Programs

Programs Built from the Platform

Internal clinical anchors plus the platform-generated opportunities and partnering channels that extend the Design Engine across additional RNA targets.

Internal Programs

ProgramAreaStage
ELN-128 (anti-miR-128)Early post-MI remodeling biologyNHP/Pig preclinical
ELN-128 expansion biologyDMD cardiomyopathy modelTranslational proof-of-concept
ELN-33 (anti-miR-33a)Dry AMDNHP preclinical
ELN-128 (Early post-MI remodeling biology). Early post-MI, post-reperfusion anti-miR-128 candidate designed to lower the risk of progression to heart failure in high-risk MI patients. A single SC dose 2 hours post-reperfusion improved LV ejection fraction, reduced ventricular volumes, and decreased infarct size over 28 days in mice. RNA-seq confirmed restoration of oxidative phosphorylation and suppression of inflammatory pathways. Does not rely on RNase H or RISC.

ELN-128 expansion biology (DMD cardiomyopathy model). Translational proof-of-concept for the anti-miR-128 mechanism in a clinically relevant pig DMD model. 8-week SC treatment improved cardiac function in a chronic cardiac failure phenotype, improved cardiac output, and reduced cardiac damage markers. Same chemistry and dosing route as the lead post-MI program.

ELN-33 (Dry AMD). Anti-miR-33a program targeting cholesterol efflux and retinal inflammation biology. Systemic SC dosing reaches retina in NHP. Supports evaluation of a systemic, non-intravitreal approach.
Platform-Generated Opportunities

Extending the Design Engine

AreaPublic framing
Splice modulationSteric-block ASOs for productive transcript restoration
Exon-skippingFrame-restoration and exon-definition applications for undruggable protein targets
Translation-modulating applicationsUnder confidential evaluation; discussed under CDA
Pharma partneringTarget-specific ASO design campaigns under collaboration
Clinical Precedent

Clinical Tolerability Precedent

Clinical programs using LNA mixmer ASOs provide human tolerability precedent for the chemistry class and help inform Elenae's development strategy.

$1.1B
Cardior / Novo Nordisk

CDR132L

Anti-miR-132 for chronic heart failure. Phase II. LNA mixmer cardiac program with clinical tolerability precedent. Acquired by Novo Nordisk for $1.1B in 2024.

Phase II
Santaris / Roche

Miravirsen

Anti-miR-122 for HCV. First anti-miR drug in clinical development. Reported tolerability through multiple dosing cohorts. Phase II completed.

Phase II
miRagen

Cobomarsen

Anti-miR-155 for CTCL. LNA mixmer evaluated clinically in oncology. Demonstrated tolerability of the chemistry class in human subjects.