University of Pennsylvania

Dr. Rimm's lab developed a high sensitivity HER2 assay in 2022, significantly improving accuracy and sensitivity. Despite initial limitations in availability, collaboration with Danaher, Leica Biosystems, and Cepheid aims to standardize and distribute the assay globally. A clinical trial, involving the assay and Cepheid's STRAT4, seeks to determine optimal HER2 thresholds for patient response to ADCs, emphasizing patient benefit over technology competition.

AACR focuses on cancer prevention and early detection, with Kornelia Polyak's pioneering research highlighting the importance of early-stage disease study. The AACR established the Cancer Prevention Working Group in 2021 to support innovative science and public education. Liquid biopsies and multi-cancer early detection (MCED) tests are being developed to improve screening. The NCI's National Cancer Plan and President Biden's proclamation of April 2024 as National Cancer Prevention and Early Detection Month emphasize the urgency of these efforts. Researchers are exploring liquid biopsies, MCED, cancer interception, and population science to advance early detection and prevention strategies.

Legend Biotech to open R&D center in Philadelphia, employing 55 staff, highlighting the city's growing biotech hub status.

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.

NCQA's 2025 credentialing updates include adjusted timelines and new requirements like license monitoring and social demographic data capture, emphasizing innovation and patient safety.

Mitchell J. Weiss, MD, PhD, chair of St. Jude Children’s Research Hospital Department of Hematology, elected to National Academy of Medicine (NAM) for research in blood cell formation and treatment development for sickle cell disease and beta-thalassemia. Weiss has published over 170 research papers and joined six other St. Jude faculty members in NAM.

Alexander Fanaroff discusses how behavioral economics, gamification, and financial incentives can increase physical activity. A study found that both gamification and financial incentives led to about 500 more steps per day, with a combination yielding 850 more steps. The intervention, guided by behavioral economic theory, showed effectiveness during its administration period.

Peter Marks discussed gene therapy advancements, including ex vivo and in vivo methods, CAR-T therapy, and potential for treating diseases like cystic fibrosis. Challenges include delivery efficiency, safety, manufacturing, and regulatory hurdles, but ongoing research shows promise.

Interius BioTherapeutics achieves milestone with first in vivo CAR therapy, INT2104, for B-cell malignancies, aiming to address supply and cost issues in CAR T cell therapy. The company plans to release early clinical data in 2025, focusing on safety before dose escalation. In vivo approach promises faster, cheaper, and potentially broader patient reach compared to ex vivo methods, though safety and efficacy data are still pending.

A $14 million NIH grant funds Penn and CHOP research on gene-editing therapies for rare metabolic diseases, focusing on urea cycle disorders. Using prime editing, a versatile CRISPR technology, the aim is to develop personalized treatments, potentially offering lifelong cures for conditions like type I citrullinemia, ASA lyase deficiency, and CPS1 deficiency.