相关临床试验
32
8 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
1861
进行中（未招募）
6
18.8%
已完成
13
40.6%
尚未招募
2
6.3%
招募中
9
28.1%
Unknown
2
6.3%
暂无批准数据
- Tevard Biosciences and academic collaborators published preclinical research in Science Advances on an engineered suppressor tRNA gene therapy for Duchenne muscular dystrophy caused by nonsense mutations. - In a preclinical DMD model, the therapy restored physiological levels of full-length dystrophin and improved muscle strength and motor coordination while being well tolerated. - Engineered suppressor tRNAs targeted disease-causing nonsense mutations while leaving normal stop codons intact, demonstrating what the company describes as exquisite selectivity. - Because the platform addresses nonsense mutations as a class, Tevard says it has potential applications beyond DMD and other muscular dystrophies.
- USC's Viterbi School of Engineering is launching its inaugural undergraduate artificial intelligence degree, teaching students to build AI systems and apply them to fields including drug discovery, robotics, and agriculture. - UCLA is opening what officials describe as the nation's first freestanding Department of Digital Humanities, with a new bachelor's degree integrating AI and digital tools into disciplines such as sociology, archaeology, and history. - Both institutions are responding to market demand, with USC expecting roughly 50 new students annually and UCLA projecting eventual enrollment of 150 to 300 students in the major. - Educators emphasize that ethics coursework is essential, as campuses grapple with concerns about cheating and fears that heavy AI use may erode critical thinking.
- A collaborative study from RMIT, MIT, and Harvard Medical School identifies key formulation conditions that protect mRNA-carrying nanoparticles during drying and rehydration in microneedle patches. - The research, published in Advanced Functional Materials, explains why some dry patch formulations outperform others, providing practical guidance for future vaccine patch design. - Eliminating cold-chain requirements could address a critical barrier to vaccine access; in 2024, 14.3 million children globally received no vaccines at all, per WHO and UNICEF. - The findings build on prior MIT research showing patches can be printed and stored at room temperature, with next steps focused on optimizing formulations and testing immune responses.
- The Ohio State University received a $4 million NSF Phase II design grant under the National Quantum Virtual Laboratory program to lead the DQS-CP quantum sensing consortium. - The two-year project aims to engineer a multi-component quantum sensing platform capable of capturing atomic and molecular interactions with sub-atomic precision for biomedical applications. - The sensing architecture uses three integrated layers — target analyte, spin-relay film, and entanglement-assisted quantum readout — to bypass classical resolution barriers. - A national network of academic partners including MIT, University of Chicago, UCSB, and CU Boulder will collaborate, with QuantCAD providing commercialization roadmapping.
- Loss of the transcription factor GATA6 reprograms colorectal cancer cells into a flexible, fetal-like state that promotes liver metastasis, according to a new study in Cell Stem Cell. - Researchers found that GATA6 loss triggers lineage plasticity, shifting cells from LGR5-positive to LGR5-negative states with enhanced metastatic potential, while having little effect on primary tumor growth. - The findings suggest epigenetic changes, rather than specific genetic mutations, may be the key drivers of colorectal cancer spread to the liver. - Low GATA6 levels could serve as a biomarker for metastatic risk, potentially helping identify patients who need closer monitoring or more aggressive treatment.
- MIT engineers have created a stamp-sized wearable ultrasound patch that paces the heart noninvasively, eliminating the need for surgical pacemaker implantation. - The system combines a one-time gene therapy injection that sensitizes heart cells to sound with an external hydrogel patch that delivers targeted acoustic pulses. - In live rats with irregular heartbeats, the ultrasound patch safely restored steady rhythm for over eight months, and the technology was also successfully tested on pig hearts. - The approach, called sonogenetics, introduces RNA to make heart cells produce sound-sensitive MscL-G22S ion channels that trigger calcium influx and cardiac contraction when stimulated.
- MIT researchers have created a hydrogel-based oral formulation that coats the esophageal lining, enabling direct drug delivery to a historically difficult-to-target tissue. - The formulation combines a polysaccharide hydrogel with bile salts (sodium chenodeoxycholate and sodium cholate) that temporarily loosen cell-cell junctions to enhance drug permeability. - In animal studies, the gel successfully delivered infliximab, an anti-TNF-alpha antibody, directly to esophageal tissue, with cell junctions returning to normal within three days. - The platform aims to reduce systemic side effects associated with immunosuppressants like infliximab by enabling site-directed delivery for conditions such as eosinophilic esophagitis and Crohn's disease.
- Rafael Holdings' subsidiary Cyclo Therapeutics has entered into an exclusive licensing agreement with MIT covering U.S. Patent No.12285440 for cyclodextrin use in ApoE4-positive Alzheimer's disease patients. - The ApoE4 gene variant is present in 50-70% of Alzheimer's patients and is believed to be a key risk factor for early onset sporadic disease development. - Preclinical data suggest Trappsol® Cyclo™ can improve cholesterol delivery between CNS cells, reduce tau pathology, and enhance learning and memory in mice. - The company expects Phase 3 results from their Transport NPC trial in Q3 2026, representing the largest study ever conducted in Niemann-Pick disease patients.
- OS Therapies appointed Craig Eagle, MD, Chief Medical Officer of Guardant Health, and Robert "Bob" Langer, PhD, co-founder of Moderna, to its newly-formed strategic advisory board in April 2026. - The advisory board will guide regulatory execution for OST-HER2 in osteosarcoma and help prioritize the company's clinical pipeline across multiple cancer indications. - OST-HER2 demonstrated statistically significant benefits in 12-month event-free survival and overall survival in its Phase 2b trial for recurrent, fully resected lung metastatic osteosarcoma. - The company anticipates receiving a Biologics License Application from the FDA for OST-HER2 in 2026, which could make it eligible for a Priority Review Voucher valued at approximately $205 million.
- The Salk Institute has received up to $41.3 million from ARPA-H to advance sonogenetics, a novel approach using ultrasound to precisely control cells equipped with ultrasound-responsive proteins. - The multi-institutional collaboration will develop core biological tools, next-generation ultrasound delivery systems, and preclinical evidence to move sonogenetics into clinical trials for peripheral neuropathy. - SonoNeu, a spin-out company co-founded by General Inception, will serve as the commercial vehicle coordinating translation of the research into therapeutic candidates. - The technology offers potential for drug-free, noninvasive treatment by providing spatial and temporal precision in cellular control that could revolutionize pharmaceutical approaches.
