CHILDREN'S HOSPITAL OF PHILADELPHIA

J&J submits sBLAs for guselkumab to treat moderate-to-severe plaque psoriasis in children aged 6+ and active juvenile psoriatic arthritis in children aged 5+. Submissions based on phase 3 studies and PK data, aiming to address treatment gaps in pediatric patients.

CHOP's CelloType AI model expedites spatial omics data analysis, enhancing cell detection, segmentation, and classification for precise diagnostics and targeted treatments across complex diseases like cancer and chronic kidney disease. The model integrates segmentation and classification tasks, outperforming traditional methods, and is available for noncommercial use.

Rocket Pharmaceuticals' RP-A501 gene therapy for Danon disease showed sustained LAMP2 protein expression and reduced LV mass index in patients up to 60 months post-treatment, with generally well-tolerated safety profile.

AMCs face AI challenges in culture, governance, leadership, funding, scale, and speed, but AI offers opportunities to enhance research and clinical care. Key steps include aligning resources, establishing new governance structures, and developing AI leadership. AMCs must balance AI initiatives across productivity, technology adoption, and innovation creation.

Gene therapy for Danon disease improved or stabilized heart structure and function in 6/7 patients over 24-54 months, with no major safety concerns. The treatment, RP-A501, involved a single dose of AAV9 vector carrying the LAMP2B transgene, showing promising results in halting disease progression and improving quality of life.

Becker's 2024 list highlights hospitals & health systems excelling in oncology, recognized for advanced clinical trials & research. Notable institutions include AdventHealth, Allina Health, Arkansas Children's, Atlantic Health System, Banner MD Anderson Cancer Center, Baylor Scott & White Health, Boston Medical Center, Brown University Health, Children's Health, Children's Hospital Colorado, Children’s Hospital Los Angeles, Children’s Hospital of Orange County, Children's Hospital of Philadelphia, Children's Nebraska, ChristianaCare, City of Hope Comprehensive Cancer Center, Cleveland Clinic, Connecticut Children’s, Covenant Health, Dana-Farber Cancer Institute, Dartmouth Health, DeTar Hospital Cancer Center, Duke Health, El Paso Children's Hospital, Emory Healthcare, Endeavor Health, Fred Hutchinson Cancer Center/UW Medicine, Geisinger, Hackensack Meridian Health, Huntsman Cancer Institute at the University of Utah, Inova, Jefferson Health, Johns Hopkins, Jupiter Medical Center, Keck Medicine of USC, Laredo Medical Center, Lehigh Valley Health Network, MUSC Health, MaineHealth, Massachusetts General Hospital, Mayo Clinic, MedStar, MemorialCare, Memorial Sloan Kettering Cancer Center, Michigan Medicine, Mount Sinai Health System, Nebraska Medicine, New York-Presbyterian Hospital-Columbia and Cornell, Northwestern Medicine, Novant Health, OSF HealthCare, OU Health, Ochsner Health, Ohio State University Comprehensive Cancer Center, Oregon Health & Science University Hospital, Orlando Health, Penn Medicine, Providence Alaska Medical Center, Providence Cancer Institute of Oregon, Providence South Division Cancer Institute, Queen's Health System, RWJBarnabas Health and Rutgers Cancer Institute, Renown Health, Rochester Regional Health, Roper St. Francis Healthcare, Roswell Park Comprehensive Cancer Center, Sanford Health, Sarasota Memorial Health Care System, Scripps Health, St. Jude Children's Research Hospital, Stanford Medicine, Stanford Medicine Children's Health, Sutter Health, Swedish Cancer Institute, Sylvester Comprehensive Cancer Center at University of Miami, Tampa General Hospital, Texas Children's Hospital, UC Davis Health, UCLA Health, UCSF Health, UK HealthCare, UNM Health System, UVA Health, UW Health, UMass Memorial Health, University of Alabama Birmingham Hospital, University of Chicago Medical Center, University of Colorado Cancer Center-UCHealth University of Colorado Hospital, University Hospitals, University of Iowa Health Care, University of Kansas Health System, University of Texas MD Anderson Cancer Center, University of Vermont Health Network, Vanderbilt University Medical Center, Virtua Health, WVU Cancer Institute, WellSpan Health, Yale New Haven Health.

Caris Life Sciences' MI Cancer Seek, an FDA-approved companion diagnostic, identifies cancer patients eligible for targeted therapies using simultaneous Whole Exome and Whole Transcriptome Sequencing.

CAR-T therapy, while effective for hematologic malignancies, has unique safety concerns like neurotoxicity (ICANS). A study in *Blood* found ICANS in children not linked to inflammation, prompting a need to consider neurotoxicity cases uniquely. First author Caroline Diorio emphasizes the importance of understanding the different pathophysiologies of these cases.

CHOP's D3B received $10M from ARPA-H for RADIANT, a project using real-time clinical data analysis to combat rare diseases, aiding informed treatment decisions and clinical trial possibilities.

Recursion's AI-driven drug REC-3964, an oral, non-antibiotic small molecule, has dosed its first patient in Phase II ALDER trial for recurrent Clostridioides difficile infection. REC-3964 targets toxin B's glucosyltransferase activity, differing from antibiotics by preserving gut microbiome. The trial aims to assess safety, tolerability, and efficacy, with results expected in Q4 2025. RecursionOS, the AI platform, accelerates drug development, reducing time and cost.