Developing a Disease-Modifying Treatment for Infantile Neuroaxonal Dystrophy (INAD)
The INADcure Foundation is leading the development of a groundbreaking AAV9-based gene therapy for Infantile Neuroaxonal Dystrophy (INAD), a rare, fatal neurodegenerative disorder caused by mutations in the PLA2G6 gene. With no approved treatments available, our mission is to advance a potentially disease-modifying therapy into clinical trials as quickly as possible.
This initiative is 100% funded by the INADcure Foundation, driven by grassroots fundraising and the unwavering dedication of patient families. While we are spearheading this effort, we actively seek industry partnerships, research collaborations, and investment to accelerate progress and bring hope to children with INAD.
The Urgency of Our Mission
Families affected by INAD cannot afford to wait for industry interest to catch up. That’s why we took action—driving this progress forward ourselves.
INAD is an ultra-rare disease with limited commercial incentives for traditional biotech or pharmaceutical investment. To bridge this gap, the INADcure Foundation has taken the lead in funding and advancing this gene therapy program, ensuring that progress continues toward a viable treatment.
“After years of fruitless door-knocking and making our case to more researchers and biotech companies than we can count, we were compelled to make a decision. Either we continue to wait for someone else to do it, or we do it ourselves.”
– Leena Panwala, Ariya’s mom and Founder of INADcure
Funding the Future
$5.8M Needed to Launch Clinical Trials
With preclinical development nearing completion and regulatory designations secured, moving to the clinical trial phase requires substantial funding.
• $1.8M is needed for GMP drug manufacturing – Ensuring clinical-grade therapy production.
• $3M is needed for clinical trial costs – Covering patient treatment, trial operations, and regulatory oversight.
We have come this far through community-driven efforts, but securing this final $5.8M is the key to bringing this therapy to the children who need it. Your support will allow us to manufacture clinical-grade material and launch a first-in-human trial—critical steps toward making this treatment a reality.
Our Investigational Therapy
Approach: Gene Replacement Therapy
Mechanism: The therapy aims to restore enzymatic function by delivering a healthy copy of the PLA2G6 gene, addressing the root cause of INAD and potentially slowing or halting disease progression.
Delivery System: AAV9 vector – Chosen for its ability to efficiently deliver a functional copy of the PLA2G6 gene to the central nervous system (CNS).
Delivery Method: Intracisternal magna (ICM) injection – a targeted approach to deliver the therapy directly to the CNS, maximizing efficacy and minimizing systemic exposure.
Preclinical Proof of Concept
Our preclinical studies have demonstrated:
✔ Efficacy: Extended survival in PLA2G6-deficient mouse models.
✔ Safety: Favorable tolerability in early toxicity studies, with no significant adverse effects observed.
✔ Dose Optimization: A dose-finding study is underway to determine the optimal clinical dose.
Development & Clinical Pathway
Current Status:
Our program is in the final stages of preclinical development, with IND-enabling studies nearing completion.
Key Milestones Achieved:
✔ Proof-of-concept studies in INAD models
✔ Initial safety and biodistribution data
✔ Development of a GMP-compliant manufacturing process
Next Steps:
Complete Toxicology and Dosing Studies: Expected completion by June 2025.
Initiate GMP Manufacturing: Target Q2 2025.
IND Submission: Target September 2025
First-in-Human Clinical Trial: Pending FDA approval and readiness of the drug.
Regulatory Designations
The INADcure Foundation’s gene therapy program has received:
• Orphan Drug Designation (ODD)
• Rare Pediatric Disease Designation (RPDD)
These designations underscore the urgency of our work and may enable expedited regulatory pathways.
Opportunities for Collaboration
We welcome partnerships with:
Biotech and pharma companies interested in co-development, strategic partnerships, or research collaborations.
Researchers and clinicians exploring complementary approaches or biomarkers.
Investors and funding organizations seeking to support rare disease innovation.
For inquiries about collaboration opportunities, please contact: 📩 [email protected]
Our Partners and Collaborations
