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.


NGLY1 Deficiency

Pursuing multiple therapeutic modalities including gene therapy, enzyme replacement therapy, and small molecules


Developing small molecule inhibitors of NGLY1 to treat various forms of cancer

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.

“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.”

Matt Wilsey, Co-founder and CEO

Rare Unlocks Common

Grace Science is leveraging NGLY1 biology to treat human disease.

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