STAT+: Patient dies in Bristol Myers Squibb autoimmune CAR-T trial
Where should the safety bar be for studies investigating CAR-T therapies for autoimmune conditions?
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CRISPR Decoded는 자체 뉴스를 보도하지 않습니다. 아래의 모든 헤드라인은 옆에 표시된 언론사의 것이며, 해당 언론사 웹사이트의 새 탭에서 열립니다. 그 사이트의 이용 약관, 유료 구독 정책, 추적 방식이 적용됩니다. 이동하기 전에 확인을 드립니다.
Where should the safety bar be for studies investigating CAR-T therapies for autoimmune conditions?
STAT
Gene-editing company Beam Therapeutics is alleging that one of its scientists stole intellectual property and used it to co-found a Chinese company
Beam Therapeutics
STAT
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저널리즘 아님: 자체 제작 콘텐츠
자체 데이터베이스 기록을 바탕으로 AI 지원을 통해 작성된 짧은 요약입니다. 다른 곳에서 보도된 내용이 아니며, 작성자 표시도 없습니다. 바로 그 이유로 위의 뉴스와 별도로 구분해 두었습니다.
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 Decoded가 수집하는 모든 언론사 목록과 현재 보유 중인 기사 수입니다.
| 출판사 | 보유 기사 수 | 최신순 |
|---|---|---|
| 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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