Clinical Trials
486
28 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1855
Active, not recruiting
20
4.1%
Approved For Marketing
2
0.4%
Available
2
0.4%
Completed
324
66.7%
Enrolling By Invitation
6
1.2%
Not yet recruiting
2
0.4%
Recruiting
74
15.2%
Suspended
1
0.2%
Terminated
35
7.2%
Unknown
2
0.4%
Withdrawn
18
3.7%
No approval data available
- Pennsylvania health officials reported four breakthrough measles cases in fully vaccinated residents, the first such cases in the state's 2026 outbreak. - The state's outbreak has reached 676 cases with 124 hospitalizations, making it Pennsylvania's largest measles outbreak in more than 30 years. - MMR vaccination demand surged in August, with providers administering 46,000 doses versus the typical 25,000 per month. - Two measles-associated infant deaths have been reported, though state and federal officials disagree on whether both should be linked to the outbreak.
- Penn Medicine has received a $50 million gift from businessman Stanley C. Middleman to support early-stage research into cancer, autoimmune diseases and infectious diseases. - The donation establishes the Middleman Fund and renames Penn's expanded 3600 Civic Center Blvd. building as the Middleman Center, housing the Colton Center for Autoimmunity. - The 217,000-square-foot, $376 million expansion added seven floors with as many as 37 labs, plus a High-Throughput Institute for Discovery and a Biosafety Level 3 lab. - Penn aims to shorten the path from laboratory discovery to market, building on its pioneering work in CAR T cell therapy and mRNA research.
- The V Foundation for Cancer Research awarded nearly $2 million to Children's Hospital of Philadelphia, marking its largest single-year grant to the institution since 2010. - Andrei Thomas-Tikhonenko, PhD, received a $1 million Pediatric All-Star grant to develop P2RX5-targeted CAR T-cell therapy for T-cell acute lymphoblastic leukemia (T-ALL). - Suzanne MacFarland, MD, received an $800,000 Pediatric Translational grant to pilot a blood-based liquid biopsy for earlier cancer detection in children with Li-Fraumeni syndrome. - The P2RX5 work may address disparities in cancer outcomes, as P2RX5-positive leukemias are more prevalent among children of African and Hispanic ancestry.
- Children's Hospital of Philadelphia received up to $38.9 million over five years to develop personalized gene-editing therapies for four groups of rare liver-related genetic disorders, including urea cycle disorders and hemophilia A. - The Jackson Laboratory and Broad Institute secured up to $34.5 million for the PERC platform targeting pediatric epilepsies, initially focusing on alternating hemiplegia of childhood and Dravet syndrome. - Both awards come through ARPA-H's THRIVE initiative, which aims to transform individualized gene-editing approaches into scalable, repeatable platforms for rare disease treatment. - The programs will leverage base editing, prime editing, and lipid nanoparticle delivery systems while addressing regulatory, manufacturing, and access challenges for ultra-rare conditions.
- Federal investment in basic science through NIH and NSF built the foundation for GPS, the internet, CRISPR, mRNA vaccines, and CAR-T therapies, with no short-term commercialization mandate. - NIH funding has declined as a share of GDP, with inflation-adjusted budgets below 2003 peaks, narrowing the discovery pipeline and threatening U.S. scientific leadership. - Every dollar invested in NIH research generates $2.57 in economic activity, driving over $822 billion in new economic activity over the last decade and supporting 370,000–390,000 jobs annually. - China, Europe, and the UK are increasing basic research investments while the U.S. trajectory points downward, risking an exodus of talent and innovation overseas.
- The Department of Health – Abu Dhabi and Children's Hospital of Philadelphia signed an MoU at the BIO International Convention to advance gene editing capabilities for rare diseases. - CHOP will provide specialized training to Abu Dhabi clinician-scientists in base-editing therapies targeting liver metabolic disorders, genetic clotting disorders, urea cycle disorders, and CNS conditions. - The collaboration leverages Abu Dhabi's nearly one million sequenced genomes through the Emirati Genome Programme to translate genomic insights into population-scale precision medicine. - CHOP recently demonstrated the potential of personalized gene editing by creating the world's first such therapy for a baby with a rare metabolic disease, targeting up to 700 inherited liver conditions.
- Researchers at Children's Hospital of Philadelphia developed VLP-Open HLA, a virus-like nanoparticle platform that mimics diseased cell surfaces to screen cancer targets rapidly. - The platform can find and activate CD8+ T cells that recognize specific cancer targets in both cell lines and human blood samples without weeks-long cell culturing. - The system works across diverse HLA protein variants and can be configured to carry multiple immune signals, enabling personalized cancer vaccines and T cell therapies. - The study, published in Science Advances on June 12, 2026, was supported by Cancer Grand Challenges, NCI, and The Mark Foundation for Cancer Research.
- Three Philadelphia scientists received the 2026 Breakthrough Prize for developing Luxturna, the first FDA-approved gene therapy for inherited genetic disease, specifically targeting Leber congenital amaurosis caused by RPE65 gene mutations. - The therapy delivers a functional copy of the RPE65 gene directly into patients' eyes using a viral vector, improving vision in 72% of trial participants who showed maximum improvement in low-light conditions. - Their groundbreaking work established the scientific and regulatory framework for gene therapy development, paving the way for more than 140 retinal gene therapy trials and over a dozen similar FDA approvals. - The collaboration between Jean Bennett, Albert Maguire, and Katherine High demonstrates how interdisciplinary expertise in molecular biology, ophthalmology, and clinical development can transform laboratory discoveries into life-changing treatments.
- A landmark clinical trial involving 1,658 participants across six U.S. academic centers found that a structured behavioral hydration program using smart bottles and coaching failed to significantly reduce kidney stone recurrence rates. - Despite participants modestly increasing their urine output through the intervention, the improvement was insufficient to prevent symptomatic stone recurrence compared to standard care over the two-year study period. - The findings highlight the significant challenge patients face in maintaining the high fluid intake necessary for kidney stone prevention, even with technological support and financial incentives. - Researchers suggest the need for more personalized approaches to kidney stone prevention, as a single hydration target may not work for all patients due to varying individual factors.
- Arctic Therapeutics has enrolled the first patients in a Phase IIa randomized, double-blind, placebo-controlled trial evaluating topical AT-004 for acne vulgaris treatment. - AT-004 represents a first-in-class acetylcholinesterase inhibitor targeting the non-neuronal cholinergic anti-inflammatory pathway to modulate TNF-α and reduce skin inflammation. - The 60-participant study will assess safety and efficacy over 12 weeks across three Danish clinical sites, with potential applications extending to other inflammatory skin conditions. - Acne vulgaris affects an estimated 650 million people worldwide and significantly impacts quality of life, contributing to anxiety, reduced self-esteem, and depression.
