Programs
Grace Science’s unique knowledge and insight about NGLY1 has created opportunities in both rare and common diseases. Synergy across these programs leads to faster and more cost effective drug development and allows us to stay focused and nimble.
PROGRAM
MODALITY
INDICATION
DISCOVERY
PRECLINICAL
PHASE 1/2
PIVOTAL
Neurodegeneration
GS-100
Gene Therapy
NGLY1 Deficiency
GS-310
Small Molecule
Undisclosed
Oncology
GS-220
Small Molecule
Undisclosed
GS-221
Small Molecule
Undisclosed
MODALITY
INDICATION
Gene therapy
NGLY1 Deficiency
DISCOVERY
PRECLINICAL
PHASE 1/2
PIVOTAL
MODALITY
INDICATION
Small Molecule
Undisclosed
DISCOVERY
PRECLINICAL
PHASE 1/2
PIVOTAL
MODALITY
INDICATION
Small Molecule
Undisclosed
DISCOVERY
PRECLINICAL
PHASE 1/2
PIVOTAL
MODALITY
INDICATION
Small Molecule
Undisclosed
DISCOVERY
PRECLINICAL
PHASE 1/2
PIVOTAL
PROGRAM
NGLY1 Deficiency Gene Therapy
GS-100 is a gene therapy being developed to treat NGLY1 Deficiency, a rare autosomal recessive disease that devastates the central nervous system. GS-100 is an AAV9 single-stranded viral vector that encodes the full length human NGLY1 protein. Learn More
PROGRAM
Oncology Small Molecule
GS-220 and GS-221 are small molecule inhibitor programs being developed to treat undisclosed cancers. Their target of inhibition is NGLY1, which in turn shuts down NFE2L1, a critical transcription factor that is difficult to inhibit directly.
PROGRAM
Neurodegeneration Small Molecule
GS-310 is a small molecule program for neurodegenerative diseases. Our research has shown that NGLY1 touches many of the same pathways impacted in common neurodegenerative diseases such as Parkinson’s.
Our
Inspiration
Drug development is incredibly challenging. What drives us to move faster and more effectively are the patients, especially those who are often marginalized and forgotten. These patients are at the core of what we do. Our community has suffered many losses over the years. Although painful, we will not stop. Their dream of a cure lives on. Their fight is our fight.
Alejandro
Alejandro was diagnosed with NGLY1 Deficiency at five years old. He is a cheerful boy. Though he is non-verbal, he is able to communicate his needs to his parents well. Going to the supermarket is his favorite activity, but he also likes dancing and watching Daniel Tiger and Dora the Explorer.
Ezaan
"You were the greatest gift of our lives. You taught us the true meaning of unconditional love and gave us strength. You were a warrior who always managed to make us smile. Our hearts break at the thought of living without you. Until we meet again!"
- Ezaan’s parents
Rupert
Rupert is an incredibly social little boy. He loves listening to music, being sung to and his true passion is books!
Emily
"We were so blessed to have almost 22 years with Emily. We miss her so dearly, everyday. Emily brought so much love, joy, laughter and sassiness to our lives. Emily loved knock knock jokes, watching Barney and singing to her favorite songs." -Emily's parents
Gage
Gage was a perpetually happy young man who wanted nothing more than to be with his family watching the St. Louis cardinals, riding in the car, and participating with his parents in triathlons.
Numa
Numa lives in the south of France. He enjoys reading with his dad, wheelchair racing and school.
Ally and Avah
Sisters, Ally and Avah enjoy listening to music and absolutely love school. Ally can play hand clapping games which is her favorite social interaction with others. Both girls are usually smiling and always looking forward to their next activity.
Tai Tai
Tai Tai enjoyed his 1st Christmas away from his home country of China. He’s adapting to the new living environment, weather, language and new school. His parents’ wish is that all the NGLY1 children can live a happy life.
Benjamin
Benjamin lives in Hawaii. He is amazed by elevators and vehicles, LOVES math and has his own gym membership! He spends a ton of time in therapies and is becoming more vocal every day.
Elora
Elora was diagnosed just before her 2nd birthday. She loves books and watching her favorite TV shows with her family. Elora is a very determined little girl and works very hard in all her therapies.
Nicholas
Nicholas has a smile that brings positivity to everyone around him. Sadly he struggles with many activities we take for granted such as communication, mobility, and feeding. Nicholas’ family prays for a miracle to save him and all other NGLY1 patients.
Owen
Owen is a very happy boy. Owen loves to crawl and walk along the couch and is motivated by food. His favorite things to do are to watch Little Baby Bum videos and play games on his iPad.
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