University of Pennsylvania

A $14M NIH grant funds Penn and CHOP research on CRISPR-based gene-editing therapies for urea cycle disorders, aiming to develop personalized treatments using prime editing technology, which could offer lifelong cures for rare metabolic diseases.

Calluna Pharma appoints Mark Gaffney as CEO; Gaffney brings 20+ years of experience in biotech, previously CEO of Oxular Limited and COO at Vedere Bio (acquired by Novartis). Gaffney will lead Calluna's strategy as CAL101 completes Phase 1 this year.

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.

KDU731, a compound inducing micronuclei in vitro and in vivo, was modified to reduce genotoxicity by substituting cyanopyridine with chlorobenzene, retaining potency. EDI048 and other soft-drug analogues were synthesized with chlorobenzene/trifluoro-oxy-benzene substitution. The article details the synthesis, analytical data, and biological assays of these compounds, including their effects on Cryptosporidium species and in vitro activity assessments.

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).