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Personalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder
March 24, 2025 at 12:30 pm US EST We developed personalized splice-modulating ASOs targeting a deep intronic variant in PEX1 in a 2 year...
10 views
RNA/DNA based analytics to guarantee product safety – challenges for N-of-1-treatments
Monday, February 24, 2025 at 12:30 pm US EST The traditional analytical life cycle for quality control of drugs fits not well with the...
104 views
Project Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
January 13, 2025 The presentation will review our development of a personalized antisense oligonucleotide (ASO) medicine for two children...
214 views
Personalized medicine for rare neurogenetic disorders: can we make it happen?
Agnies M van Eeghen , Hilgo Bruining , Nicole I Wolf , Arthur A Bergen , Riekelt H Houtkooper , Mieke M van Haelst , Clara D van...
51 views
A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases
Olivia Kim-McManus, Joseph G. Gleeson, Laurence Mignon, Amena Smith Fine, Winston Yan, Nicole Nolen, Scott Demarest, Elizabeth...
102 views
Phage Therapy for Multidrug-Resistant Bacterial Infections: Past and Future
February 10, 2025 at 12:30 pm US EST The session will begin with a presentation from our guest speaker, Dr. Jason Gill, providing an...
115 views
SNP-selective siRNA for the treatment of Fibrodysplasia Ossificans Progressiva
December 2, 2024 at 12:30 pm US EST Fibrodysplasia Ossificans Progressiva (FOP) is a devastating, ultra-rare condition, where patients...
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The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap
Anneliene H. Jonker, Elena-Alexandra Tataru, Holm Graessner, David Dimmock , Adam Jaffe, Gareth Baynam, James Davies, Shruti Mitkus,...
120 views
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