N=2 Research Sponsorship

Fueling Breakthroughs in Muscular Dystrophy Research

Your sponsorship has the power to directly accelerate life-saving advancements in genetic research. Hope for Luka’s preclinical research—targeting a rare subset of muscular dystrophy through double exon skipping of exons 6 and 7—is now in advanced stages.

Likelihood of Positive Results

Our current research is expected to result in a stable, functional 6–25 transcript and is grounded in scientific precedent: individuals with natural deletions of exons 6–18 have demonstrated milder phenotypes, confirming that 6–25, our targeted outcome of double exon-skipping research, is a viable therapeutic goal. (PPMD & CureDuchenne Registries in US, Leiden University Registries in Europe)

Potential Patients Benefitting From the Outcome

With your support, over 200 children in the U.S. and 100 children across Europe who could potentially benefit from this treatment. This effort is not just promising—it’s tangible. (Obtained and analyzed from: PPMD & CureDuchenne Registries in US, Leiden University Registries in Europe)

Creation of N=1 Blueprint From Current N=2 Research

This study will serve as the foundation for a detailed, step-by-step blueprint. This model will be adapted into personalized N=1 (or N=2) studies for other patients who also require double skipping of exons 6 and 7, but have different deletion endpoints—empowering them to pursue targeted research tailored to their unique mutation.

U54 Precision Grant - Jackson Laboratory

The Jackson Laboratory (JAX) has accepted and approved full funding for our nominated 6-25del and 8-25del mice models under their Precision Genetics U54 grant. This partnership will accelerate the development of the two mice models, with the expected readiness in 9–12 months.

Mice Model Joint Ownership with LUMC

Hope for Luka inc. has been granted joint ownership of the hDMD/del6-25 and hDMD/del8-25 mouse models in collaboration with Leiden University Medical Center (LUMC) in the Netherlands. LUMC has officially approved the use and modification of the hDMD mouse model, available through The Jackson Laboratory (JAX), for the development of these specific deletions. 

Research Sponsorship Packages

Sponsoring our research means joining the frontlines of an unprecedented precision medicine approach to rare disease. The following sponsorship tiers are tailored to support our current preclinical pipeline:

Research Ally – $1,000

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Contribute to core operational needs that keep our research infrastructure running.

Benefits include:

    • Annual research impact report

    • Recognition on our website

    • Optional listing as a “Hope for Luka Research Ally”

    Research Kickstarter – $3,000

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    Help fund the preclinical associated supporting costs.

    Benefits include:

    • Recognition on our social media presence

    • Listing on our website as “Hope for Luka Research Kickstarter”

    • Semi-annual research updates

    Discovery Partner – $5,000

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    Help cover critical costs in biomarker studies and data validation.

    Benefits include:

    • Recognition on our social media presence

    • Listing on our website as “Hope for Luka Discover Partner”

    • Quarterly research updates

    Translational Advocate – $25,000

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    Support the transition from preclinical to IND-enabling work.

    Benefits include:

    • Recognition on our social media presence

    • Listing on our website as “Hope for Luka Translational Advocate”

    • Quarterly research updates

    • Name/logo on research milestone reports

    Therapeutic Builder – $100,000

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    Fund pivotal animal studies and regulatory consultation.

    Benefits include:

    • Recognition on our social media presence

    • Listing on our website as “Hope for Luka Therapeutic Builder”

    • Quarterly research updates

    • Name/logo on research milestone reports

    • Seat at our Research Partner Roundtable (Semi-annually calls with our science advisors)

    • Recognition in key publications and presentations

    Legacy Catalyst – $250,000+

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    Become a cornerstone sponsor driving the path to clinical trials.

    Benefits include:

    • Recognition on our social media presence

    • Listing on our website as “Hope for Luka Therapeutic Builder”

    • Quarterly research updates

    • Name/logo on research milestone reports

    • Seat at our Research Partner Roundtable (Semi-annually calls with our science advisors)

    • Recognition in key publications and presentations

    • Custom naming opportunity (e.g., lab initiative or data portal)

    • Exclusive strategy briefings with our leadership and research partners

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