University of Pennsylvania

Philly biotech Adaptimmune prepares to produce its first doses of a novel cancer treatment, Tecelra, for synovial sarcoma. Pennsylvania faces the nation's largest whooping cough outbreak, and local hospitals grapple with IV fluid shortages due to Hurricane Helene. Philadelphia sees a drop in overdose deaths but racial disparities in mortality are widening.

Most vendors of synthetic nicotine vapes on Instagram ignore FDA health warnings, potentially exposing kids to harmful products. Synthetic nicotine, not derived from tobacco, evaded FDA regulation until 2022, allowing flavored products appealing to youth. Despite FDA mandates, only 13% of Instagram ads for synthetic nicotine vapes comply with health warning requirements.

Persephoni BioPartners appoints Dr. Donna Taraborelli as COO to support early-stage biotech startups. Taraborelli, with extensive experience in life sciences, previously contributed to Clearside Biomedical's IPO and FDA approval. Persephoni aims to accelerate breakthrough technologies through its venture studio model.

Beqvez (fidanacogene elaparvovec) gene therapy for hemophilia B results in stable FIX production, reducing bleeding episodes by about three times compared to prophylaxis. A single dose of Beqvez was well-tolerated, with a 71% drop in annual bleeding rates and 92% decrease in FIX consumption. Mean FIX activity at 15 months was 26.9%, though 62% of patients needed glucocorticoids for elevated liver enzymes.

Kimberlyn Ellis, a graduate student in Human Genetics, has been selected as the 2024 Vanderbilt Prize Student Scholar, receiving a $1,000 cash prize and mentorship from Sarah Tishkoff, PhD. Ellis aims to bridge genomics, bioethics, and health policy for equitable precision medicine, focusing on chronic disease in populations with significant African ancestry.

Precision Neuroscience’s Layer 7 Cortical Interface, with 1,024 electrodes, captures unprecedented neural patterns in motor cortex, revealing beta wave dynamics during movement, potentially revolutionizing BCI technology and neurological treatments.

Soligenix presents HyBryte™ therapy findings at EORTC meeting, showing improved response rates with extended treatment and increased light doses in cutaneous T-cell lymphoma (CTCL).

Penn engineers use lipid nanoparticles (LNPs) to target specific tissues, enhancing gene therapy and personalized medicine. Incorporating siloxane composites into LNPs, known as SiLNPs, significantly improves mRNA delivery, with some variants achieving up to 90% success in targeting lung tissue. SiLNPs also show potential in regenerative medicine and CRISPR-Cas-based gene editing.

Soligenix partners with Sterling Pharma Solutions to optimize synthetic hypericin production for HyBryte™ and SGX302, targeting CTCL and psoriasis. The collaboration aims to scale up manufacturing for clinical trials and commercialization, with a focus on reducing costs and advancing clinical programs.

Gene therapy shows promise for treating retinal diseases, but faces challenges like immune response and gene integration risks. Advances in delivery systems and viral vectors like adeno-associated viruses have improved efficacy, though nonviral methods are also explored. Clinical trials report improvements in visual function for inherited retinal diseases, but acquired diseases like glaucoma and age-related macular degeneration present different treatment needs. Regulatory approval and cost-effectiveness remain significant hurdles.