Clinical Research Focus. 47th Edition

Researchers Pinpoint New Antigen Targets in ‘Needle-in-a-Haystack’ Study to Advance Tuberculosis Vaccine Development

A team of scientists has used advanced molecular “needle-in-a-haystack” techniques to uncover previously hidden antigens displayed on human cells infected with Mycobacterium tuberculosis. These findings provide valuable new targets for designing more effective tuberculosis vaccines, offering hope for improved protection against a disease that still kills roughly 4,000 people every day worldwide

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Five Insights Changing Alzheimer’s Care in 2025

Alzheimer’s care is entering a new era. In 2025, breakthroughs such as blood-based diagnostics, next-generation antibody therapies, AI-driven prediction tools, viral-trigger research, and cutting-edge neural implants are reshaping how we detect, treat, and understand the disease. Instead of inevitability, the field is now defined by innovation, earlier intervention, and more personalized care — bringing new hope to millions of patients and families worldwide.

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FDA Tightens Safety Measures and Narrows Eligibility for Elevidys After Reports of Fatal Liver Complications

The FDA has added a Boxed Warning, its most serious type of safety alert, to the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) and restricted its approved use to ambulatory patients aged 4 and older with Duchenne muscular dystrophy (DMD), after reports of fatal acute liver failure in non-ambulatory patients. The move follows earlier safety communications in June 2025 about two deaths in non-walking DMD patients treated with Elevidys and the manufacturer’s voluntary suspension of its use in that population.

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Cancer Vaccines 2025, Part I: The mRNA Revolution

mRNA vaccines are rapidly transforming oncology. In 2025, personalized and off-the-shelf mRNA platforms are showing strong clinical signals across multiple cancers, marking the start of a new era in precision immunotherapy. This article explores how mRNA vaccines work, why they matter now, and what key trials reveal about the future of cancer treatment.

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EMA Endorses First Gene Therapy for Wiskott–Aldrich Syndrome, Marking Major Breakthrough for Rare Immune Disorder

The EMA has recommended EU approval of Waskyra, a one-time autologous gene therapy for patients with Wiskott–Aldrich syndrome who lack a suitable stem-cell donor. Clinical data showed sharp reductions in severe infections and bleeding episodes, supporting its use for this life-threatening immunodeficiency.

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FSP Model in Clinical Trials — Everything Sponsors Need to Know

As clinical trials become more complex, sponsors are increasingly turning to the Functional Service Provider (FSP) model — a flexible, scalable approach that preserves full control of systems and data while providing specialized expertise. With faster start-up, predictable costs, and integrated teams, FSP is quickly becoming the core operating model for modern clinical development.

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Roche’s Oral SERD Giredestrant Achieves First-Ever Adjuvant Success in Early HR+ HER2- Breast Cancer Trial

In a landmark Phase III trial, Roche announced that giredestrant significantly improved invasive disease-free survival compared with standard endocrine therapy in early-stage estrogen receptor-positive, HER2-negative breast cancer patients following surgery. This makes it the first oral selective estrogen receptor degrader (SERD) to demonstrate a benefit in the adjuvant setting, opening the door for a potential shift in post-surgical treatment for this subtype of breast cancer.

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Novartis’ GanLum Shows ~97% Cure Rate in Phase III Malaria Trial, Poised as First New Treatment in Decades

In a major advance against malaria, Novartis reported that its novel therapy combination GanLum (ganaplacide + lumefantrine) achieved a ~97 % cure rate in a Phase III trial of 1,688 adults and children across 12 African countries, outperforming the standard therapy and demonstrating strong activity against drug-resistant parasites. The Swiss pharma company now plans to seek regulatory approvals for what could be the first new antimalarial in more than 20 years.

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