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Future-proofing drug development with GenAI

Drug Target Review

Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This has led drug developers to unintentionally limit their potential within chosen therapeutic spaces.

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NEJM paper fills in details on ‘remarkable’ CAR-T result in autoimmune disease

BioPharma Drive: Drug Pricing

The full results spotlight cell therapy’s potential to treat lupus and other inflammatory conditions, but also the problems drug developers must solve first.

Disease 312
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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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First multi-chamber heart organoids unravel human heart development and disease

Science Daily: Pharmacology News

Heart disease kills 18 million people each year, but the development of new therapies faces a bottleneck: no physiological model of the entire human heart exists -- so far.

Disease 201
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BioMarin to replace CEO Bienaimé with Genentech veteran

BioPharma Drive: Drug Pricing

The rare disease drug developer is turning to former Genentech head Alexander Hardy as the launch of its Roctavian gene therapy stumbles.

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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. For years, AI and machine learning (ML) were often dismissed as little more than advanced statistics with little practical value in drug development.

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Overcoming Orphan Drug Development Challenges with Real-World Data and Evidence

PPD

While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.