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The rising impact of biomarkers in early clinical development

Drug Target Review

Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms. A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.

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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog: Drug Discovery

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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New cell therapy model accelerates cancer treatment development

Drug Target Review

Ive been involved in therapeutics development for over 25 years, working with small, medium, and large biotech companies. My focus has always been on advancing novel medicines from research to clinical development. TILs were specifically developed to target solid tumour cancers, Bock explained.

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Challenges in Clinical Development of Products for Rare Disease

thought leadership

There are over 7,000 rare diseases affecting more than 30 million people in the United States, and despite the FDA's approval of over 600 treatments for rare diseases since signing the Orphan Drug Act into law in 1983, most rare diseases still do not have a treatment.

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Discovery and Preclinical Characterization of BIIB129, a Covalent, Selective, and Brain-Penetrant BTK Inhibitor for the Treatment of Multiple Sclerosis

Covalent Modifiers

BIIB129 (25) demonstrated efficacy in disease-relevant preclinical in vivo models of B cell proliferation in the CNS, exhibits a favorable safety profile suitable for clinical development as an immunomodulating therapy for MS, and has a low projected total human daily dose.

Treatment 208
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Harnessing AI and Real-World Data: The Future of Clinical Development

PPD

ML is also used to identify predictors of response to a therapy or risk for adverse events (AEs) that can be used to inform value-based contracting or treatment strategies intended to minimize “wasted” use of a drug. Ready to learn more about convergence of real-world data and technology for clinical trials?

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Clinical Development of the GluN2B-selective NMDA Receptor Inhibitor NP10679 for the Treatment of Neurologic Deficit after Subarachnoid Hemorrhage [Neuropharmacology]

ASPET

These results further demonstrate the utility of pH-dependent neuroprotective agents and GluN2B-selective NMDAR inhibitors as potential therapeutic strategies for the treatment of aSAH. Based on allometric scaling of both toxicological and efficacy data, the therapeutic margin in man should be at least 2.