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Trial Confirms 'Life-Changing' Impact of Gene Therapy, Beqvez, for Hemophilia B

Drugs.com

25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows. THURSDAY, Sept. People with hemophilia B saw their bleeding episodes.

Therapies 116
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How long do clinical trial phases take?

Antidote

Before a potential new therapy can be administered to patients, it must go through several clinical trial phases designed to test the drug or device for safety and effectiveness. For many new treatments, it will take approximately ten years for the therapy to progress from initial discovery to being approved.

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Trial Confirms 'Life-Changing' Impact of Gene Therapy for Hemophilia B

Drugs.com

25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows.People with hemophilia B saw their bleeding episodes. THURSDAY, Sept.

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New targeted therapies show promise in lung cancer treatment

Drug Target Review

2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. 7 Recent clinical studies highlight promising developments in c-MET-targeting ADCs for NSCLC.

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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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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. Credit: Neumann EN, Bertozzi TM, et al.

Disease 142
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A guide to clinical trial phases [infographic]

Antidote

Before any new treatment or therapy is able to be used by the patient population, it must go through the process of a clinical trial — this is the case for any new prescription drug, but also true for over-the-counter medications, medical devices, and more.