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Patients with sickle cell disease are often hesitant to undergo transformative gene therapy—the current lengthy process requires multiple hospital visits to collect enough stem cells. Researchers from Boston Children's Hospital have helped smooth the road to gene therapy with a new approach that…
Sickle Cell Disease Gene Therapy (gene addition)
STAT
President Trump’s pledge to make pricey weight loss drugs widely available for low-income Americans through Medicaid is not panning out
Gene Therapy (gene addition)
STAT
The FDA put a hold on a Regenxbio gene therapy trial again after new safety concerns emerged.
Gene Therapy (gene addition)Martha’s Vineyard Hospital launches a two-year study tracking up to 300 residents to understand who develops alpha-gal syndrome after lone star tick exposure and why. The post Pioneering…
After a “comprehensive evaluation,” Bristol Myers Squibb has pulled out of its partnership with Cellares and the cell therapy manufacturer will restructure as a result. Bristol Myers…
A study in mice found that male sex hormones can suppress allergic responses by promoting sympathetic nerve connections and norepinephrine release in the lungs. The post Male Sex Hormones…
Regenxbio said it no longer expects to file for FDA approval of its Hunter syndrome gene therapy in the near future after the program was slapped with its second clinical hold this year…
Gene Therapy (gene addition)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 the…
CRISPR TherapeuticsIn this webinar, The post Is Targeted Protein Degradation the “Break” Neurology Needs? appeared first on GEN - Genetic Engineering and Biotechnology News .
Scientists see a positive trend toward using AI in a way that’s human, rather than as a self-driving lab. AI is simply a tool—albeit a powerful, adaptive one. The post Protecting Scientific…
Regeneron wins ultra-rare disease drug approval, a validating win for mRNA research, and more biotech news from The Readout
Gene Therapy (gene addition)A Regeneron drug for an ultra-rare disease that causes bone to grow where it shouldn't won U.S. regulatory approval, capping a three-decade effort
Gene Therapy (gene addition)In this GEN webinar, speakers from Flock Bio and Ribbon Bio will explore how bench-based, cell-free DNA production can complement expertise in custom DNA design and manufacturing. The post…
Ultragenyx has secured its first approved gene therapy, as the FDA cleared its asset for an ultra-rare disease that can cause life-threatening blood sugar crashes. The FDA on Wednesday…
Gene Therapy (gene addition)Capricor Therapeutics is trying to stave off FDA rejection of its cell therapy for Duchenne muscular dystrophy.
Duchenne Muscular DystrophyA human multi-organ chip models how breast cancer spreads from blood vessels to bone and lung, offering a patient-specific tool to study metastasis and potential treatments. The post Human…
CancerBlueRock says it’s helping advance the cell therapy industry by developing an automated system for analytical auditing. They say it’s among the few systems specifically designed for this…
More effective purification materials would make viral vector production more efficient, say researchers working on an affinity adsorbent-based method they claim can differentiate between…
Gene Therapy (gene addition)DNA regulatory elements, which control when, where, and to what extent specific genes are turned on or off, can be co-opted by scientists to create gene switches. The post Gene Switch Uses…
Plus news about BioArctic, Enveda, Georgiamune, Kynexis, Tolerance Bio and Network Bio. Amgen puts the "can" in TScan: Amgen is terminating its partnership with TScan Therapeutics, the…
Melbourne researchers have made a gene therapy breakthrough that could restore heart function in children with genetic heart disease, sparing them the need for transplants.
Gene Therapy (gene addition)Experts question technical improvements Huidagene tried when using a Duchenne muscular dystrophy treatment strategy largely abandoned by U.S. companies.
Duchenne Muscular DystrophyIn this GEN webinar, experts from Rose BioSolutions, a CDMO and Cell Solutions organization formed from Charles River Laboratories’ businesses, will discuss how integrating regulatory…
Gene Therapy (gene addition)A patient with an autoimmune disease has died after receiving an experimental genetic medicine from a biotech startup in China, Endpoints News has learned. The Shanghai-based company, RiboX…
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…
Investors unpack what it takes to succeed in an increasingly crowded AI biology ecosystem. The post Pharma Races to Scale AI as Billions Flow into Drug Discovery appeared first on GEN -…
Nature invented biodegradable plastic long before humans did—and animals may have been feeding on it for hundreds of millions of years. Researchers discovered that dozens of animal species…
This week I spoke to scientists who have found a way to turn male mouse embryos female. They’ve developed a CRISPR-based approach to essentially cut out the Y chromosome. It allowed them to…
For patients with blood cancers like leukemia and lymphoma, the immunotherapy known as CAR-T cell therapy can be lifesaving. Doctors remove a patient's immune cells, called T cells…
Leukemia and Lymphoma CancerScientists have deliberately turned male mouse embryos into females for the first time. A team based in Japan used a CRISPR-based approach to remove the Y chromosome from male cells and…
Bacteria-killing phage viruses souped up with DNA-destroying CRISPR appear to have helped clear up a very serious infection caused by antibiotic-resistant E. coli
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 CRISPR to make prostate cancer cells easier for the immune system to detect and destroy. The experimental treatment dramatically improved the effects of immunotherapy in…
CancerThere is no majority support for use of gene editing on non-life-threatening conditions, writes Tom Lichy of the British Deaf Association Your editorial ( The Guardian view on gene-edited…
Polling shows that the public supports this new technology, but the conversation must move beyond simple questions of safety Ever since Crispr-Cas9 gene-editing technology emerged in the…
CRISPR-Cas9The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD)…
Casgevy (exagamglogene autotemcel) Sickle Cell Disease Gene Therapy (gene addition)Researchers used genome editing to block the production of red pigments in lettuce, causing other beneficial plant compounds to build up instead. The lettuce continued to grow normally…
The Canadian entrepreneur has always pushed the boundaries of gene editing, once attempting to turn horses into unicorns. Now she is set on modifying human embryos – something her…
Married couple Jean Bennett and Albert Maguire developed Luxturna, which helped a patient see their child’s face for the first time A married couple who met over a dissected brain and went…
Gene Therapy (gene addition)Northwestern scientists have developed a new nanostructure that supercharges CRISPR’s ability to safely and efficiently enter cells, potentially unlocking its full power to treat genetic…
Researchers at the Salk Institute have used CRISPR to uncover hidden microproteins that control fat cell growth and lipid storage, identifying one confirmed target, Adipocyte-smORF-1183…
Research teams have created a versatile set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy. These delivery…
Gene Therapy (gene addition)Cancer treatment with a cell-based immunotherapy causes mild cognitive impairment, a Stanford Medicine team found. They also identified compounds that could treat it.
CancerA research team has developed an advanced delivery system that transports gene-editing tools based on the CRISPR/Cas9 gene-editing system into living cells with significantly greater…
CRISPR-Cas9A new class of lipids improve the rates at which lipid nanoparticles (LNPs) successfully deliver RNA therapeutics and gene editing tools, promising to increase their effectiveness of such…
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.
| Éditeur | Articles répertoriés | Les plus récents |
|---|---|---|
| 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.