06:30 PM ET. #ASGCT2026 #GeneTherapy #CellTherapy #DukeCAGT