Moonshots for Unicorns develops precision gene‑editing therapies to replace or repair the missing PGAP3 gene in children with PGAP3‑CDG and other ultra‑rare single‑gene disorders.
Funding
Funding not disclosed
Founders
Product
Problem
Children with single-gene disorders such as PGAP3 deficiency experience severe neurological symptoms, including intellectual disability, lack of speech, muscle weakness, and refractory seizures, with no existing curative treatments.
Solution
Moonshots for Unicorns focuses on developing gene‑editing therapies that replace or repair the missing PGAP3 gene in affected cells. By partnering with a leading pediatric gene‑therapy center, the organization accelerates preclinical research, vector design, and clinical trial initiation to achieve a curative outcome within an 18‑month timeline. The approach leverages precision genome‑editing tools and scalable viral delivery platforms to restore normal cellular communication and prevent disease progression. Ongoing fundraising directly supports laboratory work, manufacturing, and regulatory activities needed to bring the therapy to patients as quickly as possible.
Target Audience
Primary beneficiaries are children diagnosed with PGAP3‑CDG and other ultra‑rare single‑gene disorders, as well as their families and the clinicians who treat them.
Features
- Precision genome‑editing strategy targeting the PGAP3 locus to restore functional protein expression
- Use of clinically validated viral vectors for efficient delivery to multiple tissue types
- Integrated preclinical pipeline with rapid in‑vitro and animal model testing to de‑risk clinical development
- Collaboration with Nationwide Children’s Hospital Center for Gene Therapy for GMP‑grade manufacturing and trial oversight
- Transparent funding model that channels donor contributions directly into research, development, and trial costs