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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...
177 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...
74 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...
67 views
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...
190 views
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...
100 views
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...
119 views
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...
51 views
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...
257 views
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...
220 views
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...
128 views
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...
188 views
"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...
323 views
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...
235 views
Patient Partners: Harnessing synergies to pursue ASOs as part of the AHC research portfolio
Monday January 29, 2024 12:30 PM US EST Mary will share her son Henry's case and describe her efforts to develop an n=1 ASO for him. Nina...
220 views
Parents of Pioneers: Real experiences in individualized medicines today
Monday, December 18, 2023 at 12:00 pm ES EST Julia Vitarello, Mila's Mom, Mila's Miracle Foundation Julia founded Mila's Miracle...
379 views
Regulatory: The Future Pathway to Access
Monday, November 6, 2023 at 12:30 pm US EDT - Join us for an informative and engaging webinar where we delve into the complex regulatory...
191 views
Regulatory Pathway: Where We Are Today
Monday, October 16 at 12:30 pm US EDT This session will start with an overview of the contents of an N=1 IND application process from...
221 views
Potential Treatment: CMT2S Caused By IGHMBP2 Cryptic Splice Site Variant With ASO Based Therapeutic
October 2, 2023 12:30-1:30 pm US EDT VCA-894A is a novel antisense oligonucleotide (ASO)
242 views
ASO treatment for SCN2A-encephalopathy, allele-specific vs non-specific
September 11, 2023 12:30-1:30 pm US EST - We will discuss the idea of ASO treatment for gain-of-function SCN2A-encephalopathy, including...
292 views
Selecting the right candidate: Which disease-causing variants are eligible for N=1 ASOs?
September 18, 2023 12:30-1:30 pm US EST - Individualized genetic treatments provide new opportunities for targeted therapies for rare...
269 views
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