Genetically-defined targets

RNA-targeted drugs

decoded by AI

Virtually 0% of the human genome is therapeutically addressed today
We are overcoming these limits. We resolve the whole genome, not just the exome, for novel disease targets. We then design precision therapeutics (GiTx) to drug these targets at the transcript, sidestepping undruggability limitations.
Announcing the GiTx umbrella of program-specific subsidiaries
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Our technology

Our platform supports our two core beliefs to (1) explore the unexplored 99% of the genome to identify genetically-defined targets and (2) drug the target at the RNA transcript stage using small molecule or oligonucleotide modalities
The Genetic Intelligence
To produce precision therapeutics, our platform relies on four primary layers: Bergspitze, Franklin, Orisha, and Lea. Each layer and its sub-components have been carefully built and validated to address specific challenges of the drug discovery process.
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Tames the noise of the whole genome to pinpoint genetic positions causal of disease.

Employs dynamic Bayesian networks, non-linear clustering, and neural nets.
Provides coherent etiology model for the disease and confirms targets to modulate for a cure, whether the causal genetic lesion or derivative nodes in the attendant biological pathway.

Employs gene regulatory networks (GRNs), knowledge graphs, neural nets, and interactive visualizations.
Designs RNA-targeted molecules to drug a target’s transcript at the sequence level (oligonucleotides) and/or at the structure level (small molecules).

Employs knowledge graphs, clustering, and neural nets.
Confirms experimentally the advanced therapeutic candidates produced by the computational layers.

Employs patient-derived iPSC (stem cell) assays primarily, with animal models for pre-clinical safety confirmation.

Our pipeline

We are addressing the most incurable and devastating diseases head on
with GiTx (Genetic Intelligence therapies)
  • Amyotrophic lateral sclerosis
  • Alzheimer's disease
  • Idiopathic pulmonary fibrosis
  • Undisclosed
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