Rare Disease Day (Feb, 28, 2025) – Breaking Barriers in Rare Disease Gene Therapy
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.
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.
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.
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.
Details
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.
At PackGene, we believe in science-driven impact—ensuring no patient is left behind.