Grace Science

Executive Summary

Grace Science is a private biotechnology company based in San Diego, focused on developing therapies for NGLY1 deficiency, a rare and severe congenital disorder. Founded in 2017, the company has assembled a multidisciplinary team to unravel the mechanistic basis of NGLY1 loss and identify strategies to restore its function. Leveraging expertise in RNA and gene therapy, small molecules, and viral technology, Grace Science is advancing a pipeline aimed at correcting the underlying enzyme deficiency. While the company remains in early stages with no disclosed clinical candidates, its research has provided biological insights that could lead to first-in-class treatments for this orphan disease. Given the high unmet need and limited competition, the company represents a high-risk, high-reward opportunity in the rare disease space.

Upcoming Catalysts (preview)

• Q3 2026IND submission for lead NGLY1 gene therapy candidate70% success
• TBDPublication of preclinical proof-of-concept data in a peer-reviewed journal90% success
• Q4 2026Strategic partnership or licensing deal for NGLY1 program40% success