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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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Covalency in PROTACs: Mechanisms and applications [@RPNowak]

Covalent Modifiers

Nowak Annual Reports in Medicinal Chemistry , 2024 [link] Proteolysis targeting chimeras (PROTACs) are hetero-bifunctional molecules that remove disease-causing proteins through the means of targeted protein degradation (TPD). Geiger, Radosław P.

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A transatlantic collaboration is uncovering the role of key genetic variants in common diseases like type 2 diabetes and obesity

Broad Institute

By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.

Disease 72
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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog: Drug Discovery

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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Open Targets Platform 25.06 has been released!

The Open Targets Blog

integrated their GWAS findings with functional genomics data from relevant tissues, identifying 700 genes with high confidence of being involved in osteoarthritis, and eight biological processes key to the disease development, including circadian clock and glial cell functions. GWAS credible sets from Hatzikotoulas et al. GCST90566795 ).

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Researchers map communities of single cells in metastatic breast cancers

Broad Institute

Researchers map communities of single cells in metastatic breast cancers By Corie Lok October 30, 2024 Breadcrumb Home Researchers map communities of single cells in metastatic breast cancers The team compared different single-cell and spatial profiling methods to explore the diversity of cells in metastatic breast cancer biopsies.

Research 127
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The biotech mapping thousands of hidden therapeutic clues

Drug Target Review

“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. Today’s broad-spectrum drugs often miss the mark, especially for chronic diseases like COPD or heart failure.

Disease 59