February 28th is the 16th International Rare Disease Day with the theme of “Shine lights on your life” .

Rare diseases, also known as “orphan diseases”, have a low incidence rate and multiple disease types. Most of them are chronic, lifelong, severe, and life-threatening. The World Health Organization (WHO) defines rare diseases as diseases with an incidence rate between 0.65-1‰ of the total population. In May 2010, the Chinese Medical Association’s Medical Genetics Branch reached a consensus on rare diseases: they are diseases with a low incidence rate of less than one in 500,000 in adults and less than one in 10,000 in newborns.

To support rare disease therapy, PackGene Biotechnology Co., Ltd. has recently obtained DMF registrations for GMP plasmids: DMF28932 and DMF29120 from Food and Drug Administration (FDA). These DMP registrations cover information related to the fermentation, purification, and release testing of plasmid products. The DMF registration of GMP plasmid can provide pharmaceutical-related production, raw material, and quality control information for IND applications, shortening internal communication, review, and evaluation time, accelerating IND filing and benefiting rare disease therapy development.

In October 2022, PackGene completed the DMF registration of AAV auxiliary plasmids with the US FDA and received a confirmation letter with registration number DMF28932.


In January 2023, PackGene completed the DMF registration of AAV packaging plasmids with the US FDA and received a confirmation letter with registration number DMF29120.

About PackGene

PackGene is a world-leading Adeno-Associated Virus (AAV) vector CRO and CDMO company, providing economical, reliable, and scalable plasmid DNA and AAV viral vector production for early-stage drug discovery, preclinical development, and clinical trials for Cell and Gene Therapy (CGT). The proprietary π-Alpha™ 293 AAV High-Yield Platform uses uniquely designed RC plasmid in the triple-plasmid transfection system to increase AAV production by three to eight times in various AAV serotypes. By combining both in-process upstream and downstream QbD optimizations, the total AAV yield can be increased up to ten times. PackGene’s services are based on major technology platforms including the π-Alpha™ 293 cell AAV high-yield platform and π-Omega™ plasmid high-yield platform, with comprehensive analytical development and quality control capabilities. For more information, please visit https://www.packgene.com.

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