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Towards a divalent siRNA therapy for prion disease: an academic, patient-scientist led effort
Monday, September 8, 2025 at 12:30 pm US EDT Prion disease is a rapidly fatal neurodegenerative disease with no current standard or care....
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Advancing human hematopoietic stem cell therapies: from sickle cell disease to personalized treatments for bone marrow failure disorders
Monday, September 22, 2025 at 12:30 pm US EDT Dr. Shengdar Tsai will describe progress in our efforts to advanced personalized genome...
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Splice-Altering Transposon Insertions in Neurodegenerative Diseases and Its Application in an Individualized ASO Therapy for Retinitis Pigmentosa
Monday, June 23, 2025 at 12:30 pm US EDT Transposon insertions represent a significant yet understudied source of disease-causing...
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Preclinical development of personalized ASO therapeutics using patient-derived organoid systems
October 6, 2025 at 12:30 pm US EDT This presentation will cover our laboratory's ongoing efforts in the areas of personalized and...
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Towards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders
June 2, 2025 at 12:30 pm US EDT Featured in the News: Join us for a seminar spotlighting the groundbreaking case of KJ Muldoon— the first...
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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...
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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...
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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...
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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...
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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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Treating the Nano-Rare: Redefining Clinical Management, One Patient at a Time
November 18, 2024 at 12:30 pm US EST This presentation will cover the progress made in the clinical management and treatment of...
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Snakeholders and Stakeholders: How Zookeepers and Border Smugglers Set the Stage for Antivenom Commercialization in the USA
October 21, 2024 at 1:00 pm US EDT Leslie V. Boyer, MD , is a graduate of Harvard Medical School, a Professor Emerita of the University...
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The challenges and opportunities in rare disease
November 4, 2024 at 4:00 pm US EST Four hundred million people world wide have a rare disease and yet still less than 5% of patients have...
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Transforming Patient Access to High-Cost, One-Time Treatments through Financial Engineering Innovation
August 26, 2024 at 12:30 pm US EDT Self-insured employers collectively cover over 120 million lives in the United States. Many of these...
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Preclinical Development for Personalized Therapy Using Disease-Relevant Cell Models
Monday, July 15 2024, 12:30 pm US EDT iXCells Biotechnologies is specialized in providing innovative pre-clinical drug discovery...
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Stopping the clock on KIF1A associated neurological disorder
September 16, 2024, 12:30 pm US EDT KIF1A associated neurological disorder is a rare neurodegenerative neurological disorder associated...
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Fast Track to Final Drug Product: Understanding Costs, Challenges, and Timelines in Personalized Medicine Manufacturing
May 6, 2024 at 12:30 pm US EDT In this webinar, speakers from Berkshire Sterile Manufacturing (BSM) and Argonaut Manufacturing Services...
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PRAX-222 Therapy for a Child: Prenatal Onset SCN2A Developmental and Epileptic Encephalopathy in a Named Patient Setting
Monday, April 22 2024 12:30 pm US EDT Early onset SCN2A developmental and epileptic encephalopathy (SCN2A-DEE) is caused by...
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"Control of starting material quality for oligonucleotide therapeutics" and "To Synthesis and Beyond…Production of oligos for ‘N-of-1’ therapies"
Monday, March 25 2024 12:30 pm US EDT This presentation focuses on the control of starting material (SM) quality in the manufacture of...
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Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting
Monday, March 4, 2024 12:30 pm US EST From an initial email request or conversation at a meeting… to (for some) the possibility of...
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