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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

The Clinical Trials Grants Program provides funding for clinical trials that evaluate the safety and efficacy of potential treatments for rare diseases to help move promising treatments through clinical development. This study design is both innovative and efficient, leveraging a single-arm, baseline-controlled design.

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New cell therapy model accelerates cancer treatment development

Drug Target Review

In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.

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Coordinating the Cell Journey: Essentials in Early Clinical Development

PPD

Within cell therapy clinical trials, apheresis collections contribute to the specific constituents of the given therapy. Whether this is via an allogeneic (healthy donors) or autologous (patient) approach, the material collected is then manufactured and returned to patients for infusion therapy.

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BioMarin pares Roctavian spending as it narrows sales focus

BioPharma Drive: Drug Pricing

The company plans to limit sales of the hemophilia gene therapy to the U.S., Italy and Germany, while ending most clinical development work.

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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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Novel Bispecific therapies in oncology

SugarCone Biotech

The critical question then becomes one of clinical translation finding tumor types that are EGFR-positive and are believed to be immunosuppressed by TGF-. Bicara has focused ficerafusp alfa clinical development on head and neck squamous cell carcinoma (HNSCC), adding pembrolizumab to separately block the PD-1 pathway.

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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.

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