CRISPR Medicine News’ Post

📢 Mission Bio Unveils Single-Cell CRISPR T-Cell Data 🔗 Read more: https://lnkd.in/g3K6CNGY Mission Bio has unveiled new single-cell #CRISPR-engineered T-cell analysis data. The findings highlight advances in precision cell therapy characterization. About the Company: • Single-cell multi-omics company • Focus on #genomic analysis • Advanced #biotech platforms About the Research: • #CRISPR-engineered T-cell analysis • Single-cell #DNA & #RNA profiling • Cell therapy development support Key Highlights: • New data released • Enhances #CRISPR analysis precision • Supports next-gen cell therapies "Congratulations to Mission Bio." 🎉 Matthew Cato, Adam Sciambi, Zivjena Vucetic, Brian Kim 🤝 “Better cell analysis tools will accelerate the entire #CRISPR ecosystem.” #CRISPR #CellTherapy #Biotech #Innovation #Genomics FDA The National Institutes of Health Centers for Disease Control and Prevention ISO - International Organization for Standardization World Health Organization European Medicines Agency American Society of Gene & Cell Therapy ISCT, International Society for Cell & Gene Therapy Society for Immunotherapy of Cancer (SITC) Cancer Research Institute (CRI) || cGxP.wire || cGxP.Directory || cGxP.jobs || cGxP.Tech ||

Excited to present this work at #ASGCT26 on behalf of the Serif team. These data demonstrate how the core components of our Modified DNA platform come together resulting in reduced innate immune activation, improved tolerability, and durable expression in multiple preclinical therapeutic models.

Great work by the Serif Biomedicines team! We’re looking forward to sharing data at #ASGCT26 that reflects meaningful progress toward a long-standing goal in biotechnology: establishing DNA as a therapeutic modality.

Serif Biomedicines has been selected for an oral presentation at the ASGCT 2026 Annual Meeting, where the company will share preclinical data supporting Modified DNA as a potential new therapeutic modality. The findings highlight our progress in creating DNA medicines: IV administered Modified DNA-LNPs are well-tolerated in NHPs with minimal innate immune stimulation and, when combined with mRNA co-factors, in rodents drives high, durable gene expression in the liver and T cells.   For more about #ASGCT26 visit: https://lnkd.in/dHgkVjV

🔬 RNAi Therapeutic (ARO-APO(a)-LRx) for Elevated Lipoprotein(a) ✨ You can now witness a clinical trial aiming to cure the once‑incurable risk of high Lipoprotein(a)–driven heart disease. We are shifting from merely treating symptoms to rewriting biology, using targeted Gene‑silencing technology that redefines cardiovascular prevention in patients with elevated Lp(a). ✓ 🩺 Elevated Lipoprotein(a) drives cardiovascular risk; no approved therapies lower it, leaving patients vulnerable to heart attacks and strokes. ✓ 🧬 GalNAc‑siRNA ARO‑APO(a)‑LRx silences hepatic LPA gene, cutting plasma Lipoprotein(a) up to eighty percent. ✓ 💊 Approval enables twice‑yearly subcutaneous injections, substantially lowering cardiovascular events for high‑Lp(a) patients. 🟢 Would you trust this gene‑silencing therapy for yourself or a loved one? #LpA #GeneTherapy #CardiovascularHealth #ClinicalTrials #Innovation

🔬 RNAi Therapeutic (ARO-APO(a)-LRx) for Elevated Lipoprotein(a) ✨ You can now witness a clinical trial aiming to cure the once‑incurable risk of high Lipoprotein(a)–driven heart disease. We are shifting from merely treating symptoms to rewriting biology, using targeted Gene‑silencing technology that redefines cardiovascular prevention in patients with elevated Lp(a). ✓ 🩺 Elevated Lipoprotein(a) drives cardiovascular risk; no approved therapies lower it, leaving patients vulnerable to heart attacks and strokes. ✓ 🧬 GalNAc‑siRNA ARO‑APO(a)‑LRx silences hepatic LPA gene, cutting plasma Lipoprotein(a) up to eighty percent. ✓ 💊 Approval enables twice‑yearly subcutaneous injections, substantially lowering cardiovascular events for high‑Lp(a) patients. 🟢 Would you trust this gene‑silencing therapy for yourself or a loved one? #LpA #GeneTherapy #CardiovascularHealth #ClinicalTrials #Innovation

🔬 RNAi Therapeutic (ARO-APO(a)-LRx) for Elevated Lipoprotein(a) ✨ You can now witness a clinical trial aiming to cure the once‑incurable risk of high Lipoprotein(a)–driven heart disease. We are shifting from merely treating symptoms to rewriting biology, using targeted Gene‑silencing technology that redefines cardiovascular prevention in patients with elevated Lp(a). ✓ 🩺 Elevated Lipoprotein(a) drives cardiovascular risk; no approved therapies lower it, leaving patients vulnerable to heart attacks and strokes. ✓ 🧬 GalNAc‑siRNA ARO‑APO(a)‑LRx silences hepatic LPA gene, cutting plasma Lipoprotein(a) up to eighty percent. ✓ 💊 Approval enables twice‑yearly subcutaneous injections, substantially lowering cardiovascular events for high‑Lp(a) patients. 🟢 Would you trust this gene‑silencing therapy for yourself or a loved one? #LpA #GeneTherapy #CardiovascularHealth #ClinicalTrials #Innovation

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

Intellia’s Phase 3 result in hereditary angioedema may be remembered as more than a positive rare-disease readout. It may be the moment in vivo CRISPR started moving from scientific possibility to therapeutic category. The data are impressive: a single infusion of lonvoguran ziclumeran, lonvo-z, reduced HAE attacks by 87% versus placebo during the primary evaluation period, and most treated patients were both attack-free and free from ongoing therapy during that window. The therapy is designed to edit KLKB1 in vivo, lowering kallikrein activity and reducing the bradykinin biology that drives attacks. That is not incremental medicine. That is a different therapeutic proposition. But it also raises the harder question the field now has to face more openly: When is permanent editing justified? HAE is a rational place to ask that question. The pathway is well validated. The biology is clean. The disease burden is real. Existing kallikrein-targeting therapies already support the mechanism, which lowers some of the scientific risk. Still, a one-time in vivo CRISPR therapy is not just a more convenient drug. It is a different kind of medical commitment. For patients, regulators, physicians, and payers, the calculus is no longer only efficacy versus safety. It is efficacy, safety, durability, reversibility, uncertainty, and trust. Six months of Phase 3 efficacy is highly encouraging. Longer-term follow-up will determine how transformative this really becomes. The most interesting shift is that the central CRISPR question may be changing. Not: Can we edit the genome in vivo? But: In which diseases does permanent editing offer enough benefit to justify its permanence? That is where the next phase of the field will be won or lost.

🧬 Epidermolysis Bullosa (EB) – Weekly Research Update EB research continues to move forward in ways that are incremental, meaningful, and deeply patient‑centered. 🧪 What’s advancing Gene‑corrected skin grafts and cell‑based therapies continue to demonstrate long‑term durability in treated areas, strengthening skin integrity and reducing repeated wound breakdown. These approaches aim to repair skin at a biological level, not just manage symptoms. 🔍 Current EB research priorities: Expanding gene and cell therapies beyond single wound sites Improving delivery methods and repeat‑treatment potential Measuring outcomes that reflect pain reduction, infection risk, and quality of life 💡 Why this matters For people living with EB, progress is measured in less pain, fewer infections, and more independence. Each step toward biological repair is a step away from lifelong damage control. ⭐ Weekly EB Spotlight EB Research Partnership (EBRP) — founded by Eddie Vedder — continues to accelerate gene, cell, and protein‑based therapies aimed at curing EB, not just treating it. 📌 Progress may be steady rather than fast, but for the EB community, it is deeply personal. 🔗 Sources • Gene‑corrected epidermal grafts in EB (long‑term outcomes, NEJM): https://lnkd.in/gx2TxWdQ • EB Research Partnership (founded by Eddie Vedder): https://ebresearch.org • NIH Rare Diseases overview of EB research: https://lnkd.in/gf9bvFi8 #EpidermolysisBullosa #EBResearch #RareDisease #GeneTherapy #EBRP #RareDiseaseAdvocacy