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CRISPR Decoded kendi haberlerini yayınlamaz. Aşağıdaki her başlık, yanında adı geçen yayıncıya aittir ve onların web sitesinde yeni bir sekmede açılır; orada kendi koşulları, ücretli içerikleri ve izleme uygulamaları geçerlidir. Sizi göndermeden önce onay isteyeceğiz.
Caribou Biosciences is shutting down after failing to raise the money necessary to fund a late-stage clinical trial of its off-the-shelf CAR-T therapy for lymphoma.
Caribou Biosciences Jennifer Doudna Leukemia and Lymphoma
STAT
Why have so many Americans lost trust in science? And what's the deal with a new gene-editing lawsuit? Find out on this week's episode of "The Readout LOUD" podcast.
STAT
Before the end of September, UniQure is expected to report four-year results from the closely tracked clinical trial of its gene therapy for Huntington’s disease.
Gene Therapy (gene addition)Cell therapy biotech Artiva Biotherapeutics abruptly announced Tuesday morning that it is effectively shutting down and laying off almost all of its staff after two patients died in its…
Plus news about Genmab, Rumi, Prana and Fractyl. Outpace Bio ends cell therapy trial: The Seattle startup canned a Phase 1 ovarian cancer trial of its mesothelin autologous CAR-T, called…
CancerBeam's first data on a rare disease gene editing therapy: Beam Therapeutics shared initial data from a Phase 1/2 trial of a base editing therapy for glycogen storage disease type Ia ...
Beam Therapeutics Base EditingEpicrispr Biotechnologies continued to show encouraging findings from testing of its epigenetic editing treatment for the genetic muscle disease facioscapulohumeral muscular dystrophy…
Epigenetic Editing Duchenne Muscular DystrophyCAR-T therapy has revolutionized blood cancer treatment, particularly in multiple myeloma. But an ongoing concern remains: If patients’ cancers progress after receiving CAR-T, what other…
Leukemia and Lymphoma CancerBeam Therapeutics on Tuesday shared additional data from an early-stage trial of its gene therapy candidate in a rare disease that causes lung and liver damage. In six patients in a Phase…
Beam Therapeutics Gene Therapy (gene addition) Base EditingMany researchers believe cancer cell therapies called CAR-Ts could shift the treatment paradigm for patients with autoimmune disease. But three recently disclosed deaths in Novartis’…
CancerUnder the agreement, Andelyn will transition Genprex's adeno-associated virus (AAV)-based diabetes gene therapy candidate with process optimization, scale-up, and IND-enabling and…
Gene Therapy (gene addition) DiabetesResearchers at Charité report promising results from the first CAR T-cell trial for treatment-refractory rheumatoid arthritis, with all six patients improving and three achieving sustained…
MUNICH — CRISPR Therapeutics’ gene editing candidate showed mixed durability results a year after patients were dosed, according to early-stage data presented Friday at the annual meeting…
CRISPR TherapeuticsGazetecilik değil: kurum içinde yazılmıştır
Kendi veritabanı kayıtlarımızdan yapay zeka yardımıyla üretilmiş kısa özetler. Başka hiçbir kaynak tarafından haber yapılmamıştır, imza taşımaz ve tam da bu nedenle yukarıdaki haberlerden ayrı tutulur.
CRISPR Decoded organises its 92 pages across four evidence levels, and the distinction matters because it signals how much human data sits behind any claim. Laboratory research, covering 5 pages, describes work done entirely in cells or computer models. No human has received anything at this stage. Preclinical research, covering 12 pages, adds animal studies but still involves no human trials. Both levels indicate that a technology or disease application is at an exploratory stage, and according to CRISPR Decoded records, conditions such as Parkinson's disease sit at laboratory research while Duchenne muscular dystrophy remains preclinical. Clinical research is the largest category, with 59 pages. It covers every phase of human trials, from first-in-human Phase I studies through to Phase III registrational programmes. Being in clinical research means human data exists, but it does not mean a therapy is proven safe, effective or available. Approved treatment, covering 16 pages, is reserved for therapies that have cleared regulatory review. According to CRISPR Decoded records, only one gene editing therapy, Casgevy, holds that status, making the approved category the narrowest and most consequential distinction of all.
16h agoApproved gene editing treatments exist today for sickle cell disease and beta thalassemia, and the records of CRISPR Decoded point to a shared reason: both diseases involve blood stem cells that can be removed from a patient, edited outside the body, and returned. This ex vivo approach sidesteps one of the hardest problems in the field, namely delivering editing machinery reliably to cells inside a living person. Casgevy, a CRISPR-Cas9 therapy, reached approval on exactly this basis. Conditions that require editing inside the body face a steeper path. The liver is relatively accessible via lipid nanoparticles, which is why ATTR amyloidosis and hereditary angioedema have reached Phase III. Tissues such as muscle, the central nervous system and the lung are harder to reach, and Duchenne muscular dystrophy, Huntington's disease and cystic fibrosis remain preclinical in these records. A 2025 death in the MAGNITUDE liver-targeting trial also illustrated a further constraint: an irreversible in vivo edit cannot be withdrawn if something goes wrong, raising the safety threshold regulators apply.
16h agoAccording to CRISPR Decoded records, the most recent developments span 2023 to 2026 and range from a landmark approval to the first positive Phase 3 result for in vivo gene editing. In November 2023, the UK MHRA authorised Casgevy, a CRISPR-Cas9 therapy, for sickle cell disease and beta thalassemia, with FDA authorisation following in December 2023 and January 2024. This represents an approved treatment, the highest evidence level recorded. In 2025, results from PM359 in chronic granulomatous disease provided the first clinical evidence for prime editing, rated clinical research: prime-edited stem cells engrafted and restored immune function. That same year, a participant in the Phase 3 MAGNITUDE trial of nexiguran ziclumeran died following severe liver injury, prompting a voluntary pause and an FDA clinical hold. The hold on MAGNITUDE was later lifted. In 2026, Intellia reported positive Phase 3 results, clinical research evidence, for lonvoguran ziclumeran in hereditary angioedema, described in CRISPR Decoded records as the first successful registrational trial of in vivo gene editing, with a licence application signalled for later that year.
16h agoAs reflected in CRISPR Decoded records, one gene editing treatment has reached regulatory approval: Casgevy, a CRISPR-Cas9 therapy for sickle cell disease and beta thalassemia, authorised first by the UK MHRA in November 2023 and subsequently by the FDA. Two approved lentiviral gene-addition therapies, Lyfgenia and Zynteglo, address the same blood disorders but use a different mechanism and are not gene editing. Four gene editing programmes have reached Phase III. Nexiguran ziclumeran targets ATTR amyloidosis, lonvoguran ziclumeran targets hereditary angioedema and reported the first positive Phase III result for in vivo gene editing, risto-cel uses base editing for sickle cell disease, and BEAM-302 applies base editing to alpha-1 antitrypsin deficiency. Several earlier-stage trials cover cancer, familial hypercholesterolemia, chronic granulomatous disease and diabetes. A significant portion of the broader landscape remains preclinical or at discovery stage, including programmes for Duchenne muscular dystrophy, cystic fibrosis, hemophilia, Huntington's disease, Parkinson's disease and Alzheimer's disease, underlining how much of gene editing's potential has not yet been tested in humans.
16h agoCRISPR Decoded'ın haber topladığı her yayıncı ve şu anda barındırdığı makale sayısı.
| Yayıncı | Tutulan makaleler | En yeni |
|---|---|---|
| GEN — Genetic Engineering News | 65 | Oct 27, 2026 |
| Endpoints News | 50 | Oct 27, 2026 |
| Nature Genetics | 30 | Oct 26, 2026 |
| STAT | 24 | Oct 27, 2026 |
| Phys.org — Molecular Biology | 20 | Oct 27, 2026 |
| Medical Xpress — Genetics | 15 | Oct 22, 2026 |
| Science Daily — Gene Therapy | 6 | Sep 7, 2025 |
| The Guardian — Genetics | 6 | Sep 21, 2026 |
| Science Daily — Genes | 5 | Oct 7, 2026 |
| Science Daily | 2 | Aug 17, 2026 |
| New Scientist | 2 | Sep 25, 2026 |
| FDA | 2 | Sep 17, 2026 |
| MIT Technology Review | 2 | Aug 14, 2026 |
Yalnızca başlıklar, yayıncı adları ve bağlantılar. Makaleler, adı geçen yayıncıların mülkiyetinde kalmaya devam eder. Tarihler, her beslemenin UTC olarak bildirdiği yayın tarihlerdir. Gösterilen görseller yayıncı tarafından sunulur ve onlara aittir.
Bu bağlantı, bizim işletmediğimiz bir web sitesini açar. İçeriğini, doğruluğunu veya kendi çerezleri ve izleme uygulamaları dahil verilerinizi nasıl işlediğini denetleyemeyiz. Orada okuduklarınız bizim tarafımızdan incelenmemiştir.