The splicing platform deep dive will provide an overview of PTC's validated splicing platform which leverages its cutting-edge technology to selectively and specifically identify molecules that modulate mRNA splicing to upregulate or downregulate protein production. The deep dive will review the mechanisms underlying the splicing platform obtained over 10 years of development that have driven the platform's progress to date and yielded PTC's discovery of risdiplam. PTC will also provide a clear roadmap for the vast potential of the splicing platform moving forward.
The webinar can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the webinar and providing the passcode 9153632. A live, listen-only webcast can be accessed on the Events and Presentations page under the investor relations section of
About the Splicing Platform
PTC uses its alternative splicing technology to identify molecules that modulate mRNA splicing. Approximately 94% of all human genes undergo splicing. Through alternative splicing, by including or excluding exons, one gene can generate several mRNA products that can have different biological functions. PTC has developed a powerful high-throughput drug discovery technology that enables us to identify small molecule modifiers of pre-mRNA splicing. Using this technology, we have successfully identified oral small molecules to upregulate splicing of a key protein for spinal muscular atrophy. Based on the knowledge and experience from the SMA program, we believe that other small molecule drug candidates can be identified that modify alternative splicing of genes, promote inclusion of specific exons into mRNA or force skipping of undesired exons from the mature mRNA. We believe that this technology has a broad potential applicability to a large number of target genes in all therapeutic areas.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.
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