Consultancy
WinQure Medicine provides scientific and development support for individualized antisense oligonucleotide (ASO) therapies. We work with families, clinicians and organisations worldwide to help bring therapies for ultra-rare genetic diseases closer to patients.
How we can support
We help families, clinicians, foundations and genetics companies develop individualized ASO therapies, from genetic diagnosis to a therapy ready for clinical use. We can manage the entire program or support individual stages.
We provide scientific oversight, design development strategies and coordinate activities across each stage. We work with academic laboratories, research institutions and contract research organizations (CROs), selecting and managing the partners best suited to each program.
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Assessment
Diagnosis and variant interpretation
Review of genetic findings to establish the disease mechanism and understand how the variant affects gene function.
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Feasibility
ASO feasibility assessment
Evaluation of whether an ASO can address the disease mechanism and identification of the most suitable therapeutic approach.
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Discovery
ASO design and pre-clinical validation
Design and screening of ASO candidates, followed by proof-of-concept studies in patient-derived cells and relevant disease models.
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Toxicology
Pre-clinical safety assessment
Coordination of toxicology studies to assess ASO safety and support progression to clinical use.
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Manufacturing
Clinical-grade ASO manufacturing
Coordination of ASO manufacturing, quality control and release testing for clinical use.
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Clinical
Preparation for clinical use
Preparation of the ASO and supporting development data for transfer to the treating physician.
Individualized programs
These are individualized programs, separate from our active programs pipeline. We do not publish gene or variant details. Each program is run with the patient’s family, their clinicians and the funding organisation, who decide what is shared and when.
Developing an individualized ASO therapy?
Contact us to discuss how we can support your program.
Are you already developing an individualized ASO therapy and need support completing development, or are you considering starting a new program? Contact us to learn how we can help.
