Clinical Research Focus. 46th Edition

Nobel Laureate Unveils Technique to Turn Rogue T Cells into Their Own Treatment

Just weeks after receiving the 2025 Nobel Prize for discovering regulatory T cells, Shimon Sakaguchi and colleagues have published two Science Translational Medicine papers showing how disease-causing T cells can be reprogrammed into regulatory ones. The approach suppressed inflammation and autoimmune reactions in mouse models, paving the way for first-in-human trials by Osaka-based RegCell.

Learn more: Paper 1 | Paper 2

ESMO 2025: Notes from the Floor — What Will Really Change Oncology in 2026

At this year’s ESMO Congress in Berlin, over 35,000 oncology professionals gathered to explore data that will shape the next era of cancer research. Vlad Bogin, MD, FACP, CEO of Cromos Pharma, shares his expert takeaways from the meeting,highlighting breakthroughs that are poised to redefine oncology in 2026 and beyond. From ADCs entering the curative setting to radioligand therapies moving upstream, Dr. Bogin offers a clear perspective on what these advances mean for sponsors, investigators, and patients alike.

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FDA Launches National Priority Voucher Program to Accelerate Breakthrough Therapies       

The U.S. FDA announced the first nine recipients of its new Commissioner’s National Priority Voucher (CNPV) program, a pilot designed to fast-track drugs addressing major national priorities such as infertility, Type 1 diabetes, pancreatic cancer, and nicotine addiction. The vouchers shorten review timelines to 1–2 months and offer enhanced sponsor communication throughout development, marking a bold step toward faster access to high-impact therapies.

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EMA Recommends First Treatment for Non-Cystic Fibrosis Bronchiectasis

On October 17, 2025, the EMA’s CHMP recommended approval of Brinsupri (brensocatib), the first therapy for non-cystic fibrosis bronchiectasis. In a Phase III trial with 1,767 patients, Brinsupri reduced pulmonary exacerbations by 19% and delayed the first episode by 14 weeks, marking a major advance for patients with chronic lung disease.

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CRO Regulatory Affairs: Bridging Strategy, Compliance, and Speed

Bringing a new therapy to market is as much a regulatory journey as a scientific one. In our latest insight, Cromos Pharma explores how a proactive, globally integrated regulatory strategy,  spanning FDA, EMA, and CTIS frameworks, helps sponsors achieve faster approvals while maintaining full compliance.

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Regeneron’s Gene Therapy Restores Hearing in Children with Genetic Deafness

Regeneron has reported breakthrough results from its investigational gene therapy DB-OTO, which restored hearing in children born with profound genetic deafness. Most participants experienced significant and lasting improvements in hearing and speech, marking a major milestone for gene therapy in sensory disorders.

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Novartis to Acquire Avidity Biosciences, Expanding its RNA Therapeutics and Neuroscience Pipeline

Novartis has signed a $12 billion deal to acquire Avidity Biosciences, gaining its late-stage RNA programs for rare neuromuscular diseases and the AOC™ platform for targeted RNA delivery. The acquisition, expected to close in early 2026, will strengthen Novartis’ neuroscience pipeline and accelerate its RNA medicine strategy.

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Early Neuroprotection Signal in Stroke: IGF-1–Based “Designer Drug” Shows Promise in Phase 2a

At WSC 2025 (Barcelona), Scp776—an IGF-1–engineered “designer drug” that targets injured brain tissue—showed early signs of benefit in acute ischemic stroke patients treated up to 24 hours after onset (LVO cohort). In the ARPEGGIO Phase 2a trial, Scp776 trended toward better NIHSS at 7 days and improved mRS at 90 days vs placebo, with a favorable safety profile. While not statistically significant in this small study, the signal supports plans for a Phase 2b/3 program in 2026.

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From Numbers to Meaning — Sensitivity, Specificity & Predictive Value

At Cromos Pharma, we’re continuing our Pharma Lingo series — short explainers that unpack key concepts in clinical research and pharma.In this episode, we take a closer look at sensitivity, specificity, and predictive values, essential measures for understanding diagnostic accuracy and making informed clinical decisions. Watch the video to refresh your knowledge and share it with your colleagues!

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