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Single-dose gene therapy is potentially life-changing for adults with hemophilia B

Science Daily: Pharmacology News

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.

Therapies 294
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New Gene therapy trial shows restored hearing and speech in children born deaf, treated in both ears

Science Daily: Pharmacology News

A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source localization, ability to hear in noisy environments, and for two children, abillity to appreciate music.

Therapies 304
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Synthesis and Investigation of Peptide–Drug Conjugates Comprising Camptothecin and a Human Protein‐Derived Cell‐Penetrating Peptide

Chemical Biology and Drug Design

Here we investigated the ability of PDCs comprising a human protein-derived cell-penetrating peptideplatelet factor 4-derived internalization peptide (PDIP)as a targeting strategy to improve the selectivity of camptothecin (CPT), a topoisomerase I inhibitor that suffers from off-target toxicity.

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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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Early-stage stem cell therapy trial shows promise for treating progressive MS

Science Daily: Pharmacology News

An international team has shown that the injection of a type of stem cell into the brains of patients living with progressive multiple sclerosis (MS) is safe, well tolerated and has a long-lasting effect that appears to protect the brain from further damage.

Therapies 233
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Scaling Phage Therapy

Codon

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

Therapies 124
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The Power of Worldwide Networks in Data and Safety Monitoring Boards

Advarra

In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Enhanced objectivity: A DMC composed of diverse international experts can mitigate potential biases arising from local or regional perspectives. local standards of care).