University of Pennsylvania

Ben Samelson-Jones presented long-term safety data for Pfizer's Beqvez (fidanacogene elaparvovec), an AAV-based gene therapy for hemophilia B, at the 66th ASH Annual Meeting. The analysis, covering 60 patients over 3-6 years, showed no thrombotic complications, factor IX inhibitors, or malignancies. Adverse events were mild and unrelated to the vector, with infusion reactions absent. Challenges in gene therapy uptake include its one-time treatment nature and high cost, requiring different institutional processes and approvals.

Experts at Asia Economic Dialogue stressed the need to address vaccine hesitancy and global vaccine inequity, highlighting India's role as a major vaccine manufacturer. They emphasized greater government engagement with manufacturers and community education for vaccination, noting India's rapid vaccine development and regulatory efficiency. The discussion also touched on future pandemic preparedness and the utilization of current vaccine production capacities.

Recent medical breakthroughs include CRISPR therapy for sickle cell anemia, stem cell treatment restoring vision, artificial ovaries for fertility, CAR T-cell therapy for brain cancer, and gene therapy enabling a deaf child to hear.

OU Health Sciences leads national trial on a device aiming to help PCOS-related infertility through less invasive ablation. The device, used with transvaginal ultrasound probes, delivers pre-calculated electrothermal energy to ovaries. The REBALANCE trial, co-led with U Penn, will enroll 195 PCOS patients to study safety, effectiveness, and pregnancy rates.

A consortium, funded by a $30 million NINDS grant, plans human trials of PET radiotracers to detect toxic proteins like alpha-synuclein and 4-repeat tau, aiding in diagnosing and monitoring Parkinson’s and similar diseases. The project involves multiple universities and uses a computational tool to identify effective tracers.

Soligenix opens patient enrollment for FLASH2, a Phase 3 study evaluating HyBryte™ for cutaneous T-cell lymphoma (CTCL). The study builds on previous successful trials and aims to demonstrate HyBryte™'s efficacy over 18 weeks of continuous treatment.

Soligenix opens patient enrollment for FLASH2 Phase 3 study evaluating HyBryte™ for cutaneous T-cell lymphoma (CTCL), building on previous successful studies. The study aims to demonstrate HyBryte™'s efficacy over 18 weeks of continuous treatment, with interim analysis expected in early 2026.

Soligenix opens patient enrollment for FLASH2 Phase 3 study evaluating HyBryte™ for cutaneous T-cell lymphoma (CTCL), building on previous successful studies. The study aims to demonstrate HyBryte's™ efficacy over an 18-week continuous treatment period, with enrollment expected in the U.S. and Europe, and a formal interim analysis anticipated in early 2026.

Prevention is crucial in cancer research, with vaccines and therapies like tamoxifen showing efficacy. Recent advancements focus on vaccines, dose optimization, and RANKL pathways for breast cancer prevention in BRCA1/2 carriers. Ongoing trials explore vaccine efficacy for colon and breast cancers, while RANKL inhibition aims to reduce risk in high-risk populations.

FEVR is an inherited retinal disease often misdiagnosed due to symptom overlap with ROP, Coats disease, and PFV. Genetic testing confirmed FEVR in 55 of 104 pediatric cases, revealing 60% had initial incorrect diagnoses. Fluorescein angiography and OCT aid differentiation, highlighting key imaging distinctions. Genetic testing, though not always conclusive, is crucial for accurate diagnosis, guiding treatment, and family counseling.