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GSK to buy liver disease drug for $1.2B

BioPharma Drive: Drug Pricing

The new acquisition from Boston Pharmaceuticals adds to a wave of dealmaking undertaken by GSK to build its liver disease franchise.

Disease 265
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How integrated real-world data helps identify and improve outcomes for rare disease patients

BioPharma Drive: Drug Pricing

Pharmaceutical companies are turning to integrated RWD approaches to overcome challenges in rare disease research.

Disease 278
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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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Startup AIRNA emerges from stealth to bring RNA editing to a rare disease

BioPharma Drive: Drug Pricing

AIRNA is one of several biotechs to debut plans for target alpha-1 antitrypsin deficiency in recent years, with competitors such as AlveoGene, Wave Life Sciences and Arrowhead Pharmaceuticals.

RNA 317
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How AI will reshape pharma by 2025

Drug Target Review

Artificial intelligence (AI) has revolutionised many industries, yet its adoption in pharmaceutical drug development has been notably slower. Looking ahead, 2025 could represent a major turning point for the pharmaceutical sector.

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‘No tolerance for failure’: An oral history of the first CRISPR medicine

BioPharma Drive: Drug Pricing

A new sickle cell disease therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals is now approved in the U.S. This is the story of how it came to be.

Therapies 357
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How GPCR-targeting therapies are advancing the fight against inflammatory disease

Drug Target Review

Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. This makes conventional approaches, which attempt to target the orthostatic binding sites, less effective.

Disease 59