The TECPR2 Research Foundation, a nonprofit organization established to advance research and development of a treatment for patients with TECPR2, and Forge Biologics (Forge), a leading manufacturer of gene therapies and member of Ajinomoto Bio-Pharma Services, announced a development and manufacturing partnership to advance an AAV gene therapy for patients with TECPR2, an ultra-rare neurodegenerative genetic disease.
Under the partnership, Forge is providing process development, analytical development and qualification, and manufacturing services, including the manufacture and release of material for toxicology studies, to support the program’s continued advancement. The program is leveraging Forge’s proprietary FUEL platform, including its HEK293 suspension Ignition Cells, pEMBR 2.0 adenovirus helper plasmid, rep/cap plasmids, and program-specific optimization packages, designed to drive manufacturing efficiencies.
“For our families, this has never simply been a research program. It began with a few children and a commitment to do everything we could to create a path forward for everyone living with TECPR2,” said David Ogman, co-founder of the TECPR2 Research Foundation. “That mission has brought together an incredible group of scientists and partners who believe, as we do, that children with ultra-rare diseases deserve the same opportunity for scientific progress as anyone else.” “Forge shares that sense of purpose, and we are grateful to have their team and expertise behind us as we take these important steps toward a life-changing treatment,” said Michael Kaplan, M.D., co-founder of the TECPR2 Research Foundation.
TECPR2 is an ultra-rare genetic disorder associated with developmental delays and progressive neurological complications. TECPR2 plays an important role in autophagy, the cellular process responsible for degrading and recycling damaged or unnecessary cellular components. The investigational gene therapy is designed to use an AAV vector to deliver a functional copy of the TECPR2 gene, with the goal of addressing the underlying genetic cause of the disease.
The gene therapy program is being advanced by a research team led by AAV gene therapy pioneer Steven Gray, Ph.D., director of the gene therapy programme at UT Southwestern Medical Center, together with Xin Chen, M.D., Ph.D., Assistant Professor at UT Southwestern. Their research is focused on advancing the TECPR2 Research Foundation’s AAV gene therapy toward clinical development.
“At the heart of this partnership is a shared belief in what gene therapy can make possible for patients and families,” said John Maslowski, president and chief executive officer of Forge. “When we developed our FUEL platform, we set out to improve manufacturing efficiency so treatments could reach more patients, with the needs of both larger patient populations and ultra-rare diseases in mind. For programs like TECPR2, making the most of every manufacturing run is critical. We are honoured to bring our manufacturing technology and expertise to a program that is so deeply personal to the TECPR2 community.”
TECPR2-related disorder is an ultra-rare genetic neurodevelopmental and neurodegenerative condition caused by pathogenic variants in the TECPR2 gene. TECPR2 plays an important role in autophagy, a cellular process involved in the degradation and recycling of cellular components. The disease can cause developmental delays and progressive neurological complications, including respiratory dysfunction. The investigational TECPR2 gene therapy program uses an AAV vector to deliver a functional copy of the TECPR2 gene and is being developed with the goal of addressing the underlying genetic cause of the disease.
The TECPR2 Research Foundation is a nonprofit founded by physicians and families worldwide with a single mission: to advance research and curative treatments for TECPR2, a rare and fatal childhood disease also known as SPG49 or HSAN9. The foundation unites families, leading clinicians, geneticists, and biotech partners to accelerate first of its kind therapeutics for children living with the disease.
Forge Biologics is a gene therapy contract development and manufacturing organization (CDMO) enabling access to life-changing gene therapies by helping bring them from concept to reality. |