Series B — $210M raised

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.

Explore pipeline See trial data

CRISPR-based editing Cell & gene therapy AI-driven discovery Series B — $210M

5

Pipeline assets

180+

Publications

<0.01%

Off-target rate

EDIT PRECISION

<0.01%

off-target frequency

AI TARGETS

2,400+

validated targets

Platform

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.

Core tech

Cell Engineering

Scalable CAR-T and iPSC manufacturing pipelines from concept to GMP batch in under 14 weeks.

Manufacturing

AI Discovery Engine

Proprietary ML models trained on 38 billion protein-sequence pairs predict therapeutic target druggability.

AI / ML

Safety-by-Design

Every candidate passes our 7-layer genotoxicity screen before entering the development queue.

Safety

In-House CRO

Full-service preclinical suite: in vitro, in vivo, 14 species, bio-analytics, and regulatory documentation.

Preclinical

Research pipeline

From discovery to clinic

Five programs. Two in preclinical. One heading to IND.

HLX-001 Discovery

PCSK9 (Hypercholesterolaemia)

Lead candidate selected

100%

HLX-002 Preclinical

ANGPTL3 (Hypertriglyceridaemia)

NHP tox study Q3 2026

80%

HLX-003 IND-enabling

TTR (Transthyretin amyloidosis)

IND filing expected Q1 2027

55%

HLX-004 Phase I

ATTR-CM (Cardiomyopathy)

Dose escalation planned

20%

HLX-005 Phase II/III

SCD-1 (Sickle Cell Disease)

Partnership discussion

0%

Data & results

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)

Phase I data

Off-target frequency

% mean off-target events vs. platforms

93%

Max LDL-C reduction

0.008%

Off-target rate

32

NHP subjects

9 mo

Durable response

Scientific team

Built by the world's top genomicists

Hover any card to see their 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

Publications

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

Roche GSK Moderna BioNTech AstraZeneca Novartis Pfizer Sanofi Illumina BROAD Institute

Our process

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

Precision base editing

Our proprietary editors achieve single-nucleotide resolution across diverse cell types. Automated delivery optimisation selects the best LNP or viral vector for each candidate.

Single-nucleotide precision
LNP + AAV delivery
Off-target <0.01%

Rapid in vivo validation

A fully in-house preclinical suite compresses the path from lead candidate to IND-enabling package from 3 years to under 9 months via parallel study execution.

In-house GLP toxicology
Parallel multi-species studies
Integrated bioanalytics

Clinical development

Helix operates as its own CRO for Phase I/II. Real-time genomic biomarker monitoring enables adaptive dose decisions and faster regulatory milestones.

Adaptive trial design
Genomic biomarker suite
FDA/EMA alignment

5

Pipeline candidates

$210M

Series B raised

180+

Peer-reviewed papers

14wk

Cell mfg cycle time

Investor relations

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.

Request data room Schedule call

a16z Bio Foresite Capital ARCH Venture GV Nextech Invest

FAQ

Frequently asked questions

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.

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.

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.

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].

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.

We engineer biology at the base level.