During Rare Disease Week, PackGene reaffirms its mission to make gene therapies accessible and affordable for ultra-rare conditions. Through our expertise in plasmid DNA and viral vector manufacturing, we are driving innovative treatments for underserved diseases.
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- Gene Therapy for SPG56
- Partnered with Genetic Cures for Kids and Weill Cornell Medicine’s Belfer Gene Therapy Core Facility to develop a gene therapy for SPG56, an ultra-rare neurological disorder.
- Aims to provide life-changing treatment and a scalable model for rare disease therapies.
- Gene Therapy for SPG56
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- Restoring Hearing for OTOF Patients
- Collaborated with Shanghai Refreshgene Technology Co., Ltd. on RRG-OTOF therapy, which received FDA Orphan Drug Designation (Dec 2023).
- Clinical success: Hearing restored in 10 of 11 treated children.
- Restoring Hearing for OTOF Patients
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- Advancing Rare Disease Solutions
- Cutting-edge manufacturing technologies enhance therapy quality, yield, and affordability.
- Building a scalable, cost-effective gene therapy framework to serve more patients.
- Advancing Rare Disease Solutions
We recently published “Breaking Barriers: PackGene Facilitates the Development of Gene Therapy for OTOF Hearing Loss and SPG56,” showcasing our commitment to rare disease innovation.
Poster
Solutions for Making AAV Gene Therapy Affordable for Rare Disease`
At PackGene, we believe in science-driven impact—ensuring no patient is left behind.
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