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In March 2025, a six-year-old girl received the first reported in vivo base-editing treatment directed at the human CNS. The girl, given the pseudonym “Mei” — meaning “beautiful” in Chinese — had Snijders Blok-Campeau syndrome, an extremely rare genetic condition associated with mutations in the CHD3 gene. Her condition was not life-threatening, and she was on the milder side of the disease spectrum. Tragically, a week after receiving the experimental treatment, she died. Learn more in our latest article. https://lnkd.in/gd_2GAqe

CRISPR-based gene-editing tools have transformed the biomedical field, but delivering these systems directly into human tissues — known as in vivo gene editing — remains a major challenge. Many therapeutic CRISPR approaches still rely on ex vivo editing, where the patient’s cells are removed, edited outside the body, and reinfused. This method is used partly because commonly used CRISPR proteins are relatively large, making it difficult to package them efficiently into delivery vehicles such as adeno-associated viruses (AAVs), which have strict cargo limits. To overcome these packaging restrictions, researchers have increasingly focused on smaller CRISPR-Cas systems that could be delivered directly into the body. One promising family is the Cas12f enzymes — compact bacterial “molecular scissors” that are significantly smaller than traditional CRISPR editors. Researchers from the University of Texas at Austin, funded by the National Institutes of Health (NIH) and in partnership with the biotech company Metagenomi, have identified and characterized a naturally occurring Cas12f variant, Al3Cas12f, that demonstrates robust genome editing across multiple human cell lines. Learn more in our latest article. https://lnkd.in/gvRVJhyz #ots #oligotherapeutics #oligonucleotidetherapeutics #biotech #biotechnology #science #research #medicalresearch #medicalresearchers #CRISPR #geneediting #biomedical

Chronic hepatitis B is a lifelong condition that leads to T-cell exhaustion, impaired B-cell responses, and quiet liver damage over many years. Those with chronic hepatitis B have a higher risk of developing a serious liver disease or liver cancer. More than 250 million people worldwide live with hepatitis B, which requires lifelong medication to suppress. Recent results published in the New England Journal of Medicine suggest that researchers may have developed an ASO therapy that does much more than suppress the virus, providing a finite treatment with a functional cure. Read more in our latest article. https://lnkd.in/g9b4tjwf #ots #oligotherapeutics #oligonucleotidetherapeutics #biotech #biotechnology #science #research #medicalresearch #medicalresearchers #medicalarticle #hepatitis #hepatitisb #clinical

🌐 Can’t make it to Denver? You don't have to miss out. Our virtual platform brings the premier gathering in oligonucleotide therapeutics directly to your screen, wherever you are in the world. You’ll get access to: 🔸 Live Streams: Watch groundbreaking presentations in real time, including Keynote Speaker Dr. Thomas R. Cech. 🔸 On-Demand Recordings: View scientific sessions on your own schedule, anytime, anywhere. 🔸 Global Networking: Connect digitally with hundreds of researchers, clinicians, and industry leaders. Enjoy live sessions, interactive Q&As, and on-demand access to all the groundbreaking science on your own time zone. Register now to virtually join our global OTS community October 11–14, 2026: https://lnkd.in/gYQrMyyq #ots #oligotherapeutics #oligonucleotide #biotech #biotechnology #science #research #pharmaceuticals #medicalresearch #medicalresearchers #oligonucleotidetherapeutics #ots2026 #annualoligomeeting #oligomeeting #ots26

On July 9, Roche announced it would discontinue two ongoing clinical studies investigating treatments for Huntington's disease (HD) after separate analyses showed the programs would not achieve their intended goals. While the news is a significant disappointment for the HD community, it does not mark the end of huntingtin-lowering research. Tominersen showed it’s possible to reduce mutant huntingtin protein in people and helped establish important biomarkers that will guide future clinical trials. Both studies provided valuable insights that are expected to shape future research. With other investigational therapies still advancing, including uniQure’s AMT-130 and Roche's ongoing RG6662 gene therapy programs, the lessons learned from these studies will help shape more effective treatments. Learn more in our latest article. https://lnkd.in/gQCC73Qi #ots #oligotherapeutics #oligonucleotidetherapeutics #biotech #biotechnology #science #research #medicalresearch #medicalresearchers

🏔️ Join our Annual Meeting in Denver—or stream from anywhere in the world! 🌐 Registration is still open for the 22nd Annual OTS Meeting, and we are thrilled to offer two distinct ways to experience the premier global gathering in oligonucleotide therapeutics this October. Choose the path that fits your schedule and budget: ~ Attend In-Person (Denver, CO): Immerse yourself in the full conference experience. Network face-to-face with hundreds of global leaders, explore the poster sessions, and experience our vibrant scientific community firsthand. ~ Attend Virtually: Bring the cutting edge of oligonucleotide therapeutics directly to your screen. Enjoy live-streamed presentations (including Keynote Speaker Dr. Thomas R. Cech), participate in interactive Q&As, and access session recordings on-demand. No matter how you choose to participate, you’ll be part of the vital conversations shaping the future of RNA medicine. 👉 Explore the agenda and register: https://lnkd.in/gYQrMyyq #ots #oligotherapeutics #oligonucleotidetherapeutics #ots26 #biotech #biotechnology #science #research #medicalresearch #medicalresearchers #conference #annualmeeting