RNA therapeutics · Innate immunity · CNS
RNOVA Tx is developing cell-targeted antisense therapeutics designed to modulate SRSF3 and reprogram dysfunctional innate immune responses in neurodegenerative disease.
Conceptual illustration — not experimental data.
01 The challenge
Microglia are the resident immune cells of the central nervous system (CNS). They maintain tissue homeostasis, survey their environment and respond to injury.
In chronic neurodegenerative disease, microglia can become persistently activated and functionally dysregulated. Published work in ALS models shows that, over the course of disease, they progressively lose protective immune functions such as phagocytosis.
RNOVA Tx focuses on that loss of immune competence — not simply on “too much inflammation”.
Conceptual transition of microglial function in chronic disease. Conceptual illustration — not experimental data.
02 The translational brake
In activated microglia, the most highly induced innate immune mRNAs can reach the ribosome yet fail to become protein. The team’s published work identified the RNA-binding protein SRSF3 at this regulatory checkpoint.
Innate immune challenge strongly increases transcription of selected immune genes.
The transcripts are exported and associate with ribosomes in the cytoplasm.
A cluster of highly upregulated immune transcripts is not translated into protein.
Repression acts through the transcripts’ 3′UTR and involves the RNA-binding protein SRSF3.
Reducing SRSF3 in experimental systems restored synthesis of selected immune proteins.
Sources Boutej et al., Cell Reports, 2017 · Rahimian et al., Molecular Therapy, 2024 Explore the published science →
03 The RNOVA approach
Rather than broadly suppressing inflammatory signalling, RNOVA Tx is developing SRSF3-directed antisense therapeutics that aim to restore functional innate immune responses at the level of RNA translation.
Antisense oligonucleotides designed to reduce SRSF3 in innate immune cells.
Aim: lift translational repression of selected immune transcripts.
De novo immune protein synthesis has been observed in experimental systems.
Goal: a more functional innate immune response in CNS disease.
All RNOVA Tx programs are at the research and preclinical stage.
04 Targeted RNA therapeutics
Antisense strategy
SRSF3-directed antisense oligonucleotides conjugated to a cell-penetrating peptide to support uptake into immune cells. Built on the target biology described in RNOVA’s published research and foundational patent.
Next-generation targeting
An emerging cell-targeted delivery strategy designed to improve therapeutic access of SRSF3-directed antisense to selected innate immune cell populations.
05 Therapeutic reach
ALS is our lead indication. Because dysfunctional innate immunity is a shared feature of many neurodegenerative diseases, the SRSF3 approach may extend to related CNS disorders.
Amyotrophic lateral sclerosis — the focus of RNOVA Tx’s lead program and the disease in which microglial dysfunction has been characterized by the team.
Including the ALS–frontotemporal dementia (FTD) spectrum and other conditions with microglial dysfunction.
Under exploratory investigation. Validation is less advanced than for ALS.
Indications beyond ALS are exploratory and have not reached the same level of experimental validation.
06 Built on published science
07 Pipeline
08 From discovery toward translation
RNOVA Tx welcomes conversations with pharmaceutical, biotechnology, scientific and development partners interested in RNA therapeutics and neurodegenerative disease.