CRISPR Medicine News

* 𝗔𝗜𝗥𝗡𝗔'𝘀 𝗥𝗡𝗔 𝗘𝗱𝗶𝘁𝗼𝗿 𝗦𝗶𝗱𝗲𝘀𝘁𝗲𝗽𝘀 𝗗𝗡𝗔 𝗶𝗻 𝗔𝗔𝗧𝗗 𝗖𝗼𝗿𝗿𝗲𝗰𝘁𝗶𝗼𝗻 * AIRNA has taken a different route to correcting the genetic lesion behind alpha-1 antitrypsin deficiency (AATD). Its oligonucleotide 𝗔𝗜𝗥-𝟬𝟬𝟭 𝗿𝗲𝘀𝘁𝗼𝗿𝗲𝗱 𝗺𝗼𝗿𝗲 𝘁𝗵𝗮𝗻 𝟰𝟬 µ𝗠 𝗼𝗳 𝗳𝘂𝗻𝗰𝘁𝗶𝗼𝗻𝗮𝗹 𝗔𝗔𝗧 in a transgenic mouse model 𝘄𝗶𝘁𝗵𝗼𝘂𝘁 𝗮𝗹𝘁𝗲𝗿𝗶𝗻𝗴 𝗮 𝘀𝗶𝗻𝗴𝗹𝗲 𝗯𝗮𝘀𝗲 𝗼𝗳 𝗴𝗲𝗻𝗼𝗺𝗶𝗰 𝗗𝗡𝗔, according to data presented at the American Society of Gene and Cell Therapy (ASGCT) annual meeting. The results underpin the ongoing Phase 1 RepAIR1 trial and 𝗽𝗼𝘀𝗶𝘁𝗶𝗼𝗻 𝗥𝗡𝗔 𝗲𝗱𝗶𝘁𝗶𝗻𝗴 𝗮𝘀 𝗮 𝗰𝗿𝗲𝗱𝗶𝗯𝗹𝗲 𝗮𝗹𝘁𝗲𝗿𝗻𝗮𝘁𝗶𝘃𝗲 𝘁𝗼 𝘁𝗵𝗲 𝗖𝗥𝗜𝗦𝗣𝗥-𝗖𝗮𝘀 approaches that dominate gene correction. »𝘍𝘳𝘰𝘮 𝘳𝘦𝘱𝘢𝘪𝘳𝘪𝘯𝘨 𝘥𝘪𝘴𝘦𝘢𝘴𝘦-𝘤𝘢𝘶𝘴𝘪𝘯𝘨 𝘷𝘢𝘳𝘪𝘢𝘯𝘵𝘴 𝘵𝘰 𝘪𝘯𝘵𝘳𝘰𝘥𝘶𝘤𝘪𝘯𝘨 𝘱𝘳𝘰𝘵𝘦𝘤𝘵𝘪𝘷𝘦 𝘷𝘢𝘳𝘪𝘢𝘯𝘵𝘴, 𝘸𝘦 𝘣𝘦𝘭𝘪𝘦𝘷𝘦 𝘙𝘕𝘈 𝘦𝘥𝘪𝘵𝘪𝘯𝘨 𝘰𝘧𝘧𝘦𝘳𝘴 𝘢 𝘱𝘰𝘸𝘦𝘳𝘧𝘶𝘭 𝘢𝘯𝘥 𝘧𝘭𝘦𝘹𝘪𝘣𝘭𝘦 𝘢𝘱𝘱𝘳𝘰𝘢𝘤𝘩 𝘵𝘰 𝘵𝘩𝘦𝘳𝘢𝘱𝘦𝘶𝘵𝘪𝘤 𝘥𝘦𝘷𝘦𝘭𝘰𝘱𝘮𝘦𝘯𝘵,« Sriram Sathyanarayanan, chief scientific officer of AIRNA, says in a press release. Continue reading on the 𝗖𝗥𝗜𝗦𝗣𝗥 𝗠𝗲𝗱𝗶𝗰𝗶𝗻𝗲 𝗡𝗲𝘄𝘀 website 👇 https://lnkd.in/eCKaayqx #crisprmedicinenews #crisprmedicine #crispr #geneediting #genomeediting #aatd #alpha1antitrypsindeficiency #adar #rnaediting

The CRISPR medicine community will meet again at #CRISPRMED27, April 19-22, 2027., Cph, DK Save the date!

CRISPRMED27 Announced - The 4th CRISPR MEDiCiNE Conference is Officially On! #CRISPRMED27 #genomicmedicine #rarediseases #ultrararediseases #genetherapies #celltherapies

* 𝗙𝗧𝟴𝟭𝟵 𝗥𝗲𝗺𝗼𝗱𝗲𝗹𝘀 𝗟𝘂𝗽𝘂𝘀 𝗕 𝗖𝗲𝗹𝗹𝘀 𝗶𝗻 𝗖𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝗧𝗿𝗶𝗮𝗹 * Updated Phase 1 data presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) meeting suggest that Fate Therapeutics Inc.’s off-the-shelf CAR T-cell therapy FT819 can induce clinically meaningful responses in systemic lupus erythematosus (SLE) without conditioning chemotherapy. The findings also strengthen evidence that deep and durable remodelling of pathogenic B-cell clones may underpin these responses. Continue reading on the 𝗖𝗥𝗜𝗦𝗣𝗥 𝗠𝗲𝗱𝗶𝗰𝗶𝗻𝗲 𝗡𝗲𝘄𝘀 website 👇 https://lnkd.in/ep3Ttwgu #crisprmedicinenews #crisprmedicine #crispr #geneediting #genomeediting

Join the AIChE’s Society for Biological Engineering at GEAR 2026 - where science meets engineering to accelerate real-world impact in gene editing, RNA, and delivery systems. From breakthrough research to scalable applications, GEAR brings together the community driving the next generation of biotech innovation. 👉 Learn more and join us: https://lnkd.in/evFjfZKq #GEAR2026 #Biotech #GeneEditing #RNA #AITherapeutics #ComputationalBiology Society for Biological Engineering (SBE)

CMN is proud to have supported this milestone as a dissemination partner of The European Genomic Medicine Consortium (EGMEDC) - and to have contributed to this launch film alongside so many inspiring voices shaping the future of genomic medicine in Europe. We look forward to continuing to support the visibility, dialogue, and collaboration that #EGMEDC deserves as the real work begins... #CRISPR #GenomicMedicine #PrecisionMedicine #CRISPRMED26 #rarediseases #ultrararediseases

𝗚𝗲𝗻𝗲 𝗲𝗱𝗶𝘁𝗶𝗻𝗴 𝗶𝘀 𝗻𝗼 𝗹𝗼𝗻𝗴𝗲𝗿 𝗮 𝗳𝘂𝘁𝘂𝗿𝗲 𝗽𝗿𝗼𝗺𝗶𝘀𝗲 𝗯𝘂𝘁 𝗮 𝗴𝗹𝗼𝗯𝗮𝗹 𝗰𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝗿𝗲𝗮𝗹𝗶𝘁𝘆 We analysed 285 publicly disclosed clinical trials involving CRISPR, base editing, TALENs, zinc finger nucleases, and other emerging gene-editing technologies to better understand where the field stands today. 𝗛𝗲𝗿𝗲 𝗮𝗿𝗲 𝗮 𝗳𝗲𝘄 𝗸𝗲𝘆 𝘁𝗮𝗸𝗲𝗮𝘄𝗮𝘆𝘀: 🧬 CRISPR-Cas9 still dominates the landscape, accounting for over half of all trials 🩸 Blood cancers and haematological disorders remain the leading therapeutic focus 🚀 Base editing is now the second most-used modality in gene-editing clinicals trials 💡 Delivery technologies and ex vivo vs. in vivo approaches are beginning to define the next competitive frontier It’s also amazing to see the growing range of diseases now being targeted, from oncology and rare metabolic disorders to infectious, autoimmune, and visual diseases. 𝗪𝗮𝗻𝘁 𝘁𝗼 𝗲𝘅𝗽𝗹𝗼𝗿𝗲 𝘁𝗵𝗲 𝗱𝗮𝘁𝗮 𝗳𝗼𝗿 𝘆𝗼𝘂𝗿𝘀𝗲𝗹𝗳? Then check out the sample CMN Clinical Trials Global Report 2026 attached to this post. CMN clinical trials database is continuously updated as new gene-editing trials are disclosed globally. Get in touch to hear more about how you can access this unique database. 𝐖𝐡𝐢𝐜𝐡 𝐚𝐫𝐞𝐚 𝐨𝐟 𝐠𝐞𝐧𝐞 𝐞𝐝𝐢𝐭𝐢𝐧𝐠 𝐝𝐨 𝐲𝐨𝐮 𝐭𝐡𝐢𝐧𝐤 𝐰𝐢𝐥𝐥 𝐬𝐞𝐞 𝐭𝐡𝐞 𝐛𝐢𝐠𝐠𝐞𝐬𝐭 𝐜𝐥𝐢𝐧𝐢𝐜𝐚𝐥 𝐛𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡 𝐨𝐯𝐞𝐫 𝐭𝐡𝐞 𝐧𝐞𝐱𝐭 𝟑–𝟓 𝐲𝐞𝐚𝐫𝐬? #crisprmedicinenews #crisprmedicine #geneeditingtrials #crisprtrials #crispr #baseediting #raredisease #crisprmed

* 𝗛𝗼𝘄 𝗰𝗮𝗻 𝗘𝘂𝗿𝗼𝗽𝗲 𝗺𝗼𝘃𝗲 𝗴𝗲𝗻𝗼𝗺𝗶𝗰 𝗺𝗲𝗱𝗶𝗰𝗶𝗻𝗲 𝗳𝗿𝗼𝗺 𝘀𝗰𝗶𝗲𝗻𝘁𝗶𝗳𝗶𝗰 𝗽𝗿𝗼𝗺𝗶𝘀𝗲 𝘁𝗼 𝗰𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝗿𝗲𝗮𝗹𝗶𝘁𝘆? * In a new EGMEDC article published via CRISPR Medicine News, Tom Foti, Vice President of Scientific Partnerships at Aldevron, discusses why Aldevron has joined the The European Genomic Medicine Consortium (EGMEDC) – and why relationships, manufacturing readiness, quality systems and regulatory understanding are becoming central to the next phase of CRISPR-based medicine. The article takes the Baby KJ case as a starting point: an extraordinary example of what can happen when clinicians, researchers, regulatory partners and specialised manufacturing expertise come together around urgent patient need. For EGMEDC, the wider question is how Europe can build systems that make such collaboration possible earlier, more routinely and across borders. As Foti puts it: »𝘐𝘧 𝘺𝘰𝘶 𝘥𝘰𝘯’𝘵 𝘩𝘢𝘷𝘦 𝘢 𝘯𝘦𝘵𝘸𝘰𝘳𝘬 𝘰𝘧 𝘳𝘦𝘭𝘢𝘵𝘪𝘰𝘯𝘴𝘩𝘪𝘱𝘴, 𝘺𝘰𝘶 𝘥𝘰𝘯’𝘵 𝘬𝘯𝘰𝘸 𝘸𝘩𝘢𝘵 𝘵𝘩𝘦 𝘱𝘢𝘵𝘪𝘦𝘯𝘵 𝘤𝘩𝘢𝘭𝘭𝘦𝘯𝘨𝘦𝘴 𝘢𝘳𝘦.« Read the full article here:  https://lnkd.in/dUw4tpbg #crisprmedicinenews #crisprmedicine #crispr #geneediting #genomeediting #egmedc #regulation #clinicaltrials #babykj

#CRISPRMED26 - Looking Back on an Inspiring Gathering of the CRISPR Medicine Community in Copenhagen Special access: Explore the CRISPRMED26 Abstract Book and revisit the science, discussions, and collaborations that defined this year’s event.

* 𝗜𝗻𝘁𝗲𝗹𝗹𝗶𝗮 𝗙𝗶𝗹𝗲𝘀 𝗕𝗟𝗔 𝗔𝗳𝘁𝗲𝗿 𝗟𝗼𝗻𝘃𝗼-𝘇 𝗠𝗲𝗲𝘁𝘀 𝗣𝗵𝗮𝘀𝗲 𝟯 𝗘𝗻𝗱𝗽𝗼𝗶𝗻𝘁𝘀 * Intellia Therapeutics, Inc. has initiated a rolling biologics license application (BLA) with the U.S. Food and Drug Administration for lonvoguran ziclumeran (lonvo-z) in hereditary angioedema, positioning the candidate to become the 𝘄𝗼𝗿𝗹𝗱'𝘀 𝗳𝗶𝗿𝘀𝘁 𝗮𝗽𝗽𝗿𝗼𝘃𝗲𝗱 𝙞𝙣 𝙫𝙞𝙫𝙤 𝗖𝗥𝗜𝗦𝗣𝗥-𝗖𝗮𝘀 𝗴𝗲𝗻𝗲 𝗲𝗱𝗶𝘁𝗶𝗻𝗴 𝘁𝗵𝗲𝗿𝗮𝗽𝘆. The Cambridge, Massachusetts-based company expects to complete the filing in the second half of 2026 and 𝗹𝗮𝘂𝗻𝗰𝗵 𝗰𝗼𝗺𝗺𝗲𝗿𝗰𝗶𝗮𝗹𝗹𝘆 𝗶𝗻 𝘁𝗵𝗲 𝗳𝗶𝗿𝘀𝘁 𝗵𝗮𝗹𝗳 𝗼𝗳 𝟮𝟬𝟮𝟳, pending approval. »𝘈𝘴 𝘵𝘩𝘦 𝘧𝘪𝘳𝘴𝘵 𝘗𝘩𝘢𝘴𝘦 3 𝘥𝘢𝘵𝘢 𝘳𝘦𝘱𝘰𝘳𝘵𝘦𝘥 𝘧𝘰𝘳 𝘢𝘯 𝘪𝘯 𝘷𝘪𝘷𝘰 𝘨𝘦𝘯𝘦 𝘦𝘥𝘪𝘵𝘪𝘯𝘨 𝘵𝘩𝘦𝘳𝘢𝘱𝘺, 𝘵𝘰𝘥𝘢𝘺'𝘴 𝘏𝘈𝘌𝘓𝘖 𝘳𝘦𝘴𝘶𝘭𝘵𝘴 𝘳𝘦𝘱𝘳𝘦𝘴𝘦𝘯𝘵 𝘢 𝘱𝘳𝘰𝘧𝘰𝘶𝘯𝘥 𝘮𝘪𝘭𝘦𝘴𝘵𝘰𝘯𝘦 𝘧𝘰𝘳 𝘐𝘯𝘵𝘦𝘭𝘭𝘪𝘢, 𝘵𝘩𝘦 𝘣𝘳𝘰𝘢𝘥𝘦𝘳 𝘊𝘙𝘐𝘚𝘗𝘙 𝘢𝘯𝘥 𝘱𝘳𝘦𝘤𝘪𝘴𝘪𝘰𝘯 𝘮𝘦𝘥𝘪𝘤𝘪𝘯𝘦 𝘧𝘪𝘦𝘭𝘥𝘴 𝘢𝘯𝘥, 𝘮𝘰𝘴𝘵 𝘪𝘮𝘱𝘰𝘳𝘵𝘢𝘯𝘵𝘭𝘺, 𝘵𝘩𝘦 𝘏𝘈𝘌 𝘤𝘰𝘮𝘮𝘶𝘯𝘪𝘵𝘺,« said John Leonard, Intellia's president and chief executive. Continue reading on the 𝗖𝗥𝗜𝗦𝗣𝗥 𝗠𝗲𝗱𝗶𝗰𝗶𝗻𝗲 𝗡𝗲𝘄𝘀 website 👇 https://lnkd.in/efaWPdxy #crisprmedicinenews #crisprmedicine #crispr #geneediting #genomeediting #HereditaryAngioedema Aleena Banerji Massachusetts General Hospital Markus Magerl Charité - Universitätsmedizin Berlin