China-based PackGene has entered into another partnership with compatriot firm SineuGene. This time, the focus is on plasmid pharmaceutical research, adeno-associated virus (AAV) pharmaceutical research, AAV batch release testing, analytical methodology development, stability research, and more. The collaboration aims to support SineuGene’s lead pipeline candidate SG001, a genetic therapy under development to treat amyotrophic lateral sclerosis (ALS).
ALS Background and Treatment Needs
ALS is a fatal neurodegenerative disease with an unclear pathogenesis and a global prevalence of around 4-6 per 100,000 people. The average survival time for ALS patients is only 2-5 years. Currently, there is no effective drug for the treatment of ALS worldwide, and there is an urgent need to meet clinical demands. In recent years, global research and development related to ALS treatment has mainly focused on the improvement of dosage forms for traditional medicines and the development of cell and gene therapy.
SineuGene’s SG001 and Pipeline
SineuGene, a gene therapy specialist focusing on neural system diseases, is developing ALS targets that are viewed as having great potential. Its most advanced product, SG001, is expected to prolong the survival of ALS patients by saving the motor neurons of patients through gene therapy. The drug is anticipated to enter clinical trials at the beginning of next year.
PackGene’s Expertise and Platforms
PackGene, a CRO/CDMO focused on recombinant rAAV vector packaging, boasts its π-Alpha 293 cell AAV high-yield platform and π-Omega plasmid high-yield platform, along with three other technology platforms. These capabilities will support the research and development efforts in the partnership, particularly in the areas of AAV pharmaceutical research and batch release testing.-Fineline Info & Tech
