The TECPR2 Research Foundation, a nonprofit organisation focused on advancing treatments for TECPR2-related disorder, has entered into a development and manufacturing partnership with Forge Biologics to advance an investigational AAV gene therapy for the ultra-rare neurodegenerative genetic disease.

Under the partnership, Forge will provide process development, analytical development and qualification, as well as manufacturing services, including the production and release of material for toxicology studies. The programme will use Forge’s FUEL platform, which includes HEK293 suspension Ignition Cells, pEMBR 2.0 adenovirus helper plasmid, rep/cap plasmids and programme-specific optimisation packages.

TECPR2-related disorder is caused by pathogenic variants in the TECPR2 gene and is associated with developmental delays and progressive neurological complications, including respiratory dysfunction. The TECPR2 protein plays a role in autophagy, the cellular process responsible for degrading and recycling damaged or unnecessary cellular components.

The investigational gene therapy uses an Adeno-Associated Virus (AAV) vector to deliver a functional copy of the TECPR2 gene, with the aim of addressing the underlying genetic cause of the disease.

The programme is being advanced by a research team led by Steven Gray, Ph.D., director of the Gene Therapy Programme at UT Southwestern Medical Centre, and Xin Chen, M.D., Ph.D., assistant professor at UT Southwestern. The team is working to advance the programme toward clinical development.

The TECPR2 Research Foundation was founded by physicians and families to support research and development of treatments for TECPR2-related disorders, also known as SPG49 or HSAN9. The foundation works with clinicians, geneticists and biotechnology partners to advance therapeutic development for the condition.