The governance backbone for gene-editing clinical trials.

Run consent, audit, and data provenance for CRISPR trials on GxP-validated infrastructure built for 21 CFR Part 11, ICH E6(R3), and GDPR — sitting alongside the Medidata and Veeva systems you already run, not replacing them.

Become a design partner See what we're building

Built for trial sponsors, CROs, and authorised treatment centres running gene-editing programs — and the regulatory, clinical-ops, and security teams who have to sign off on them.

Why gene-editing trials break existing tools

Your CTMS wasn't built for this.

Gene-editing programs stack four demands that conventional trial software handles individually, at best — and never together.

01

Genomic patient selection

Eligibility depends on variant-level genomic analysis across federated datasets that legally can't be centralised. Your EDC can't score patients on data it's not allowed to hold.

02

Auditability regulators haven't seen before

Editing a human genome demands a level of tamper-evident provenance and consent traceability that spreadsheet-era audit trails simply don't provide.

03

Multi-site consent that can't be rewritten

Consent and provenance span jurisdictions and treatment centres. When a patient withdraws, that has to propagate everywhere — provably, and without anyone quietly altering the record.

04

Getting patients treated is an operations problem, not just a governance problem

Casgevy has 75+ authorised treatment centres but only ~60 patients treated in two years. The bottleneck is stem cell collection logistics, ATC capacity scheduling, and manufacturing slot coordination — problems your CTMS doesn't even try to solve.

How the three technologies divide the work

AI decides. CRISPR acts. Blockchain proves.

Not three technologies loosely "integrated" — three that each own a distinct, regulator-grounded stretch of the trial and hand off cleanly. The AI works out what to do, CRISPR does it, and every step in between is consented and provable.

AI · DECIDES Who to treat · what to edit · how to run the trial CRISPR · ACTS Engineer · manufacture · deliver the therapy ⇄ HAND-OFF EVENT Find & stratifypatients Designguide RNA Design theadaptive trial Engineerthe therapy Manufactureedited cells Treat &reinfuse BLOCKCHAIN · PROVES Consent · Provenance · Audit · Submission — every step recorded & irreversible Outcome data feeds back — the models keep improving
AI — decides CRISPR — acts Blockchain — proves (spans every stage)
What it does for your program

Outcomes, not modules.

Twelve regulatory-grounded capabilities, each mapped to a specific instrument. Here's what they mean for the people running the trial.

🧬

Find eligible patients without moving their genomes

Federated scoring and stratification across genomic datasets — Tier-1 data never leaves the originating institution.

Patient Intelligence · ICH E6(R3), GDPR, GINA
⚙️

Design guides with governed AI

Guide-RNA design, off-target prediction, and delivery recommendation — with the model governance the EU AI Act and FDA AI/ML guidance now require.

Molecular Intelligence · EU AI Act, FDA AI/ML
📐

Adapt the protocol without losing rigour

Adaptive design, simulation, interim analysis, and safety-signal detection under an ICH E9(R1) estimands framework.

Trial Design · ICH E6(R3), E9(R1)
✍️

Prove consent, always

Granular consent with a full, tamper-evident lifecycle — and 60-second withdrawal propagation across every capability. Smart-contract option available via Provenance+ upgrade.

Consent Governance · GDPR Art 6–9, HIPAA
🔗

Submit with a defensible chain of evidence

Hash-chained WORM trails and audit-timestamped filings that stand up to inspection. Blockchain timestamping available via Provenance+.

Data Provenance · 21 CFR Part 11
🏭

Keep chain of custody from vein to vein

LIMS/MES integration, batch records, and QC across manufacturing and reinfusion — patient-safety chain of custody intact.

Manufacturing Integration · 21 CFR 210/211
📈

Track outcomes for the full 15 years

Engraftment, biomarkers, adverse events, and long-term follow-up on the timelines gene-therapy guidance mandates.

Outcome Monitoring · FDA Gene Therapy (15yr)
📋

Assemble IND/IMPD packages faster

eCTD assembly, safety reports, and timestamped filings — the regulatory spine of the program in one place.

Regulatory Submission · FDA eCTD, ICH M4
🛡️

Give regulators read-only audit on demand

A dedicated inspection surface so an FDA/EMA/MHRA reviewer can verify trails and submissions without touching live data.

Identity & Access · 21 CFR Part 11, GDPR
🧪

Take a target from design to therapy

A versioned target-to-therapy design workflow with full design history — the scientific pipeline that feeds your IND.

Therapy Pipeline · FDA Gene Therapy, EMA ATMP
🏥

Qualify and activate sites for gene editing

ATC qualification, manufacturing-capability assessment, capacity scheduling, and patient-logistics coordination — the operational layer that gets more patients treated.

Site Qualification & Activation · ICH E6(R3), FDA Gene Therapy Guidance
📁

A trial master file built for gene editing

Standard TMF Reference Model compliance plus gene-editing-specific document types — gRNA design records, off-target analyses, batch certificates, provenance proofs — all anchored to the audit trail.

Trial Master File · ICH E6(R3), FDA/EMA TMF Guidance
For your Data Protection Officer

Right-to-erasure and an immutable ledger — reconciled.

HelixChain's default audit infrastructure uses conventional WORM storage — append-only records with cryptographic hashing. For organisations that want additional tamper-evidence, the Provenance+ upgrade adds blockchain-anchored audit. Here's how the GDPR erasure concern is resolved for blockchain deployments:

The objection every DPO raises about blockchain in a trial: you can't delete from an immutable ledger, so how do you honour a GDPR erasure request? HelixChain answers it structurally.

Personal data lives off-chain; only cryptographic hashes go on-chain. Erasing the off-chain record and destroying its per-record key renders the on-chain hash meaningless — practical erasure achieved, ledger integrity preserved. It's the difference between a platform your DPO blocks and one they can approve.

1
OFF-CHAIN STOREPatient personal data, encrypted per-record with its own key
↓
2
ON-CHAIN HASHOnly a cryptographic hash is written to the ledger
↓
3
ERASURE REQUESTDelete off-chain data + destroy the per-record key
↓
✓
RESULTHash is now meaningless. Ledger stays intact. GDPR satisfied.
Fits the stack you already run

It sits alongside your systems. It doesn't replace them.

Standards-first integration, with proprietary connectors only where no standard exists. Keep Medidata Rave and Veeva Vault; HelixChain adds the genomic, consent, and provenance layer they lack.

Connects toStandardDirection
Electronic Health RecordsHL7 FHIR R4Patient data in, outcomes out
Genomic data sourcesGA4GH htsget / DRSFederated variant queries in
EDC (Medidata Rave, Veeva Vault)CDISC ODM / SDTMBidirectional CRF exchange
LIMS / MESHL7 v2 / SiLA2Manufacturing data both ways
Regulatory portalseCTD / FHIRSubmission packages out
Your identity providerOAuth2 / OIDC / SAMLFederated single sign-on

Integration with existing EDC and institutional IT is real work — we scope it explicitly with you up front rather than pretending it's plug-and-play. Professional-services effort is quoted, not hidden.

For your security review

Security posture.

What's specified, what's planned, and what's on the certification roadmap — so your vendor assessment gets straight answers.

Genomic data (T1)
Never centralised. Federated access only, HSM-held keys.
Specified
Encryption
AES-256-GCM at rest · TLS 1.3 + mTLS in transit
Specified
Key management
HSM, FIPS 140-2 Level 3
Specified
Access control
RBAC + ABAC via Open Policy Agent, consent-aware
Specified
SOC 2 Type II
Controls in place; audit period underway
Planned — Year 1
ISO 27001
Targeted alongside SOC 2
Planned — Year 1
GAMP 5 validation
Computerised-system validation package
Planned — full platform
Blockchain audit acceptance
FDA / MHRA equivalence to GxP systems
Under active engagement

Security controls are specified in the architecture and will be validated during design-partner deployments. Certification timelines begin from first production deployment.

Why not just stitch point solutions together

The integrated coverage no single tool gives you.

Existing platforms each own a slice. Running a gene-editing trial across four of them means four integrations, four audit surfaces, and four vendors pointing at each other when something breaks.

PlatformPatient IntelMolecular IntelConsent GovManufacturingData ProvenanceSite Qual.TMF
Medidata (Dassault)Limited———PartialStrongStrong
Veeva SystemsBasic———PartialStrongMarket leader
Recursion PharmaStrongStrong—————
Insilico Medicine—Strong—————
ConsentChain / DWARNA——PoC only—PoC only——
HelixChainFullFullFullFullFullGene-editingGene-editing

Medidata and Veeva are strong on site qualification and TMF for conventional trials. HelixChain's versions are gene-editing-specific — ATC qualification, manufacturing-capability assessment, and gene-editing document types (gRNA design records, off-target analyses, provenance proofs).

A portal for every stakeholder

Everyone who signs off gets their own view.

Sponsor Portal

Full-program visibility for sponsors, biostatisticians, molecular biologists, regulatory affairs, and pharmacovigilance.

Trial Sponsor · Reg Affairs

Investigator Portal

Site-scoped data capture, patient care, and manufacturing for clinical investigators and manufacturing leads.

Clinical Investigator

Patient Portal

Consent, withdrawal, and own-data access for trial participants. Own data only.

Patient

Regulator Portal

Read-only audit surface for FDA/EMA/MHRA reviewers to inspect trails and submissions.

Regulator

The Team

Christian Macedo

Christian Macedo

Founder

Christian Macedo is a change and transformation leader with over 20 years designing operating models and delivering complex programmes across regulated industries — banking (HSBC, Lloyds, Standard Bank), travel, healthcare, and the public sector (NHS Test & Trace, Ofgem). He founded HelixChain to bring that discipline to gene-editing trials, pairing a capability-driven approach to modular system architecture with hands-on, AI-native product development. He holds a BSc (Hons) in Computing & Information Systems.

Work with us

Help shape the governance foundation for gene-editing trials.

We're building the Governance Foundation with a small group of treatment centres and sponsors running CRISPR programs. Design partners shape what we build, see it first, and help establish regulatory acceptance — before anyone else. Not ready for that? A 30-minute discovery conversation is a good place to start.

Tell us who you are and we'll follow up about the design-partner program, a discovery call, or the raise — whichever fits. No spam, no list.