Our Approach
By assembling a world class, multidisciplinary team of experts and encouraging them to share expertise and knowledge, we’ve developed biological insights that suggest why NGLY1 loss leads to disease and how to restore it. We have also learned that the biology underlying NGLY1 Deficiency is central to more common indications, most notably cancer. We will target:
NGLY1 Deficiency
Cancer
Neurodegeneration
This overlapping discovery is game-changing.
Our Inspiration
NGLY1 Deficiency is an extremely rare genetic disorder caused by mutations in the NGLY1 gene, which results in the deficiency of an enzyme known as N-Glycanase 1. Patients with this disease suffer from a lifetime of debilitating symptoms including severe cognitive impairment, liver dysfunction, and motor defects. Their fight is our fight.
Emily, 20 years old
Ontario, Canada
At 20 years old, Emily is one of the older known patients. She is also one of the few patients that can speak and she’s very sociable. One of the things she enjoys the most is giving people around her hugs.
Tai Tai, 7 years old
Hong Kong, China
Tai Tai is a very happy boy: he can take independent steps and loves to explore the environment around him. He enjoys playing with small objects and can even draw straight lines with a pencil.
Mariam, 5 years old
New York, USA
Mariam enjoys clicking around and playing with touch screens. Her 2 year old younger brother also has NGLY1 Deficiency.
“We’re leveraging rare disease biology and applying to more common indications. Given our race against time, we’re bringing together top talent and dropping them in a fast-paced, collaborative environment that is highly iterative.”
Rare Unlocks Common
Grace Science is leveraging NGLY1 biology to treat human disease.