Ivy's Pathway to Treatment
May 2026Ivy is 1.5 years old
1
Generate Neurons from Ivy's Blood
Create patient-derived neurons for ASO research and testing.
2
Sequence Ivy's DNA
Identify all possible ASO targets across Ivy's genetic code.
3
Study Ivy's Variant
Understand exactly how her variant harms her neurons to guide ASO development.
4
Design & Synthesize 500 Candidate ASOs
Build a broad library of antisense oligonucleotides tailored to Ivy.
5
Screen to Find the Top 20
Test all 500 ASOs in Ivy's own neurons to find the most promising candidates.
6
Small-Scale Manufacturing
Produce the top 20 ASOs for further safety evaluation.
7
Safety Investigation in Rats
Evaluate the safety of the top 20 ASOs through preclinical studies.
8
Manufacture the Optimal ASO
Scale up production of the single best-performing candidate.
9
Final Safety & Toxicity Testing
Comprehensive safety studies on the optimal ASO.
10
Prepare the Finished Product
Formulate the final treatment to be administered to Ivy.
11
Start Ivy on Treatment ✦
Begin Ivy's personalized antisense oligonucleotide therapy.
2028–2029Ivy is 4–5 years old