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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 years of research in its Jerry R. Mendell Center for Gene Therapy to help patients with devastating diseases.
Gene Therapy (gene addition)
STAT
A gene therapy approved for Sanfilippo syndrome, good and bad news for Xenon Pharma, and more biotech news
Gene Therapy (gene addition)
STAT
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)FDA 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…
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) BCL11AAfter 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)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…
Sanfilippo 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)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, news about Regeneron, Novocure, Jazz's Ziihera, BioMarin, Ascendis, AstraZeneca, Hutchmed and TScan Therapeutics. Another road bump for Tavneos: The Medicines and Healthcare products…
Gene Therapy (gene addition)UniQure has submitted its Huntington’s gene therapy to the FDA, investors remain unenthusiastic on Novo Nordisk, and more of the latest pharma headlines in today's Pharmalittle newsletter.
Gene Therapy (gene addition)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)The FDA put a hold on a Regenxbio gene therapy trial again after new safety concerns emerged.
Gene Therapy (gene addition)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)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 DystrophyDNA 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…
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)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)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)No es periodismo: escrito internamente
Resúmenes breves producidos con asistencia de IA a partir de nuestros propios registros de base de datos. No han sido reportados por ningún otro medio, no llevan firma y se mantienen separados de las noticias anteriores precisamente por esa razón.
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 los editores de los que recopila información CRISPR Decoded, con el número de artículos que contiene actualmente de cada uno.
| Editor | Artículos almacenados | Más reciente |
|---|---|---|
| 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 |
Solo titulares, nombres de editores y enlaces. Los artículos siguen siendo propiedad de los editores indicados. Las fechas son las fechas de publicación comunicadas por cada feed, en UTC. Las imágenes, cuando se muestran, se sirven desde el editor y les pertenecen.
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