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In eigen beheer geschreven met AI-ondersteuning van Atlas-records · 14h ago
How to read the evidence levels on this site
The CRISPR Atlas organises every page it holds — covering treatments, technologies, diseases, trials, scientists, and more — into one of four evidence levels, and as of Atlas records the distribution across those levels tells a revealing story about where the field stands. Laboratory research, the starting point, means work conducted in cells or computational models with no human testing yet. Five Atlas pages carry this label, reflecting how recently some technologies and disease areas entered serious investigation. Preclinical research moves a step further: experiments in animals or organ models that justify human testing without yet reaching it. Twelve Atlas pages sit here, covering conditions such as Duchenne muscular dystrophy, cystic fibrosis, and Huntington's disease. Clinical research is the largest group by far, with 57 Atlas pages. It spans every phase of human trials — early safety studies through large Phase 3 programmes — but critically does not mean a therapy has been shown to work well enough for routine use. Nothing at this level should be described as available or proven. Approved treatment, held by 16 Atlas pages, is the only level where a therapy has cleared a regulatory authority. The distinction matters because therapies that edit the genome permanently cannot be withdrawn if something goes wrong, making the boundary between promising research and demonstrated, authorised safety a consequential line to draw clearly.
In eigen beheer geschreven met AI-ondersteuning van Atlas-records · 14h ago
The hardest unsolved problems
According to Atlas records, the clearest dividing line between approved and earlier-stage gene editing is whether a therapy can be delivered to accessible cells outside the body. Casgevy, the only approved CRISPR gene-editing treatment in the Atlas, targets blood stem cells for sickle cell disease and beta thalassemia. Those cells can be removed from a patient, edited in a laboratory, and reinfused — a process that sidesteps the hard problem of reaching tissue inside the body. Diseases such as Duchenne muscular dystrophy, Huntington's disease, and cystic fibrosis remain preclinical, in part because their target tissues — muscle, brain, and lung respectively — are far harder to access safely with editing machinery. Even liver-targeting approaches, which benefit from that organ's natural uptake of lipid nanoparticles, have encountered safety signals: Atlas records note a patient death during the Phase III MAGNITUDE trial for ATTR amyloidosis in 2025. Cancer and diabetes programmes are in early clinical stages, reflecting both delivery complexity and the evidence still required. Approval, the records show, has so far followed the path of least biological resistance.
In eigen beheer geschreven met AI-ondersteuning van Atlas-records · 14h ago
What has changed most recently
According to Atlas records, the most recent developments span 2025 and 2026 and range from a landmark success to a serious safety event. In 2026, Intellia reported positive Phase 3 results for lonvoguran ziclumeran in hereditary angioedema — described in Atlas records as the first successful registrational trial of gene editing performed inside the body. This is a result at the clinical research evidence level. A licence application was signalled for the second half of that year, which remains a plan, not an outcome. Also in 2026, the FDA reduced its default requirement from two adequate and well-controlled trials to one and issued draft guidance under which improvement in a single participant could, in defined circumstances, support approval for ultra-rare diseases. In 2025, a participant in the Phase 3 MAGNITUDE trial of nexiguran ziclumeran died following severe liver injury. The FDA imposed a clinical hold on both MAGNITUDE studies; the hold on MAGNITUDE was later lifted. Separately in 2025, clinical research results for PM359 showed that prime-edited stem cells engrafted and restored immune function in chronic granulomatous disease patients — the first clinical evidence for prime editing. An infant with CPS1 deficiency also received a individually designed base editor and improved, though this remains an isolated case.
In eigen beheer geschreven met AI-ondersteuning van Atlas-records · 14h ago
Where gene editing actually stands today
As Atlas records stand, one CRISPR-based gene editing treatment has reached full regulatory approval: Casgevy, which uses CRISPR-Cas9 to treat sickle cell disease and beta thalassemia. Two additional approved therapies for those same conditions use lentiviral gene addition rather than gene editing and are a distinct category. At the late-stage frontier, four gene editing programmes are in Phase III trials. Nexiguran ziclumeran targets ATTR amyloidosis, lonvoguran ziclumeran targets hereditary angioedema and has reported positive Phase III results with a licence application signalled, while risto-cel and BEAM-302 apply base editing to sickle cell disease and alpha-1 antitrypsin deficiency respectively. Several earlier-phase programmes address cancer, familial hypercholesterolemia, chronic granulomatous disease and diabetes, with prime editing and CRISPR-Cas12a also entering human trials for the first time. Despite this clinical momentum, Atlas records show that conditions including Duchenne muscular dystrophy, cystic fibrosis, hemophilia, Huntington's disease and retinitis pigmentosa remain at the preclinical stage, and Alzheimer's and Parkinson's diseases are still at laboratory discovery level. The field is advancing, but most of its ambitions have yet to reach patients.
Waar deze krantenkoppen vandaan komen
| Uitgever | Artikelen opgeslagen | Meest recent |
|---|---|---|
| GEN — Genetic Engineering News | 7 | Aug 19, 2026 |
| Science Daily — Gene Therapy | 6 | Sep 7, 2025 |
| The Guardian — Genetics | 5 | Aug 5, 2026 |
| MIT Technology Review | 2 | Aug 14, 2026 |
| Medical Xpress — Genetics | 2 | Aug 19, 2026 |
| Science Daily | 2 | Aug 17, 2026 |
| Endpoints News | 2 | Aug 19, 2026 |
| STAT | 1 | Aug 19, 2026 |
| Science Daily — Genes | 1 | Jul 26, 2026 |
| Nature Genetics | 1 | Aug 18, 2026 |
| FDA | 1 | Jul 1, 2026 |
| New Scientist | 1 | Aug 11, 2026 |
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