From a single patient to a multi-patient program. A $925,000 leadership gift to Mayo Clinic unlocks the next phase of ADLD treatment.
First-in-Human Underway
n=1 trial active at Mayo Clinic
Mayo Clinic Lead
RNA Therapeutics Discovery Program
n-Lorem Foundation Partner
Individualized ASO development
Where We Are
For patients living with adult-onset autosomal dominant leukodystrophy, no disease-modifying treatments have ever been approved or formally studied in clinical care. ADLD progresses slowly but relentlessly, often over more than a decade, ultimately leading to death.
That changed when Mayo Clinic, in collaboration with the n-Lorem Foundation, developed a first-of-its-kind antisense oligonucleotide (ASO) therapy targeting the root cause of ADLD: overexpression of the LMNB1 gene. ASOs have already proven successful in genetic and neurologic disorders including spinal muscular atrophy, ALS, and Duchenne muscular dystrophy.
Our single-patient trial at Mayo has shown signs that the therapy may be safely reducing disease-related damage. The patient has tolerated multiple doses well, with initial decreases in markers of neurodegeneration.
The Path Forward
Up to five participants enrolled in a clinical trial targeting the known disease mechanism. Each patient is treated and monitored over two years.
Expand enrollment, refine biomarkers from the Natural History Study, and strengthen the multi-patient dataset that anchors regulatory engagement.
Prepare for regulatory review and grow durable program infrastructure so this platform can serve future ADLD therapies and other rare diseases.
The Investment
Mayo Clinic requests a leadership gift of $925,000, payable over five years, to build and scale this program from one patient to a regulator-ready multi-patient trial.
Cost Breakdown
5-Year Program
Trial Management and Oversight
Clinical trial coordination, regulatory affairs, monitoring
$250,000
Participant Costs
$100,000 per patient over two years; covers dosing, imaging, lab work, and follow-up for up to five participants
$500,000
Program Infrastructure
Principal investigator, research and clinical operations, data management and reporting
$175,000
Total Leadership Gift
$925,000
Why This Matters
Multi-patient data gives the FDA the evidence needed to consider expanded access, IND filings, and a path to approval.
Mayo Clinic gains an established ASO trial infrastructure that can be adapted for future ADLD therapies (shRNA, repurposed drugs) and other rare diseases.
A funded multi-patient program de-risks the science and positions ADLD for grants, biotech partnerships, and additional philanthropy that single-patient trials cannot attract.
A successful trial platform creates a path for every ADLD patient who has been waiting for a treatment, not just the first.
Fund the Expansion
Every dollar goes directly to Mayo Clinic's ASO trial program. 100% to science, no overhead.
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