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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 315
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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 204
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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.

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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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Macrophage cell therapy: a new hope for chronic liver disease patients

Drug Target Review

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

Therapies 116
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Key Trends Drug Developers Need to Know to Succeed

PPD

As the pharmaceutical industry continues to evolve, drug developers encounter new challenges and opportunities in their pursuit of innovation. In response, the PPD clinical research business of Thermo Fisher Scientific conducted its third global survey of 150 drug developers to capture a comprehensive view of these evolving trends.