VANDERBILT UNIVERSITY MEDICAL CENTER

ATH434 shows potential as a disease-modifying therapy for MSA, with 30% of participants in Phase 2 trials showing stable or improved clinical outcomes and biomarkers.

Eight potential human bird flu cases reported in Missouri, with antibody tests pending. If confirmed, it could indicate the virus is spreading among humans, raising pandemic concerns.

Poseida Therapeutics announced interim clinical data from its Phase 1 trial of P-BCMA-ALLO1 in relapsed/refractory multiple myeloma, showing a 91% overall response rate (ORR) in an optimized lymphodepletion arm, with 100% ORR in BCMA-naïve patients and 86% ORR in those previously treated with BCMA-targeting therapies. The therapy demonstrated no dose-limiting toxicities, low rates of CRS and ICANS, and no GvHD or Parkinsonism. P-BCMA-ALLO1 has been granted RMAT designation by the FDA and is being evaluated in a Phase 1/1b clinical trial.

Capricor Therapeutics plans to file a Biologics License Application (BLA) in October 2024 for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, based on existing cardiac and natural history data. The initial label will include all DMD cardiomyopathy patients, with potential label expansion for skeletal muscle myopathy post-approval.

Vanderbilt University Medical Center received $12 million in NIH grants to develop a genomic-enabled learning health system (gLHS) network, aiming to integrate genomic data and clinical informatics to improve precision medicine. The network, including a coordinating center called DRAGON, will focus on diverse settings and underserved populations, with potential projects like integrating family health histories into EHRs and implementing personalized genomic reference ranges for lab tests.

Capricor Therapeutics plans to file a Biologics License Application (BLA) for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, based on existing cardiac and natural history data. The BLA submission is expected by end of 2024, with a rolling submission starting October 2024. Deramiocel has shown potential in clinical trials to attenuate DMD's cardiac implications.

NIH awards $27M to establish a Genomics-enabled Learning Health System (gLHS) Network, aiming to integrate genomic information into patient care. The network includes six clinical sites and a coordinating center, focusing on developing and refining genomic medicine practices. The initiative seeks to create generalizable knowledge and tools for broader patient care, including under-resourced settings.

A second health care worker exposed to a Missouri bird flu patient developed respiratory symptoms but wasn't tested, raising concerns about person-to-person transmission of H5N1. The CDC is investigating and will perform serological testing on the patient and household contact to determine if they have antibodies for the virus.

Whooping cough cases in the U.S. have surged, more than quadrupling since last year, due to missed vaccinations during the pandemic. Vaccines (DTap, TDap) are crucial for protection. Symptoms mimic common colds, complicating early diagnosis. Severe cases affect infants most, with vaccination recommended for pregnant individuals to protect newborns.