The call will include a snapshot from the ongoing InSight Phase 1/2 extension study of sepofarsen, including new preliminary data on second eye treatment. Participation in this extension study was offered to patients who completed their participation in the Phase 1/2 study of sepofarsen.
The call will feature a presentation by
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Following the presentation and discussion, a portion of the call will be dedicated to Q&A. The archived presentation will be available on the Company’s website for approximately 30 days following the presentation date.
Ian M. MacDonald MSc, MD CM is one of the top experts in the Inherited Retinal Diseases field, and is currently Professor Emeritus in the
Sepofarsen (QR-110) is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to bind to the mutated location in the pre-mRNA and enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein.
In a Phase 1/2 clinical trial sepofarsen reported rapid, significant and durable improvements in vision with a favorable benefit/risk profile. Sepofarsen is currently being investigated in a pivotal Phase 2/3 clinical trial.
Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such statements include those relating to sepofarsen. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the
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Source: ProQR Therapeutics N.V.