Gene therapy program

About Grace Science

Grace Science is a private, pre-clinical stage biotech leveraging a decade of proprietary research on the NGLY1 pathway to develop novel therapeutics. The company's pipeline includes a lead gene therapy program for the ultra-rare NGLY1 Deficiency and exploratory small molecule programs targeting broader indications in cancer and neurodegenerative diseases. With a world-class scientific foundation, notable co-founders including a Nobel Laureate, and a strategic collaboration with Takeda, Grace Science aims to validate NGLY1 as a pivotal therapeutic target with potential impact across multiple disease areas.

About Holostem Tarapie Avanzate

Holostem is a pioneering, commercial-stage Italian biotech specializing in epithelial stem cell-based regenerative medicine. The company has achieved a significant milestone with its lead product, Holoclar, receiving full marketing authorization from the European Commission for treating limbal stem cell deficiency. Operating from within the University of Modena's Centre for Regenerative Medicine, Holostem leverages a strong academic foundation to advance its pipeline of cell and gene therapies, recently gaining recognition as an Important Project of Common European Interest (IPCEI).

Therapeutic Areas