Our Research
Our R&D activities are based on an antisense oligonucleotide (ASO) platform designed to target the precise molecular mechanisms involved in severe pathologies. Building on recent technological advances, we develop new therapeutic approaches to address major unmet medical needs.
Our approach
THX Pharma’s platform builds on recent breakthroughs in the treatment of severe diseases such as spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and certain forms of ALS linked to SOD1. These advances have demonstrated that it is possible to precisely target RNA and influence disease progression. Recent progress in ASO chemistry has also improved safety, affinity and biodistribution, providing a strong foundation for new therapeutic developments.
ASO :
Targeting capabilities
ASOs can be administered directly into the cerebrospinal fluid, enabling broad and homogeneous distribution throughout the central nervous system. They effectively reach neurons and glial cells, with long tissue half-lives compatible with less frequent dosing schedules.
These characteristics make ASOs particularly well-suitedto treat rare neurological diseases, where options remain limited. THX Pharma’s strategy focuses on this field to address major unmet medical needs.
Our ASO platform enables direct action on diseases’ molecular origins by targeting multiple mechanisms, including splicing defects, toxic RNA, cryptic intronic mutations, and post-transcriptional dysregulation. This approach makes it possible to restore protein function, bypass harmful mutations, or activate key cellular mechanisms such as autophagy.
New therapeutic opportunities
Thanks to their high programmability, ASOs can be designed and optimized for new therapeutic targets. Combined with modern computational approaches, this flexibility accelerates development and allows precise adaptation to specific mutations. It also enables exploration of new therapeutic opportunities, supporting the expansion of THX Pharma’s pipeline from rare diseases to broader indications.
Our Pipeline
TFEB Protein (THN-ASO 1)
A first target focused on the TFEB protein, in collaboration with Inserm and DIVERCHIM, to promote autophagy in lysosomal diseases.
Pipeline
Gliobastoma
A second target involved in glioblastoma progression, one of the most aggressive brain tumors in adults Glioblastoma has one of the most dire prognoses in oncology, with a median survival of approximately 15 months after diagnosis and a 5-year survival rate below 7%—compared with 13% for pancreatic cancer, 25% for lung cancer, and 90% for breast cancer. Each year, nearly 14,000 new cases are diagnosed in the United States. The median age at diagnosis is around 64 to 65 years.
