Precision RNA Medicines Built to Scale
Elenae Therapeutics combines the native multi-organ biodistribution of LNA ASO mixmer chemistry with an AI-guided design engine to deliver potent, safe RNA medicines to tissues historically beyond the reach of oligonucleotide therapeutics.
Three major acquisitions totaling ~$15B in the last 18 months validate mixmer ASO chemistry, miRNA targeting, and muscle/cardiac delivery.
Despite proven success, today’s RNA medicine delivery platforms remain limited, complex, and costly.
Elenae’s LNA mixmers deliver naked, in saline, to multiple organs—no formulation, no conjugate, no receptor dependency.
LNA ASO mixmers use a steric-blocking mechanism with no RISC loading and no RNase H cleavage. The ASO binds its target RNA through high-affinity LNA base-pairing and physically blocks biological processes without cutting the RNA.
No cleavage-associated toxicity. Enables exon skipping, splice modulation, miRNA inhibition, and translational regulation from a single chemistry.
Heart, muscle, lung, retina, kidney, liver, and adipose reached after SC dosing in saline. No LNP, no GalNAc, no antibody conjugate required.
24-week NHP chronic dosing at 5 mg/kg/week SC. No hepatotoxicity, nephrotoxicity, immune activation, or injection site reactions across three species.
Dual engines for miRNA targeting and exon skipping. 15 years of proprietary parameter libraries. Target-to-candidate in weeks. Iterative optimization from experimental data.
Validated across mouse, pig, and nonhuman primate models using subcutaneous administration in saline.
| Study | Species | Duration | Dose | Result |
|---|---|---|---|---|
| Chronic toxicity (N=4) | Cynomolgus NHP | 24 weeks | 5 mg/kg/wk SC | No hepatotox, nephrotox, immune activation; stable body weight |
| Safety follow-up (N=7) | Cynomolgus NHP | 20 weeks | 5 mg/kg/wk SC | Confirmed clean safety |
| DMD efficacy (N=4) | Pig | 8 weeks | 5 mg/kg/wk SC | Cardiac function rescued; no adverse effects |
| Human precedent | — | Phase I-II | Various | Cardior, Santaris, miRagen: clean safety in humans |
Data from oncogene exon-skipping program — different ASO, same LNA mixmer chemistry platform.
Tolerability: Weight gain, ALT, AST, creatinine, BUN — all non-significant vs saline control.
Pharmacokinetics: Measurable serum concentration time-course with confirmed solid tissue drug levels across target organs.
After a single subcutaneous dose of naked ASO in saline, LNA mixmers distribute broadly across major organs. Intrinsic to the chemistry, not engineered per-tissue. Compatible with conjugation strategies when tissue-restricted delivery is desired.
Target engagement measured by miR-128-3p knockdown. Distribution intrinsic to LNA mixmer chemistry.
Elenae advances transformative clinical assets internally while deploying the platform through accelerated target-nomination design sprints for pharma partners.
Three independent clinical programs using LNA mixmer ASOs have demonstrated clean human safety profiles, establishing the regulatory foundation for Elenae's platform.
Anti-miR-132 for chronic heart failure. Phase II. LNA mixmer cardiac program with clean safety. Acquired by Novo Nordisk for $1.1B in 2024.
Anti-miR-122 for HCV. First anti-miR drug in clinical development. Clean safety through multiple dosing cohorts. Phase II completed.
Anti-miR-155 for CTCL. LNA mixmer with clean safety in oncology. Demonstrated tolerability of the chemistry class in human subjects.
Professor of Metabolic Biology, UC Berkeley (formerly Harvard/MGH). 25+ years in transcriptional regulation, miRNA biology, and metabolic disease. Pioneer in LNA ASO mixmer therapeutics. Inventor on multiple patents.
Extensive experience with preclinical studies focusing on metabolic homeostasis and therapeutic approaches targeting metabolic dysfunction.
Executive Director, Solve FSHD. Extensive neuromuscular drug development experience from preclinical through human Phase 1/2 trials at Pfizer, Cytokinetics, and NMD Pharma.
Extensive oligo CMC and drug development experience, serving as consultant for numerous drug and biotech companies.
Extensive LNA ASO drug development experience from BMS, Wave Life Sciences, and Deep Genomics.
Cardiology Clinical Research Director and Advanced Heart Failure Comprehensive Care Center Chief at UCSF.
CEO and Founder, Kimia Therapeutics. Co-founder of multiple biotech companies, including Carmot Therapeutics (acquired by Roche for $2.7B).
Former Partner at The Column Group. Extensive experience in biotech and venture capital, board member at multiple biotech startups.
Professor at UCSD School of Medicine, Dept. of Cellular & Molecular Medicine. Specializes in RNAi therapeutics. Biotech founder, serves on SABs for biotechs and pharma.
Executive Director, UC Berkeley Molecular Therapeutics Initiative and founding Director of the UC Berkeley Drug Discovery Center. 10+ years industry experience, 5+ molecules in the clinic.
RegenMed Advisors, LLC. Regulatory strategy and FDA guidance for advanced therapeutics.
Woodward Consulting, LLC. 20 years early-stage life science consulting. 10 years Partner, Ventac Partners A/S. Variety of life science CxO roles.
We are actively engaging with pharmaceutical companies interested in deploying steric-block LNA mixmer chemistry against extrahepatic RNA targets through our target-nomination design sprint.
anders@elenaetx.com
