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University of Missouri researchers have created rice plants that resist bacterial blight, a devastating disease that threatens one of the world's most important food crops. The advance could help farmers in parts of Asia and Africa, where the disease can destroy harvests and threaten livelihoods.
STAT
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)LANCASTER, 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)GEN editors Uduak Thomas and Julianna LeMieux, PhD, recap ASHG, highlighting key sessions, Ultima Genomics’ user group meeting, Illumina news, and insights from PacBio’s new CEO Mark Van…
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…
CancerIn this GEN webinar, Bernd Bodenmiller, PhD (University of Zürich), illustrates this integrated approach through two recent examples. The post Spatial Proteomics for Precision Oncology…
CancerBristol Myers Squibb reported that one patient died after receiving its investigational CAR-T therapy in an early autoimmune trial
CancerWhere should the safety bar be for studies investigating CAR-T therapies for autoimmune conditions?
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 ...
A patient has died in a Bristol Myers Squibb study of autoimmune CAR-T, the same technology that was called into question this summer when both Bristol Myers and Novartis paused similar…
Human cells hold about two meters (6.6 feet) of DNA, folded and compacted to fit inside a nucleus. That packing is essential for life, but it creates a problem for scientists. Some of the…
Huntington's disease is caused by repetitive "stutters" in DNA—specifically repeated CAG sequences—that expand abnormally over time, leading to toxic protein accumulation that contributes…
In this GEN webinar, Asimov’s VP of Business Development and Commercial Strategy Jamie Freeman, PhD, will show how Asimov combines bio-foundation models, AI agents, and synthetic biology to…
Synthetic BiologyVector production costs have a disproportionate influence on gene therapy prices, according to researchers who say that, given this, choosing the vector manufacturing method that best suits…
Gene Therapy (gene addition)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…
CancerUnlocking the Arctic’s subterranean DNA must ensure a delicate balance between scientific rigor and ethical stewardship. Synthetic biology and chemistry could help discovery of new…
Synthetic BiologyPeter Mac scientists have developed a way to precisely target the "unique molecular fingerprint" created when two genes become abnormally fused together, a discovery that could lead to a…
Germany's Merck KGaA agreed to buy UK drug developer Silence Therapeutics for $1.3 billion, building out its work in rare and specialty diseases using genetic medicines. Silence is…
RNA InterferenceThis eBook brings insight on integrated end-to-end solutions for precision engineered therapeutics. The post ADC Development from Concept to Clinic appeared first on GEN - Genetic…
This 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…
Estuary Biotherapeutics raised the curtain on Friday with $103 million to support the advancement of two preclinical gene therapy candidates for the most common type of inherited kidney…
Gene Therapy (gene addition)5,812 protein groups were quantified in postmenopausal ovaries, uncovering molecular shifts associated with inflammation, immunity, and extracellular matrix remodeling. The post Proteomics…
Bacteriophages, or viruses that infect bacteria, have a fiendishly simple purpose in life: attach to a bacterial cell, inject their DNA and then use the host's genetic machinery to…
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…
Scientists have long used a powerful gene-editing tool called CRISPR to make precise changes to DNA. But a different version of the technology, known as Cas13a, targets RNA, the genetic…
CRISPR-Cas13Cell 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…
Some people are born with a defect in their genetic material that prevents their immune system from functioning properly. In people with a condition known as GATA2 deficiency, the body does…
Prime EditingAn international research team led by TU Darmstadt Professor Beatrix Süß, working in close collaboration with partners in Germany and Denmark, has elucidated how an RNA switch newly…
Synthetic BiologyBeam'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 EditingA new antiviral defense system discovered by Columbia scientists could be adapted in the future into a gene editor that slows down aging in our cells and improves cancer therapies. At the…
CancerCaribou 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 LymphomaA Gilead-backed cell therapy maker and an I&I biotech from BioNTech vets are looking to ride the biotech industry's IPO momentum. Aspen Neuroscience and Bambusa Therapeutics are the latest…
A study in Cameroon found that sickle cell trait is associated with distinct malaria parasite genotypes, potentially explaining reduced disease risk despite similar infection rates. The…
COVID-19 accelerated efforts to develop faster, more efficient ways of manufacturing high-quality RNA sequences. The post How COVID, Characterization, and Commerce Are Driving an RNA…
Spatial technologies preserve tissue context to guide therapeutic development and translate molecular insights into clinically actionable information. The post Spatial Biology Moves Toward…
Two DNA-recording systems traced cellular family trees across mouse embryogenesis, offering new insight into how organs form and when developing cells commit to specific fates. The post DNA…
Malaria is one of the deadliest infectious diseases on the planet, afflicting people throughout recorded history. In places where malaria is most common, it's shaped human evolution by…
A new engineered gene-editing system, proven in bacteria and showing promise for use in plants, animals and humans, addresses some limitations of current gene-editing tools, with the goal…
The 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 BiosciencesScientists at UC San Francisco have developed a way to create cancer-fighting CAR-T cells directly inside the body, potentially avoiding the slow and extremely expensive process of…
CancerCaribou 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 LymphomaThis GEN webinar follows oligo quantification from method development through conjugate characterization. The post Lock Down the Critical Factors in Oligo and Oligo-Conjugate…
Short-read RNA sequencing reporting the modifications that alter protein output is starting to make epitranscriptomics indispensable for patient selection in precision medicines. The post…
Sequencing by expansion (SBX) and Roche’s AXELIOS 1 platform introduce a new approach to single-molecule sequencing, designed to give researchers flexibility across sequencing workflows…
Dillico’s GMP-ready Continuous Manufacturing by Engineering, Not Hype The post Inside All-ScaleFlow: Continuous End-to-End RNA-LNP Manufacturing appeared first on GEN - Genetic Engineering…
Epicrispr 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 DystrophyFDA Chief Scientist Steven Kozlowski, MD, said the updated FARS would come out early in FY 2027 but offered no further specifics on timing. The post FDA to Highlight Aging, Longevity…
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.
How do bacteria adapt when resources run low? ISTA researchers have found that, under starvation stress, E. coli can use bacteriophages and CRISPR-Cas immunity to transfer and incorporate…
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)This pathway, which the authors dub the “BACH2-NRF2 axis,” is independent of the well-known BCL11A transcription regulator, the target of the approved cell therapy Casgevy for sickle cell…
Sickle Cell Disease Casgevy (exagamglogene autotemcel) BCL11AGene-editing company Beam Therapeutics is alleging that one of its scientists stole intellectual property and used it to co-found a Chinese company
Beam TherapeuticsBeam Therapeutics, a Cambridge, MA-based company developing CRISPR base editing therapies, has sued two Chinese scientists and two biotech companies where they work for allegedly stealing…
Beam Therapeutics Base EditingThe gene-editing company Beam Therapeutics is alleging that one of its scientists stole intellectual property and used it to co-found a Chinese company that made a deal with VC firms.
Beam TherapeuticsDr. 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.
Predictive cell intelligence can be used to reveal cellular states and emerging cellular trajectories earlier in the manufacturing process and to support more informed bioprocessing…
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)The assembly pathway behind refractile-body proteins may have finally been resolved, researchers from Science Tokyo report. Using genetic engineering and several techniques for analyzing…
AI-assisted genomic analysis was combined with human genetics and fruit fly experiments to link reduced BRSK1 function to a variable neurodevelopmental disorder. The post AI Tool Helps Link…
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 ...
After winning FDA approval at the end of last year, two nonprofits are now launching their rare immune disease gene therapy Waskyra with a list price of $3.25 million, they told Endpoints…
Gene Therapy (gene addition)Plus, news about MediciNova, Tolerance Bio, Tanabe, Lepu Medical and Synphatec. Scynexis’ $200M+ BARDA contract: The partnership is for SCY-247, an investigational treatment for serious…
Key leaders Kenneth Greenberg (CEO), Steve Feinstein (CSO) Headquarters San Francisco Total raised $186M Key investors Vida Ventures, ARCH Ventures Partners, Illumina Ventures, Leaps by…
Gene Therapy (gene addition) Eli Lilly and CompanyResearchers used CRISPR to remove CD33 from donor stem cells, potentially giving doctors a way to attack aggressive blood cancers without destroying the healthy cells patients need after a…
Leukemia and Lymphoma CancerThe AI company Anthropic has announced that its Claude large language model has discovered a system reminiscent of CRISPR, but that doesn’t mean the Nobel prizes will start rolling in, says…
A CRISPR-based method for disrupting genes across bacteriophage genomes has been developed, creating a faster route to uncover gene functions and engineer phages for potential therapeutic…
Plus news about Frazier Life Sciences, BigHat Biosciences, Kasvu Therapeutics, Arcturus Therapeutics, Oruka Therapeutics and Viking Therapeutics. Sarepta Therapeutics settles gene therapy…
Gene Therapy (gene addition)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…
Kyverna Therapeutics touted positive one-year results on Thursday in patients with a rare autoimmune disorder called stiff person syndrome. The condition affects about 6,000 patients in the…
Sejong University researchers have developed a high-quality soybean with improved oxidative stability, reduced saturated fat and increased seed fatty acid content. The research team led by…
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)Act for Hope founder Alexander Seyf says cell and gene therapy’s next challenge is not proving the science, but building a connected ecosystem that can turn breakthrough treatments into…
Gene Therapy (gene addition)The Abigail Wexner Research Institute at Nationwide Children's Hospital celebrated the Food and Drug Administration's (FDA) approval of FAYUVI for Sanfilippo syndrome type A, following…
Gene Therapy (gene addition)A biomimetic nanoparticle platform inspired by photosynthesis helped activate surviving retinal nerve cells in mice with advanced retinal degeneration. The post Biomimetic Nanoparticles…
A synthetic biology technology has been developed that converts easily degraded messenger RNA (mRNA) into a ring-shaped form inside microbial cells, increasing protein production.
Synthetic BiologyIn 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…
In this GEN webinar, our expert speakers will discuss the latest trends, challenges, and innovations shaping fluid transfer and freeze-thaw workflows. The post Emerging Trends in Fluid…
In a study published in Molecular Therapy Advances a team of researchers led by Professor Haruhisa Inoue (Center for iPS Cell Research and Application, Kyoto University) and Professor…
Base EditingSanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease that primarily affects the central nervous system and is marked by rapid neurodegeneration beginning in…
Gene Therapy (gene addition)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…
Beacon Therapeutics has succeeded in a genetic eye disease where large drugmakers have failed, including Johnson & Johnson and Biogen. On Monday, Beacon said its gene therapy laru-zova for…
Retinitis Pigmentosa Gene Therapy (gene addition)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 CancerCRISPR may be a powerful technology for gene editing, but the system existed in bacteria long before scientists began using it. For billions of years, CRISPR has acted as a natural immune…
Many 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)A gene therapy approved for Sanfilippo syndrome, good and bad news for Xenon Pharma, and more biotech news
Gene Therapy (gene addition)A new open-access study introduces RiSpy, a data-driven fingerprinting framework that makes the identification of genome-edited (GE) rice lines more robust, scalable and broadly applicable…
The FDA approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s.
Gene Therapy (gene addition)The FDA granted full approval to a rare disease gene therapy from Ultragenyx on Thursday, giving the company a much-needed boost in an otherwise difficult year. The therapy, called UX111…
Gene Therapy (gene addition)The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA)…
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…
A new genome-editing method, prime assembly, precisely inserts long DNA sequences into targeted genomic locations, potentially enabling mutation-agnostic gene therapies for diverse genetic…
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)Não é jornalismo: produzido internamente
Resumos curtos produzidos com auxílio de IA a partir dos nossos próprios registros de banco de dados. Não são reportados por nenhum outro veículo, não têm assinatura e são mantidos separados das notícias acima exatamente por esse motivo.
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 agoTodos os veículos dos quais o CRISPR Decoded coleta conteúdo, com o número de matérias que cada um tem atualmente.
| Editor | Artigos disponíveis | Mais recente |
|---|---|---|
| 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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