hemophilia - International Biopharmaceutical Industry

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Subsection - Neurodegenerative Diseases

From Depression to Deep-Learning: Leveraging iPSC and AI Platforms for Evidence-Based Depression Treatment

Subsection - Oncology

Essential DMPK Strategies for IND Success

Subsection - Cell and Gene Therapy

When the Gene Editing Guide is the Drug

news

Sanofi, with positive data in hand, to ask for approval of longer-lasting hemophilia therapy

Sanofi and its development partner, the Swedish drug company Sobi, said Wednesday they plan to submit an experimental, longer-lasting hemophilia treatment for approva...

14 March 2022 news Drug | hemophilia

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Fresh data show UniQure’s hemophilia gene therapy appears to hold up

Gene therapies have the potential to essentially fix many diseases. Yet, those currently in development are administered just once, raising questions about whether th...

10 December 2021 news blood-clotting | Gene Therapies

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Pfizer gene therapy research delayed by trial changes, safety questions

In both hemophilia and Duchenne, unexpected findings have led Pfizer to redraw its development plans. The company recently paused patient screening and dosing in a P...