Drugs / CRISPR-Cas9
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT07053462 | Jun 2025 → Dec 2027 expected | end stage renal failure, ischemic disease, kidney failure, kidney neoplasm +1 | AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC | Recruiting | No outcome recorded |
| Phase 1/2 | NCT07053488 | Jun 2025 → Dec 2027 expected | cirrhosis of liver, fatty liver disease, liver cancer, liver failure +1 | AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 20
| Date | Issuer | Release |
|---|---|---|
| 2026-07-09 | METiS Pharmaceuticals | Results 唯一获奖中国企业 | 剂泰科技获评国际释控协会基因递送与编辑杰出研究者奖 metispharma.com ↗
研究显示公司自主开发的心肌细胞靶向tLNP（CM-tLNP）可高效将Cre mRNA及CRISPR/Cas9编辑系统精准递送至心肌细胞， CM-tLNP实现了近乎完全（~100%）的心肌细胞转染，CRISPR-Cas9 系统的递送在心肌细胞产生了约50%的基因组编辑，并同时在肝脏、脾脏及骨骼肌等非靶组织保持极低信号，展现出优异的器官选择性。 |
| 2026-07-09 | Metis Techbio | Results 唯一获奖中国企业 | 剂泰科技获评国际释控协会基因递送与编辑杰出研究者奖 metistechbio.com ↗
CRISPR-Cas9 系统的递送在心肌细胞产生了约50%的基因组编辑，并同时在肝脏、脾脏及骨骼肌等非靶组织保持极低信号，展现出优异的器官选择性。 |
| 2026-05-20 | METiS Pharmaceuticals | Results 全球首创心肌细胞精准递送！剂泰科技受邀在CRS 2026做最新技术突破口头报告 metispharma.com ↗
此外，CRISPR-Cas9 系统的递送导致心肌内产生了显著的基因组编辑，证实了该平台在心脏应用中的强大治疗潜力。 |
| 2026-05-20 | Metis Techbio | Results 全球首创心肌细胞精准递送！剂泰科技受邀在CRS 2026做最新技术突破口头报告 metistechbio.com ↗
此外，CRISPR-Cas9 系统的递送导致心肌内产生了显著的基因组编辑，证实了该平台在心脏应用中的强大治疗潜力。 |
| 2025-07-21 | Metis Techbio | Results 剂泰科技在2025国际释控年会展示AI纳米递送研发成果 metistechbio.com ↗
以 CRISPR-Cas9 介导的 TTR 敲低为例， AiLNP 设计 LNP 在 1.5 mg/kg 剂量下实现血清 TTR 水平 ~90% 的降低。 |
| 2024-10-21 | Genethon | Results Genethon to Showcase the Latest Advances in Gene Therapies for Multiple Diseases at the ESGCT 31st Annual Congress October 22 – 25, 2024 in Rome, Italy genethon.com ↗
CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy. |
| 2024-06-03 | Hopstem Biotechnology Inc. | Results 2024【五月精选】领域最新进展 hopstem.com ↗
该研究开发了一种经过工程改造的iGeoCas9，其基因编辑效率比野生型GeoCas9提高了100倍以上。 |
| 2024-05-23 | Hangzhou Qihan Biotech Co., Ltd. | Results Qihan Biotech Announces Publication in Microbiology Spectrum of CRISPR-Cas Multiplexed Genome Editing for Reducing African Swine Fever Virus qihanbio.com ↗
The study indicates that the long-term expression of CRISPR-Cas, specifically targeting the pathogen genome in living organisms, holds promise as an effective method to inhibit the replication and infection of the African Swine Fever Virus (ASFV). |
| 2023-10-24 | Genethon | Results The Latest Research of 14 Généthon Scientists to be Featured at the European Society of Gene & Cell Therapy’s 30th Annual Congress, Oct. 24-27, 2023, in Brussels, Belgium genethon.com ↗
Using the CRISPR-Cas9 tool, it is possible to cut and modify the DNA of cells to change the genetic information that determines the amount of utrophin. |
| 2023-04-18 | EvolveImmune United, Inc | Results EvolveImmune Therapeutics Reports First Results from CRISPR-Cas9 T Cell Exhaustion Screening Platform at American Association for Cancer Research (AACR) Annual Meeting 2023 evolveimmune.com ↗
EvolveImmune scientists utilized this screen to identify specific genes that promote T cell exhaustion, which are associated with different signaling and regulatory pathways than the PD-1/PD-L1 checkpoint pathway. |
| 2021-05-04 | Editas Medicine, Inc. | Results Editas Medicine to Present Preclinical Data Demonstrating Progress in Development of Gene Editing Medicines for the Treatment of Genetic Ocular Diseases at the American Society of Gene and Cell Therapy Annual Meeting editasmedicine.com ↗
In addition, the Company is presenting preclinical data demonstrating therapeutically relevant levels of editing using a dual AAV CRISPR-Cas9 system as a therapeutic strategy for the treatment of retinitis pigmentosa 4 ( RP4 ) on May 11 . |
| 2021-05-01 | Editas Medicine, Inc. | Results Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases at the Association for Research in Vision and Ophthalmology Annual Meeting editasmedicine.com ↗
The Company is presenting preclinical data on its Usher Syndrome 2A (USH2A) and retinitis pigmentosa 4 ( RP4 ) programs. |
| 2020-12-05 | Vertex Pharmaceuticals Incorporated | Results CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine vrtx.com ↗
CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new data on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing therapy, CTX001, that show a consistent and sustained response to treatment. |
| 2020-09-20 | Hangzhou Qihan Biotech Co., Ltd. | Results Qihan Biotech Publishes First Clinical Xenotransplantation Prototype, ‘Pig 3.0’, in Nature Biomedical Engineering qihanbio.com ↗
The results, which were published today in Nature Biomedical Engineering, show enhanced immunocompatibility of xenogeneic organs and full eradication of porcine endogenous retroviruses (PERV). |
| 2020-06-27 | Verve Therapeutics, Inc. | Results Verve Therapeutics Presents New Data in Non-Human Primates Validating Gene Editing as a Treatment Approach for Coronary Heart Disease at the ISSCR 2020 Virtual Annual Meeting vervetx.com ↗
The findings are very encouraging and add to our growing body of evidence in using both base editing and CRISPR-Cas9 in vivo against various gene targets. |
| 2020-06-22 | Nkarta, Inc. | Results Nkarta Therapeutics Presents Preclinical Findings Supporting Gene Editing of Natural Killer Cells and the Combination of Engineered NK plus T Cells at the American Association for Cancer Research (AACR) Virtual 2020 Annual Meeting II nkartatx.com ↗
An optimized CRISPR/Cas9 gene editing system was used successfully in engineered CAR NK cells to disrupt TGFβR2 and CISH, genes related to the downregulation of cytokine signaling and immune cell activation. |
| 2020-05-14 | Vertex Pharmaceuticals Incorporated | Results New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001™ for Severe Hemoglobinopathies Accepted for Oral Presentation at the 25th European Hematology Association (EHA) Congress vrtx.com ↗ |
| 2019-10-16 | Guangzhou Reforgene Medicine Co., Ltd. | Results PNAS：构建出提高CRISPR-Cas9基因编辑精确度的新变体SaCas9-HF reforgene.com ↗
在这项新的研究中，这些研究人员鉴定出一种命名为SaCas9-HF的基因改造变体，它在实验室环境中可显著提高人细胞中24个基因组位点的基因靶向准确性。 |
| 2019-06-26 | Genethon | Results First in vivo proof-of-concept in Steinert’s myotonic dystrophy, a neuromuscular disease genethon.com ↗
Ana Buj Bello’s team, a researcher at Genethon, has made the proof-of-concept of a CRISPR-Cas9 approach in a mouse model of Steinert’s myotonic dystrophy, the most common neuromuscular disease in adults. |
| 2017-11-10 | CRISPR Therapeutics | Results CRISPR Therapeutics Highlights New Additions to Portfolio of Allogeneic CRISPR-based CAR-T Therapies at SITC Annual Meeting crisprtx.com ↗
Today at the SITC Annual Meeting, scientists from CRISPR presented encouraging data from our CD19 Allogeneic CAR-T development program including in vitro and in vivo data demonstrating potent anti-tumor activity. |
All press releases naming this drug 25 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | CRISPR-Cas9 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07053488 ↗ |
| Action | Inhibit | “This genetic intervention knocks out HLA-A/B on donor cells and disables expression of HLA-DR, -DQ, -DP by disrupting CIITA (essential for class II antigen presentation).” NCT07053462 ↗ |
| Modality | CRISPR / gene editing | “This trial is a applying ex vivo CRISPR-Cas9 gene editing to donor organs to reduce HLA expression prior to transplantation.” NCT07053462 ↗ |
| Target | CIITA | “The donor kidney is treated outside the body with CRISPR-Cas9 ribonucleoprotein complexes targeting the genes HLA-A, HLA-B, and CIITA.” NCT07053462 ↗ |
| Target | HLA-A | “The donor kidney is treated outside the body with CRISPR-Cas9 ribonucleoprotein complexes targeting the genes HLA-A, HLA-B, and CIITA.” NCT07053462 ↗ |
| Target | HLA-B | “The donor kidney is treated outside the body with CRISPR-Cas9 ribonucleoprotein complexes targeting the genes HLA-A, HLA-B, and CIITA.” NCT07053462 ↗ |