We engineer
biology at the
base level.
Helix combines precision base-editing, AI-driven target discovery and integrated GMP manufacturing to build curative genomic medicines.
Full-stack genomic medicine
Five integrated pillars from discovery to GMP clinic — owned end-to-end.
Genomic Precision
Base-editing at single-nucleotide resolution with <0.01% off-target events, validated across 2,400 targets.
Cell Engineering
Scalable CAR-T and iPSC manufacturing pipelines from concept to GMP batch in under 14 weeks.
AI Discovery Engine
Proprietary ML models trained on 38 billion protein-sequence pairs predict therapeutic target druggability.
Safety-by-Design
Every candidate passes our 7-layer genotoxicity screen before entering the development queue.
In-House CRO
Full-service preclinical suite: in vitro, in vivo, 14 species, bio-analytics, and regulatory documentation.
From discovery to clinic
Five programs. Two in preclinical. One heading to IND.
PCSK9 (Hypercholesterolaemia)
Lead candidate selected
ANGPTL3 (Hypertriglyceridaemia)
NHP tox study Q3 2026
TTR (Transthyretin amyloidosis)
IND filing expected Q1 2027
ATTR-CM (Cardiomyopathy)
Dose escalation planned
SCD-1 (Sickle Cell Disease)
Partnership discussion
Outcomes that speak for themselves
Phase I/II data from HLX-002 (NHP study) and benchmark comparison across platforms.
HLX-002 — Efficacy vs Placebo
% LDL-C reduction over 9 months (NHP, n=32)
Off-target frequency
% mean off-target events vs. platforms
Built by the world's top genomicists
Each card shows their role, field and publication record.
Dr. Amara Osei
CEO & Co-founder
Molecular biology, Harvard PhD
Dr. Lena Voss
CSO
CRISPR engineering, MIT post-doc
Dr. Jin-Ho Park
CTO
Computational biology, Stanford PhD
Dr. Sofia Reyes
VP Clinical
Gene therapy trials, UCSF
Dr. Karim Nabil
VP Discovery
Protein engineering, Pasteur
Dr. Priya Mehta
Chief Science Officer Emeritus
Genomics & bioinformatics
Peer-reviewed science
180+ publications. Selected highlights from 2024–2026.
| Title | Journal | Type | IF |
|---|---|---|---|
Single-base editing with sub-0.01% off-target frequency in primary human cells DOI: 10.1038/nbt.xxxxx · 2026 | Nature Biotechnology | Research article | 46.9 |
AI-guided target identification accelerates rare-disease candidate selection DOI: 10.1016/j.cell.xxxxxx · 2025 | Cell | Research article | 64.5 |
GMP-scale iPSC-derived CAR-T manufacturing: a 14-week protocol DOI: 10.1038/nmeth.xxxxx · 2025 | Nature Methods | Protocol | 48 |
In vivo TTR silencing with lipid-nanoparticle-delivered base editors DOI: 10.1056/NEJMoa.xxxxx · 2025 | NEJM | Clinical brief | 176.1 |
Safety-by-design: a 7-layer genotoxicity screening framework DOI: 10.1038/nm.xxxxx · 2024 | Nature Medicine | Methods | 82.9 |
Strategic partners & collaborators
How we build medicines
AI-powered target identification
Our HelixAI engine ingests multi-omics datasets from 180,000+ patient samples, ranks druggable targets by therapeutic index, and de-risks early pipeline decisions.
- Multi-omics data fusion
- Therapeutic index ranking
- Competitive landscape mapping
Join the next chapter of genomic medicine
Backed by a16z Bio, Foresite Capital, ARCH Venture and others. Request access to our investor data room or speak with our CFO directly.
Frequently asked questions
What makes Helix's base editing different from first-generation approaches?
Our HelixEdit platform couples an optimised cytosine/adenine base editor with a proprietary guide-RNA design algorithm. The result is sub-0.01% off-target frequency — over 20× better than published Cas9 benchmarks — validated in primary human hepatocytes, HSCs, and T cells.
How does Helix protect against unintended bystander edits?
Every target undergoes our 7-layer bystander screen: in silico prediction, amplicon-seq, Digenome-seq, CIRCLE-seq, GUIDE-seq, long-read WGS, and blinded third-party validation. Only candidates clearing all seven gates advance.
What is Helix's manufacturing strategy?
We run a fully integrated GMP facility in Cambridge, MA capable of producing autologous and allogeneic cell products. Our 14-week iPSC-to-infusion protocol has been validated for CAR-T, CAR-NK, and HSC programs.
Does Helix partner or out-license its technology?
Yes. We offer research licenses, co-development agreements, and full program acquisitions. Our partnership team can typically structure a term sheet within 8 weeks of first contact. Reach out at [email protected].
When do you expect the first IND filing?
HLX-003 (TTR amyloidosis) is on track for an IND filing in Q1 2027, with HLX-004 (ATTR-CM) entering Phase I dose escalation shortly after, pending FDA feedback.