Seminar SeriesDeciphering SPTAN1-associated disease mechanism and potential therapy in patient-derived cell lines
Seminar SeriesTowards a divalent siRNA therapy for prion disease: an academic, patient-scientist led effort
Seminar SeriesAdvancing human hematopoietic stem cell therapies: from sickle cell disease to personalized treatments for bone marrow failure disorders
Seminar SeriesSplice-Altering Transposon Insertions in Neurodegenerative Diseases and Its Application in an Individualized ASO Therapy for Retinitis Pigmentosa
Seminar SeriesPreclinical development of personalized ASO therapeutics using patient-derived organoid systems
Seminar SeriesTowards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders
Seminar SeriesPersonalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder
Seminar SeriesRNA/DNA based analytics to guarantee product safety – challenges for N-of-1-treatments
Seminar SeriesProject Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
Seminar SeriesSnakeholders and Stakeholders: How Zookeepers and Border Smugglers Set the Stage for Antivenom Commercialization in the USA
Seminar SeriesTransforming Patient Access to High-Cost, One-Time Treatments through Financial Engineering Innovation
Seminar SeriesFast Track to Final Drug Product: Understanding Costs, Challenges, and Timelines in Personalized Medicine Manufacturing
Seminar SeriesPRAX-222 Therapy for a Child: Prenatal Onset SCN2A Developmental and Epileptic Encephalopathy in a Named Patient Setting
Seminar Series"Control of starting material quality for oligonucleotide therapeutics" and "To Synthesis and Beyond…Production of oligos for ‘N-of-1’ therapies"
