We are excited to share that we successfully dosed the 4th NGLY1 Deficiency patient with GS-100 yesterday.?The procedure and drug infusion took place at UCSF Benioff Children's Hospitals in Oakland.?The team here is so impressive.?This is the youngest patient we have dosed to date (a 5-year-old boy). This is the 2nd patient dosed at the mid-dose.?The photo below is from our post-surgery debrief. The sign behind us says "Live Hope".?So appropriate for Grace Science and our backers.?You all?have "enabled hope" for millions of children who suffer from a rare genetic disease and can benefit from gene therapy.
关于我们
Grace Science is leveraging NGLY1 biology to treat various rare and common diseases.
- 网站
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https://gracesciencellc.com
Grace Science, LLC的外部链接
- 所属行业
- 生物技术研究
- 规模
- 11-50 人
- 总部
- Menlo Park，CA
- 类型
- 私人持股
- 创立
- 2017
地点
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主要
获取路线
US，CA，Menlo Park，94025
Grace Science, LLC员工
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Jennifer Cook
Associate Director - Assay Development at Grace Science, LLC
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Brett Crawford
Staff Scientist, Head of the Musculoskeletal Therapeutic Area in BioMarin Research
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Selina Dwight
Executive Director of Regulatory Affairs at Grace Science, LLC
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Hiten Trivedi
BioProcess | Gene Therapy | Cell Ag
动态
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Yesterday we successfully dosed the 3rd patient with our gene therapy (GS-100)! All went well. This is the 1st patient at the mid-dose as well as the 1st patient dosed at our 2nd clinical site (UCSF Benioff Children's Hospitals). #genetherapy #raredisease #ngly1
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We are tremendously excited to be 1 of 3 programs selected for the START pilot program in CBER. https://lnkd.in/g9tdvk2b
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Successful dosing of patient #2 this morning in Houston! Big thanks to our teammates at Baylor College of Medicine Texas Children's Hospital.
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