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Scientists at a biotech company have used gene editing to create two hypoallergenic beagles that don't trigger allergies Matt Walker couldn’t pet a dog without sneezing – until he and other scientists used a gene editing technique to produce a hypoallergenic beagle named Bailey. Now, he can feed…
AstraZeneca, Ionis, Cytokinetics, Novartis and CRISPR Therapeutics, among others, will get a sense of the market potential of their heart drugs when data is presented this week at…
CRISPR TherapeuticsIn this webinar, The post Is Targeted Protein Degradation the “Break” Neurology Needs? appeared first on GEN - Genetic Engineering and Biotechnology News .
New analytical tools and microbiome-based therapies are pushing the field beyond early hype toward clinical and scientific progress. The post The Microbiome Field Enters Its Next Chapter…
How synthetic DNA and RNA are becoming the discovery engines behind a new generation of precision therapies. The post Rewriting Disease: Oligonucleotides Take Aim at the Untreatable…
Not journalism — written in-house
Short summaries produced with AI assistance from our own database records. They are not reported by anyone else, carry no byline, and are kept separate from the news above for exactly that reason.
The CRISPR Atlas assigns every page one of four evidence levels, and according to Atlas records those levels are distributed as follows: Laboratory research covers 5 pages, Preclinical research 12, Clinical research 59, and Approved treatment 16. Laboratory research means work conducted entirely in cell cultures or computational models, with no animal or human data yet. Preclinical research adds animal studies that demonstrate a concept well enough to justify human testing but carry no guarantee of the same result in people. Clinical research spans the full range of human trials, from first-in-human safety studies through large Phase 3 efficacy studies; with 59 pages, this is the largest category in Atlas records, reflecting how much gene editing activity is still under investigation rather than established. Approved treatment, covering 16 pages, is reserved for therapies that have cleared a regulatory authority's full review. The distinction matters because the level signals how much uncertainty remains. An experimental therapy in clinical research may show early promise but has not yet demonstrated the safety and efficacy profile that regulators require, and the Atlas records its status as such rather than implying a conclusion has been reached.
16h agoAs reflected in Atlas records, the clearest reason some diseases have approved gene-editing treatments while others remain experimental comes down to which tissues scientists can reliably reach with an editing tool. Sickle cell disease and beta thalassemia advanced furthest because their target cells — blood stem cells — can be removed from the patient, edited outside the body, and reinfused. That ex vivo approach sidesteps the hardest delivery problem: getting molecular machinery safely into the right tissue inside a living person. Casgevy, the only approved CRISPR-Cas9 gene-editing treatment in the Atlas, works exactly this way. In vivo editing, where the tool must travel to its target organ — liver, lung, muscle, brain — inside the body, is far harder. Liver-targeting therapies such as nexiguran ziclumeran for ATTR amyloidosis reached Phase III, benefiting from lipid nanoparticles that naturally accumulate in liver tissue. Muscle and brain remain preclinical for conditions including Duchenne muscular dystrophy and Huntington's disease, because no delivery system yet moves editing tools there reliably. Safety events, including a fatality recorded in the MAGNITUDE trial, further illustrate why reaching approval demands both access and demonstrated tolerability.
16h agoAccording to Atlas records, the most recent developments span 2025 and 2026. The furthest-reaching result recorded is from 2026: lonvoguran ziclumeran (lonvo-z), a CRISPR-Cas9 therapy for hereditary angioedema, produced positive Phase 3 results in the completed HAELO trial — described in the Atlas as the first successful registrational trial of in vivo gene editing. This is a clinical research result, not an approval; a licence application was signalled for the second half of 2026. Also in 2026, the FDA reduced its default requirement from two adequate and well-controlled trials to one and issued draft guidance allowing a single patient's improvement to support approval in defined ultra-rare circumstances. In 2025, three distinct results were recorded. PM359, the first prime-editing therapy in humans, produced published clinical evidence at Phase I/II level that prime-edited stem cells could engraft and restore immune function in chronic granulomatous disease patients. A single infant with CPS1 deficiency received a bespoke base-editing therapy designed for his specific mutation; he improved and was discharged. Separately, a participant in the Phase 3 MAGNITUDE trial of nexiguran ziclumeran died following severe liver injury, prompting a clinical hold, though the hold on one MAGNITUDE study was later lifted.
16h agoAs of Atlas records, one CRISPR-based gene editing treatment has received regulatory approval: Casgevy (exagamglogene autotemcel), a CRISPR-Cas9 therapy authorised for sickle cell disease and beta thalassemia. Two additional approved therapies for those same conditions — Lyfgenia and Zynteglo — use lentiviral gene addition rather than gene editing and are classified separately. At the late-stage frontier, four gene editing candidates are in Phase III trials. Nexiguran ziclumeran is being studied in ATTR amyloidosis, and lonvoguran ziclumeran — which Atlas records note produced the first positive Phase III result for in vivo gene editing — is being studied in hereditary angioedema. Base editing approaches are also in Phase III, with risto-cel targeting sickle cell disease and BEAM-302 targeting alpha-1 antitrypsin deficiency. Earlier-stage clinical research covers cancer, familial hypercholesterolemia, diabetes, and chronic granulomatous disease, among other conditions. Meanwhile, diseases including Duchenne muscular dystrophy, cystic fibrosis, Huntington's disease, and retinitis pigmentosa remain at the preclinical stage, underscoring how much of this field is still years from human testing.
16h agoEvery publisher the Atlas collects from, with how many of their stories it currently holds.
| Yayıncı | Tutulan makaleler | En yeni |
|---|---|---|
| GEN — Genetic Engineering News | 13 | Aug 25, 2026 |
| STAT | 6 | Aug 24, 2026 |
| Endpoints News | 6 | Aug 25, 2026 |
| Science Daily — Gene Therapy | 6 | Sep 7, 2025 |
| The Guardian — Genetics | 5 | Aug 5, 2026 |
| Nature Genetics | 4 | Aug 22, 2026 |
| Medical Xpress — Genetics | 3 | Aug 25, 2026 |
| Science Daily | 2 | Aug 17, 2026 |
| MIT Technology Review | 2 | Aug 14, 2026 |
| Science Daily — Genes | 1 | Jul 26, 2026 |
| FDA | 1 | Jul 1, 2026 |
| New Scientist | 1 | Aug 11, 2026 |
Headlines, publisher names and links only. Articles remain the property of the publishers named. Dates are the publication dates reported by each feed, in UTC. Images, where shown, are served from the publisher and belong to them.