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The rising impact of biomarkers in early clinical development

Drug Target Review

Haemoglobin A1c (HbA1c) is a validated surrogate endpoint for the reduction of microvascular complications associated with diabetes mellitus; reduced HIV-RNA levels serve as an endpoint for HIV disease control; and a reduction in low-density lipoprotein (LDL) cholesterol is used as an endpoint indicating lower likelihood of cardiovascular events.

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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).

RNA 59
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A Visual Guide to Genome Editors

Codon

The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. This article provides a summary of the major CRISPR systems, 2 including the naturally occurring CRISPR-Cas9, -Cas12, and -Cas13 systems, as well as base editors, prime editors, and the recently uncovered bridge RNA system.

DNA 83
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GRouNdGAN: GRN-guided simulation of single-cell RNA-seq data using causal generative adversarial networks

Broad Institute

Through the imposition of a user-defined GRN in its architecture, GRouNdGAN simulates steady-state and transient-state single-cell datasets where genes are causally expressed under the control of their regulating transcription factors (TFs).

RNA 40
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Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

BRAF and downstream mitogen-activated extracellular signal-regulated kinase (MEK) inhibitors are combined to treat BRAF-mutated melanoma, for example. Inhibiting KDM5A, a regulator of cell proliferation, poses another interesting approach.

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Cancer immunotherapy: GDF-15’s role in Anti-PD-1 resistance

Drug Target Review

Her discoveries on the first-in-class RNA-based innate immune receptor agonist RGT100, built the foundation for the initiation of a Phase I clinical trial as well as the successful acquisition of Rigontec by MSD (known as Merck & Co. 2016 Oct 10;7(1):13119. in North America) in late 2017. Nature Communications. Walling BL, Kim M.

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SRP-001: redefining pain treatment with a safer, non-opioid analgesic

Drug Target Review

SRP-001 targets the central nervous system (CNS) by producing N-arachidonoylaminophenol (AM404) in the midbrain’s periaqueductal grey (PAG) region, crucial for pain sensation and regulation. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain. South Rampart Pharma, Inc.