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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52
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The Growing Trend of Patient-Led Clinical Research in Rare Disease

Conversations in Drug Development Trends

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?

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Organoids: the versatile platform for discovery and regeneration

Drug Target Review

Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.

Treatment 105
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A transatlantic collaboration is uncovering the role of key genetic variants in common diseases like type 2 diabetes and obesity

Broad Institute

By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.

Disease 72
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The future of mental health treatment: Zelquistinel’s role

Drug Target Review

Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.

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Sanofi Showcases Rare Disease Pipeline Innovations at ISTH

The Pharma Data

Sanofi Highlights Robust Pipeline of Innovations in Rare Blood Disorders at ISTH 2025 Sanofi is poised to take center stage at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), set to be held in Washington, D.C., from June 21 to 25, 2025. In the U.S.,

Disease 40
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FDA Approves CSL’s Andembry First Once-Monthly Prophylactic HAE Treatment Targeting Factor XIIa

The Pharma Data

This unique approach prevents attacks from occurring before they start, offering patients a new opportunity to manage their condition while reducing the immense disease burden. ANDEMBRY, a novel once-monthly subcutaneous treatment that inhibits factor XIIa, is a welcome addition to the HAE treatment landscape,” said Anthony J.