Three initial programmes.
One platform thesis.

KritRNA’s initial focus is selected premature-stop contexts in HBB, DMD and TP53. Together, they test the platform across hematology, neuromuscular disease and cancer biology.

Discovery

Target context, candidate design and assay definition

β-Thalassemia

HBB · Hematology · India-priority
Active research

Design suppressor-tRNA candidates for selected HBB nonsense-mutation contexts and restore β-globin production.

Current milestone
Program definition
Next evidence gate
Candidate design and in-vitro assay specification

Duchenne muscular dystrophy

DMD · Neuromuscular
Active research

Design suppressor-tRNA candidates for selected DMD premature-stop contexts and restore full-length dystrophin.

Current milestone
Program definition
Next evidence gate
Candidate design and reporter-assay specification

p53-deficient cancer

TP53 · Oncology
Active research

Explore suppressor-tRNA strategies for selected TP53 nonsense mutations with the aim of restoring full-length p53 protein and tumour-suppressor function.

Current milestone
Program definition
Next evidence gate
Mutation prioritisation and disease-relevant functional-assay planning

Lead optimisation

Iterative sequence, activity and selectivity refinement

Programmes enter this stage only after the preceding evidence gate is met.

Preclinical

Disease-model efficacy, delivery and safety evidence

Programmes enter this stage only after the preceding evidence gate is met.

IND-enabling

Regulatory-grade development package

Programmes enter this stage only after the preceding evidence gate is met.

One platform, multiple disease classes.

The same translation-level strategy may be applied across different genes and disease areas. Each programme still requires its own sequence design, amino-acid choice, delivery strategy, safety analysis and experimental model.

Neuromuscular

DMD and selected inherited muscular dystrophies

Hematology

HBB and selected clotting-factor nonsense variants

Oncology

Selected tumour-suppressor nonsense mutations, including TP53

Metabolic / enzyme

Selected loss-of-function enzyme disorders

Ophthalmology

Selected inherited retinal disease contexts

Additional research contexts

Evaluated after sequence, delivery and safety review