STAT
STAT+: FDA approves Ultragenyx gene therapy
Regeneron wins ultra-rare disease drug approval, a validating win for mRNA research, and more biotech news from The Readout
Gene Therapy (gene addition)
Gene-editing news
STAT
STAT+: Pharmalittle: We’re reading about a Regeneron rare disease drug, an Ultragenyx gene therapy, and more
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)
STAT
STAT+: Capricor Therapeutics’ Duchenne treatment is heading for an FDA rejection
Capricor Therapeutics is trying to stave off FDA rejection of its cell therapy for Duchenne muscular dystrophy.
Duchenne Muscular Dystrophy
Aug 20, 2026
Is Targeted Protein Degradation the “Break” Neurology Needs?
In this webinar, The post Is Targeted Protein Degradation the “Break” Neurology Needs? appeared first on GEN - Genetic Engineering and Biotechnology News .
Protecting Scientific Intent in AI-Enabled Labs by Not Letting AI Set the Mission
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…
Built Without Bacteria: Bringing Cell-Free Synthesis to the Bench
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…
FDA approves Ultragenyx's gene therapy Genglycos for glycogen storage disorder
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)
Aug 19, 2026
Human Multi-Organ Chip Offers New Insight Into Cancer Metastasis
A 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…
CancerCell Therapy Company Aims to Pioneer Analytics Automation
BlueRock 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…
Vector Production a Bottleneck for Gene Therapy Sector
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)Gene Switch Uses Electromagnetic Fields to Control Genes Remotely
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…
Amgen cans its TScan partnership; Amylyx reveals $350M offering
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…
Single gene injection reverses inherited heart disease in mice and patient-grown tissue
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)STAT+: In wake of boy’s death, gene editing experts dissect Huidagene’s decisions
Experts question technical improvements Huidagene tried when using a Duchenne muscular dystrophy treatment strategy largely abandoned by U.S. companies.
Duchenne Muscular Dystrophy
Aug 18, 2026
Embedding Regulatory Strategy in Cell and Gene Therapy Development
In 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)Exclusive: Third death revealed in China's popular but opaque trials
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…
Aug 17, 2026
The Microbiome Field Enters Its Next Chapter
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…
Rewriting Disease: Oligonucleotides Take Aim at the Untreatable
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…
Pharma Races to Scale AI as Billions Flow into Drug Discovery
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 -…
Animals have been eating nature’s original bioplastic for millions of years
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…
Aug 14, 2026
Cloning could be used to save species—or make human “organ sacks”
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…
Aug 12, 2026
In vivo CRISPR screen identifies gene edits that strengthen CAR-T therapy against solid tumors
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 just created female clones of male mice
Scientists 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…
Aug 11, 2026
CRISPR-armed phages help treat severe superbug infection
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
Aug 5, 2026
Gene-edited beagles may offer a future option for people with dog allergies
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…
Jul 26, 2026
CRISPR makes prostate cancer vulnerable to immunotherapy
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…
Cancer
Jul 15, 2026
Deaf people excluded from gene-editing debate | Letter
There 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…
Jul 5, 2026
The Guardian view on gene-edited humans: darker uses must be acknowledged alongside medical ones | Editorial
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-Cas9
Jul 1, 2026
FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease
The 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)
Jun 15, 2026
Scientists turned red lettuce green and something surprising happened
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…
May 30, 2026
‘There is no way to stop this’: ‘Biotech Barbie’ Cathy Tie on her mission to genetically modify babies
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…
Apr 19, 2026
‘Oscar of science’ awarded to team behind gene therapy that restores lost vision
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)
Sep 7, 2025
Scientists just made CRISPR three times more effective
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…
Aug 10, 2025
Scientists just found a tiny molecule that could change how we lose weight
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…
May 21, 2025
Scientists design gene delivery systems for cells in the brain and spinal cord
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)
May 12, 2025
CAR-T cell therapy for cancer causes 'brain fog,' study shows
Cancer treatment with a cell-based immunotherapy causes mild cognitive impairment, a Stanford Medicine team found. They also identified compounds that could treat it.
Cancer
Apr 9, 2025
Engineering smart delivery for gene editors
A 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-Cas9
Jan 28, 2025
Escaping the endosome: Bend lipids improve LNP mRNA delivery and gene editing
A 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
Atlas briefs
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.
How to read the evidence levels on this site
The CRISPR Atlas assigns every record one of four evidence levels, and as of Atlas records, those levels span 92 pages in total. Understanding what each means prevents a common mistake: treating experimental findings as established medicine. Laboratory research, covering five Atlas records, means work conducted in cells or animals but not yet tested in people. Preclinical research, covering 12 records, similarly describes findings that have not reached human trials, though the term often signals more advanced preparatory work. Neither level justifies conclusions about human safety or effectiveness. Clinical research, the largest tier at 59 records, means a therapy is being studied in human trials but has not been approved by a regulatory authority. Results may be promising, but trials can pause, fail, or reveal unexpected harms, as Atlas records of a 2025 trial death illustrate. Approved treatment, covering 16 records, is reserved for therapies that have cleared regulatory review. On that basis, only one gene-editing medicine, Casgevy using CRISPR-Cas9, carries that designation in Atlas records. The distinction matters because only approved treatments have met the evidentiary bar regulators require before clinical use.
19h agoThe hardest unsolved problems
As Atlas records show, the diseases with approved gene-editing treatment share a practical advantage: both sickle cell disease and beta thalassemia involve blood cells that can be removed from a patient, edited outside the body using CRISPR-Cas9, and reinfused. This ex vivo approach sidesteps one of gene editing's hardest problems — delivering molecular machinery to cells inside a living person. Conditions such as Duchenne muscular dystrophy, cystic fibrosis, and Huntington's disease remain preclinical partly because their target tissues, muscle, lung, and brain respectively, are far harder to reach safely and efficiently. The liver is an exception: it absorbs lipid nanoparticles readily, which is why ATTR amyloidosis and hereditary angioedema have reached Phase III with in vivo CRISPR approaches. Even there, Atlas records document a participant death from severe liver injury during the MAGNITUDE trial, illustrating that in vivo delivery carries risks absent from the ex vivo model. Evidence level, target-tissue access, and delivery technology together explain the gap between approved treatments and programmes that remain experimental.
19h agoWhat has changed most recently
According to Atlas records, the most recent developments span 2025 and 2026. The furthest-reaching result, classified at the clinical research evidence level, came when lonvoguran ziclumeran (lonvo-z) produced positive Phase 3 outcomes in hereditary angioedema — recorded as the first successful registrational trial of gene editing performed inside the body. A licence application was signalled for the second half of 2026. Separately, 2025 brought the first clinical evidence for prime editing: results from PM359 in chronic granulomatous disease showed that prime-edited stem cells engraft and restore immune function previously absent in patients. Also in 2025, a base editor was designed, manufactured, and authorised for a single infant with a specific mutation; the patient improved and was discharged. On the regulatory side, also recorded for 2026, the FDA reduced its default trial requirement and issued draft guidance under which improvement in one participant could, in defined circumstances, support approval for ultra-rare individualised therapies. A serious 2025 safety event — a patient death in the MAGNITUDE trial — led to a temporary FDA clinical hold, later lifted, underscoring that permanent in vivo edits carry irreversible risk.
19h agoWhere gene editing actually stands today
As reflected in Atlas records, one CRISPR-based gene editing treatment has reached approval: Casgevy (exagamglogene autotemcel), which uses CRISPR-Cas9 to treat sickle cell disease and beta thalassemia. Two further approved therapies, Lyfgenia and Zynteglo, address the same blood disorders but work through 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 targets ATTR amyloidosis, lonvoguran ziclumeran targets hereditary angioedema and has reported positive Phase III results according to Atlas records, while risto-cel and BEAM-302 apply base editing to sickle cell disease and alpha-1 antitrypsin deficiency respectively. Several earlier-phase trials are active across cancer, familial hypercholesterolemia, chronic granulomatous disease and diabetes. Despite this progress, the Atlas records conditions such as Duchenne muscular dystrophy, cystic fibrosis, Huntington's disease and Parkinson's disease as preclinical or discovery-stage only, and a substantial share of tracked pages sit at the preclinical research evidence level, underscoring how much of the field remains experimental.
19h agoWhere these headlines come from
Every publisher the Atlas collects from, with how many of their stories it currently holds.
| Publisher | Articles held | Most recent |
|---|---|---|
| GEN — Genetic Engineering News | 11 | Aug 20, 2026 |
| Science Daily — Gene Therapy | 6 | Sep 7, 2025 |
| The Guardian — Genetics | 5 | Aug 5, 2026 |
| STAT | 4 | Aug 20, 2026 |
| Endpoints News | 3 | Aug 20, 2026 |
| Nature Genetics | 2 | Aug 20, 2026 |
| Medical Xpress — Genetics | 2 | Aug 19, 2026 |
| Science Daily | 2 | Aug 17, 2026 |
| MIT Technology Review | 2 | Aug 14, 2026 |
| New Scientist | 1 | Aug 11, 2026 |
| Science Daily — Genes | 1 | Jul 26, 2026 |
| FDA | 1 | Jul 1, 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.