STAT+: After two deaths in autoimmune cell therapy trial, Artiva is shutting down
Recent deaths are setbacks in drug companies’ interest in applying cancer therapy strategies to autoimmune diseases.
Cancer
निर्देशित दृश्य
जेनेटिक्स में नए हैं? ब्राउज़ करते समय हम हर शब्द को सरल भाषा में समझाते हैं। वही पेज, बस मदद पहले से मौजूद।
विशेषज्ञ दृष्टिकोण
आप जीव विज्ञान पहले से जानते हैं। बस सामग्री, साफ़ और संक्षिप्त, कोई अतिरिक्त स्पष्टीकरण नहीं। यह डिफ़ॉल्ट व्यू है।
CRISPR Decoded अपनी कोई खबर नहीं देता। नीचे दी गई हर headline उसके बगल में लिखे publisher की है और उनकी वेबसाइट पर नई tab में खुलती है, जहाँ उनकी शर्तें, paywalls और tracking लागू होती हैं। हम आपको भेजने से पहले पुष्टि करेंगे।
Recent deaths are setbacks in drug companies’ interest in applying cancer therapy strategies to autoimmune diseases.
Cancer
STAT
Last year, U.K. gene therapy startup Bloomsbury Genetic Therapies announced it was shutting down after dosing just one patient. Now, a nonprofit is resurrecting its lead program:
Gene Therapy (gene addition)
STAT
Bristol Myers Squibb reported that one patient died after receiving its investigational CAR-T therapy in an early autoimmune trial
CancerLANCASTER, PA — This spring, Lorraine and Mark Martin, a Mennonite family with a tax business, adopted a stubby-legged cow named Royal Lite. Lorraine, Mark and Royal Lite all have one thing…
Gene Therapy (gene addition)Bristol Myers Squibb is facing one problem after another with its autoimmune CAR-T therapy program. The company says it has figured out the cause of the safety signal in a lupus trial ...
Gene therapy is no longer the future of medicine—it's reality. Treatments that replace, silence or edit genes are already changing clinical care.
Gene Therapy (gene addition)By turning a patient's own immune cells into stronger cancer-fighting cells, CAR T-cell therapy has transformed how some blood cancers are treated. But it's been harder to extend this…
CancerThis proceedings eBook, developed in partnership with GEN and Cincinnati Children’s Hospital Applied Gene and Cell Therapy Center, captures perspectives and lessons from The Cell Summit ’26…
Paul Stoffels isn’t giving up on the cell therapy plans he had at Galapagos, the Belgian drug developer that he tried turning around as CEO before abruptly departing last year. A new…
Cell and gene therapy is accelerating across Asia-Pacific, but manufacturing remains fragmented. Strategic automation, adaptable bioprocessing platforms, stronger supply chains, and…
Gene Therapy (gene addition)Shorter cell expansion steps could significantly accelerate CAR T-cell therapy production processes, according to researchers, who cite microfluidic culture systems, real-time PAT tools…
Arrowhead Pharmaceuticals has shown that a genetic medicine administered as a subcutaneous injection can seemingly travel into the brain and lower levels of tau, a protein implicated in…
Caribou has halted trials of vispacabtagene regedleucel (vispa-cel, formerly CB-010) a treatment for relapsed or refractory B cell non-Hodgkin lymphoma; and of CB-011, an allogeneic…
Leukemia and LymphomaThe moment Kate Heath saw her 12-year-old pug, Ivy, sitting silently and staring at a wall, she knew something was wrong.
Gene Therapy (gene addition) Alzheimer's DiseaseCaribou Biosciences is reaching the end of the road. The California biotech said Tuesday it is shelving two allogeneic CAR-T cell therapy programs and seeking an exit strategy after failing…
Caribou BiosciencesShort-read RNA sequencing reporting the modifications that alter protein output is starting to make epitranscriptomics indispensable for patient selection in precision medicines. The post…
Questions about the durability of an experimental Huntington's disease therapy matter to Wall Street. They matter less to those directly affected by the disease.
Gene Therapy (gene addition)Dr. Kevin Kemp and his research team at the University of Bristol have converted the high-risk Alzheimer's gene APOE4 into the lower-risk APOE3 gene.
UniQure's shares {$QURE} were rocked Tuesday morning after the company revealed its experimental Huntington’s disease gene therapy may have less benefit than originally thought. In an…
Gene Therapy (gene addition)Drugmakers and investors have ramped up their spending and R&D attention on autoimmune diseases in recent years, with a proliferation of bispecific T cell engagers, various approaches to…
UniQure's gene therapy continues to slow Huntington's progression after four years. But the magnitude of the benefit waned compared to a similar analysis from a year ago.
Gene Therapy (gene addition)Adicet Bio reported that its off-the-shelf CAR-T therapy improved symptoms for lupus patients in an early-stage study, and it plans to soon start a pivotal trial. The Bay Area company in ...
Kyverna Therapeutics is one step closer to launching the first cell therapy for an autoimmune disease, with new data in hand showing many patients remained off of other therapies a…
A slow-release liposome system delivered local anesthesia for weeks in rats, potentially enabling longer-lasting nerve blocks and new approaches to pain management. The post New Liposome…
OXFORD, England — The world's first therapy designed to fix a patient's unique genetic mutation cost $1.65 million to make, a key facilitator of the project revealed Wednesday at a rare…
Fyodor Urnov Bespoke base editor for CPS1 deficiency (the KJ Muldoon case)In this GEN webinar, our expert speaker Joshua Michael Hare, MD, will examine how therapeutically relevant research is helping shape the development of cell therapies for cardiovascular…
Exclusive: The tax-funded brainchild of Dominic Cummings hopes to find ways to speed up wild species’ evolution Vaccines for ash trees, skin treatments for newts and gene-edited swallowtail…
Researchers have uncovered a previously unknown gene that causes resistance to a leading blood cancer drug, as well as several genes that accelerate lymphoma growth, using a powerful new…
CRISPRa (gene activation) Leukemia and Lymphoma CancerMany early-stage drug developers need a more efficient path to obtaining the GMP material required to reach the clinic. SmartScale was designed to help close that gap. The post GBI…
In his new book Curved Air , Kevin Davies, PhD, portrays the fascinating story of sickle cell disease, from its origins in sub-Saharan Africa to advances in gene therapy, along with…
Sickle Cell Disease Gene Therapy (gene addition)Plus more from Reckitt, Astellas and Hovione. Cellares’ first Asian partnership: The manufacturer has inked a new partnership with Taiwanese company GenomeFrontier Therapeutics, under which…
The partnership marks Cellares’ first collaboration with a cell therapy developer in Asia and will evaluate automated manufacturing of GenomeFrontier’s virus-free CAR T process for clinical…
Cell and gene therapy developers often struggle to scale manufacturing, according to the authors of a new review, who suggest that part of the problem is the practice of developing unit…
Gene Therapy (gene addition)Researchers have taken an important step toward solving one of the biggest challenges in gene therapy for children with metabolic liver disorders: growth itself. As a child's liver grows…
Gene Therapy (gene addition)Shinshu University Hospital's expertise in CAR T-cell therapy R&D and clinical application will be combined with Teijin Regenet's manufacturing and quality-management expertise cultivated…
Intravenous gene therapy extended lifespan, reduced neurological symptoms, and improved disease markers in a Sandhoff disease cat model, supporting further investigation of systemic AAV…
Gene Therapy (gene addition)The operational challenges of making patient-specific cell therapies require careful consideration. Choosing the manufacturing model that fits the healthcare infrastructure, the logistical…
The HiBiT Target Cell Killing (TCK) platform, with off-the-shelf, mix-and-match components, measures four killing mechanisms for broad immunotherapy modality assessment. The post ADCs…
Two companies that have spent decades trying to develop cell therapies for diabetes are set to merge, with the aim of working together to take on a market also being pursued by Vertex and…
DiabetesThe collaboration brings together Synastra’s expertise in genomic engineering, AAV vector design, rare-disease gene therapy, and translational development with NewBiologix’s proprietary…
Gene Therapy (gene addition) Duchenne Muscular DystrophyWelcome back to Endpoints Weekly. Endpoints News broke the news this week on the deaths of three patients who received Novartis’ experimental CAR-T therapy. Novartis has paused immunology…
Eli Lilly and CompanyThis eBook brings together expert perspectives on several of the most pressing analytical topics facing today’s cell and gene therapy developers. The post The Analytical Blueprint for Cell…
Gene Therapy (gene addition)The deaths of two children who received CRISPR therapies in China have rattled scientists who work on similar genetic medicines in the US, adding new fuel to a yearslong debate about the…
Three patients died in Novartis trials of its CD19-directed CAR-T treatment following a dangerous immune system response, and a similar therapy from Bristol Myers Squibb has also been put…
CAR T-cell therapy dramatically reduced disease activity in six people with severe rheumatoid arthritis that had resisted other treatments, with three achieving medication-free remission…
CancerScientists have created light-activated drugs that helped blind mice perceive light again and restored visually guided behavior without gene therapy, implants, or specialized lighting. Even…
Gene Therapy (gene addition)Researchers at the University of Toronto have developed a next-generation RNA therapeutic approach with the potential to treat a wide range of genetic diseases that share certain…
Cell therapy manufacturing startup Cellares is defending itself against Bristol Myers Squibb after the drugmaker pulled out of a contract claiming Cellares’ commercial manufacturing…
Aurora Therapeutics, the first company that focused on creating bespoke gene editing treatments, has laid off several staff members and stopped its lead program. The company was using…
Base EditingPatients 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…
Sickle Cell Disease Gene Therapy (gene addition)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 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)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 DystrophyA 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…
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 -…
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 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…
CancerNorthwestern 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…
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…
पत्रकारिता नहीं: घर में लिखा गया
हमारे अपने database रिकॉर्ड से AI की सहायता से तैयार किए गए संक्षिप्त सारांश। इन्हें किसी और ने रिपोर्ट नहीं किया है, इन पर कोई byline नहीं है, और ठीक इसी कारण से इन्हें ऊपर की खबरों से अलग रखा गया है।
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 agoहर वह publisher जिनसे CRISPR Decoded सामग्री लेता है, और अभी उनकी कितनी खबरें यहाँ मौजूद हैं।
| प्रकाशक | आलेख संग्रहीत | सबसे हाल का |
|---|---|---|
| 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 |
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