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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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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52
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NGS is evolving: collaboration and tech lead the way

Drug Target Review

The latest technologies are empowering researchers with faster, more efficient and more accessible genomic tools, leading to richer data generation and accelerating progress in areas like cancer genomics, rare disease research and infectious disease surveillance.​ 2021 [cited 2025 Jun 11]. 2021 [cited 2025 Jun 11].

RNA 92
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David Liu receives Breakthrough Prize in Life Sciences

Broad Institute

The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.

Science 144
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From siloed data to breakthroughs: multimodal AI in drug discovery

Drug Target Review

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Nature, 596, 590-596 (2021) [link] Liu R, Rizzo S, Waliany S, et al.

Drugs 65
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Oncology advances through the lens of women in STEM

Drug Target Review

Despite challenges as a woman in the field, Dr Hingorani built a career focused on advancing treatments for pediatric and adult cancers. Despite the progress we are making in curing children with cancer, there remains an unmet need for new treatments that are more effective and safer.

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A spatial approach to understanding drug dynamics using mass spectrometry imaging

Drug Target Review

Multiomics MSI studies provide crucial insights into drug pharmacology and toxicity, supporting the development of innovative therapies including chimeric antigen receptor (CAR) T-cell treatments, oligonucleotide therapeutics, antibody drug conjugates (ADCs) and ribonucleic acid (RNA) therapeutics. 2021, 56 (8), e4717. Mass Spectrom.

Drugs 52