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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My

Disease 80
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RNA processing in health and disease: challenges and opportunities of the field

Drug Target Review

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

RNA 96
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The next phase of the multiomics evolution, powered by AI

Drug Target Review

Understanding health and disease requires more than reading the genomic code. The result is a more holistic, actionable view of cellular and tissue dynamics that is essential for uncovering causal mechanisms in disease. Biology, however, presents a unique challenge: it is deeply contextual, dynamic and multilayered.

RNA 71
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Sanofi Showcases Rare Disease Pipeline Innovations at ISTH

The Pharma Data

At this premier global meeting focused on thrombosis, hemostasis, and bleeding disorders, Sanofi will present compelling data across 18 scientific abstracts, including five oral presentations. In addition, two poster presentations will offer deeper insights into the LUNA 3 trial.

Disease 40
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The rising impact of biomarkers in early clinical development

Drug Target Review

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.

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Decoding stem cells for personalised regenerative medicine

Drug Target Review

“We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun. Scrutinising these distinctions aids scientists in comprehending cellular operations, thereby fostering enhanced therapeutic approaches for diverse diseases.

RNA 128
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Therapeutic Oligos 2025 Keynote Speakers Announced

Elrig

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

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