Clinical Research Focus. 52nd Edition

CRISPR Moves from Breakthrough Technology to Clinical Reality

Recent advances in gene editing are showing a clear shift from scientific breakthrough to real clinical application. A new, compact CRISPR system is addressing long-standing delivery challenges, while recent Phase III results in in vivo gene editing demonstrate that these therapies are moving closer to real-world use. Together, these developments signal a turning point for gene therapy, where curative, one-time treatments are becoming increasingly viable across multiple diseases.

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FDA Flags 2,200+ Sponsors Over Missing Trial Results

The FDA has flagged over 2,200 sponsors for missing clinical trial results, highlighting a growing issue in how data is reported and perceived. As trial designs become more flexible and datasets more complex, gaps in reporting are becoming more visible and harder to ignore. This article explores what’s behind this trend and why transparency is starting to play a bigger role in how clinical programs are evaluated.

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New siRNA Therapy Reduces Triglycerides in Rare FCS Patients

EMA has recommended approval of Redemplo (plozasiran), a first in class siRNA therapy for adults with familial chylomicronaemia syndrome, a rare condition with limited treatment options. The drug demonstrated around 80% reduction in triglyceride levels and a lower risk of acute pancreatitis in clinical studies, offering a significant new approach for managing this high unmet need disease.

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A Breakthrough in One of the Deadliest Cancers? What Daraxonrasib Really Changes

Daraxonrasib is showing one of the most meaningful advances in pancreatic cancer in decades, with Phase III data demonstrating a near doubling of overall survival compared to standard chemotherapy. This article explores what truly makes these results important, from its next generation mechanism targeting RAS mutations to the broader implications for future treatment standards, and why the real impact may extend far beyond a single breakthrough.

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FDA Approves First Gene Therapy for Rare Hearing Loss

A new gene therapy has been approved for a rare genetic form of hearing loss, marking a major milestone for the field. The approval demonstrates that gene therapies are moving beyond experimental use into real clinical practice, validating years of research and opening the door for broader applications across other genetic diseases.

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From Data to Decisions: Lessons from 10+ Years in Clinical Biometrics 

Clinical trials are producing more data than ever, but success depends on how well that data is structured and analyzed. In this article, Kristina Leus shares key biometrics lessons from real studies, showing how early alignment, integrated teams, and strong data architecture help turn complex data into reliable, decision ready evidence.

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Big Pharma Doubles Down on AI with $1B Merck–Google Deal

Merck has signed a $1 billion agreement with Google to accelerate the use of AI across drug development, signaling a deeper shift in how therapies are discovered and developed. As pharma increasingly integrates advanced analytics into R&D, AI is moving from experimental use to a core strategic capability shaping the future of clinical development.

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AI Takes a Step Toward Autonomous Scientific Discovery in Oncology

A new agentic AI framework is redefining how cancer research can be conducted by autonomously generating and testing biologically meaningful hypotheses directly from pathology data. Unlike traditional models, the system can turn ideas into analytical tools without additional training, uncovering clinically relevant biomarkers and patterns across multiple cancer types. This approach signals a shift from AI as an analytical tool to AI as an active participant in scientific discovery.

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