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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 208
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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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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.

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Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Conversations in Drug Development Trends

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.