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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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Biogen Advances Investigational SMA Therapy to Registrational Trials After Positive Phase 1 Data

The Pharma Data

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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The rising impact of biomarkers in early clinical development

Drug Target Review

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. There are several types of biomarkers to consider.

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Managing CGT trials: the role of IRT from discovery to clinical development

Drug Target Review

The journey of cell and gene therapies from preclinical discovery to clinical trials is complex and challenging, impacting every team member involved, from researchers in the lab to patients receiving treatment. These digital platforms are designed to manage and automate critical trial processes, especially related to drug management.

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Merck’s WINREVAIR Succeeds in Phase 3 Trial for Pulmonary Arterial Hypertension

The Pharma Data

Study Overview and Population The HYPERION study was designed to assess the efficacy and safety of WINREVAIR when used in combination with standard background therapy, compared to placebo, in adult patients recently diagnosed with pulmonary arterial hypertension. It follows two earlier landmark studies: STELLAR and ZENITH.

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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

“This experience underscored the gaps in rare disease innovation, motivating me to lead Alltrna, a company pioneering engineered tRNA therapies to address a broad range of genetic diseases in a profound way unlike any other genetic modality.” While commonly used in oncology, the basket trial approach is novel for rare genetic diseases.

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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

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. In October, FDA announced seven new clinical trial grants awarded in fiscal year (FY) 2024 – including one for a Phase 3 trial – totaling $17.2