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MEET LUCAS
Lucas is a happy boy just like any other child. The difference is he is losing his ability to walk and his independence.
Although you would never know from just looking at him - he is the happiest boy you will ever meet.
Lucas works incredibly hard to do things other children do naturally. Walking is what brings Lucas the most joy.
But his condition is progressive and time maters.
While other children his age are playing with their peers in daycare and pre-school, Lucas is in therapy for multiple hours a day - with a big smile on his face. DETERMINED.
THE REALITY
No treatment exists
No research exists
No roadmap exists
He is losing skill
Life expectancy is unknown
This is the reality of many "Ultra-Rare" diseases
WHAT WE'RE DOING
We are not waiting.
We are actively funding and coordinating the development of a gene therapy for RBM28.
This includes:
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Early-stage research and disease modeling
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Vector development (AAV-based gene therapy)
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Pre-clinical testing and regulatory preparatio
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International collaboration
Gene therapy is currently the only approach that can address the root cause of Lucas' condition.
WHERE WE ARE NOW
$624,000+ raised
Initial research underway
Next phase: $500K USD
WHERE DO FUNDS GO
Funds raised are used to advance a coordinated research and therapy development strategy, including:
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Gene therapy development (AAV design, vector optimization)
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Disease biology research (understanding how RBM28 impacts cells and function)
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Patient-derived cell studies (including international research collaborations)
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Drug screening and repurposing efforts using Lucas’ cells
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Pre-clinical studies and validation
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Regulatory preparation and pathway development