Clinical-stage gene therapy · AAV & in vivo base editing
VitaGene Therapeutics designs single-dose genetic medicines that correct disease at its source. Six programmes across haematology, neuromuscular, retinal, neurological and metabolic disease — four of them already dosing patients across a 19-site global network.
Scroll — pipeline & clinical data
Backed by the funds
that backed the field
The VitaGene platform
One integrated stack — computational design, engineered capsids, a GMP backbone built for rare disease volumes, and a clinical engine that has dosed 312 participants without a single insertional event.
Deep mutational scanning and in-silico off-target models clear guide and cargo candidates before a single reagent is ordered. Every design ships with a comparable-genome analysis and a wet-lab confirmation panel.
The VitaVector™ capsid library is screened in NHP for tropism, then paired with a tissue-selective promoter so the payload lands in the tissue we intend — and largely stays out of the liver.
Suspension HEK293 in 200 L single-use bioreactors at three sites, with an analytical release panel that turns around in nine days instead of nine weeks. Enough capacity to supply every programme in the clinic.
First-in-human dose escalation with a protocolised 12-week immunomodulation regimen, followed by a five-year registry that tracks expression, immunity and integration sites for every participant.
Clinical pipeline
Filter by therapeutic area. Stage is as of the last data cut; next milestone is the event our investors are watching.
Clinical development
Gene therapy is a one-time intervention, so the clinical path is long by design. Here is exactly where each stage stands — and what closes it out.
Stage 01 · Discovery
Target selection, off-target clearance and GLP toxicology in two species before any regulatory filing.
Stage 02 · Phase I/II
Ascending single doses with a protocolised 12-week immunomodulation regimen, then cohort expansion.
Stage 03 · Phase III
Delayed-treatment control in severe haemophilia B, with annualised bleeding rate as the primary endpoint.
Stage 04 · Registration
BLA and MAA dossiers, plus the long-term follow-up registry regulators now expect of every gene therapy.
312 participants dosed · 4 programmes in the clinic · 0 treatment-related deaths and no insertional oncogenesis observed in five years of follow-up.
Registry cut-off 30 Jun 2026The research team
Founded in 2018 out of four academic labs, VitaGene still runs as a research organisation first — 71% of operating expense goes into R&D.
President & CEO
MD, PhD · Haematology
Twenty years from bench to BLA. Took VG-101 through Phase I/II and both FDA and EMA pre-submission meetings.
Selected papers
Chief Scientific Officer
PhD · Vector engineering
Built the VitaVector™ capsid library. Previously led AAV discovery at a top-ten gene therapy company; 41 papers on vector design.
Selected papers
Chief Medical Officer
MD · Gene therapy clinician
Has run nine first-in-human dose-escalation studies and sits on two independent data safety monitoring boards.
Selected papers
SVP · Research & gene editing
PhD · Base & prime editing
Leads the editing portfolio. Her lab produced the hepatocyte editing platform licensed into VG-402 and the HBB edit behind VG-620.
Selected papers
VP · Clinical operations
MD · Trial delivery
Runs the 19-site global network and the immunomodulation protocol. Previously delivered two gene therapy registrational studies on schedule.
Selected papers
Head · Translational medicine
MD · Rare disease
Thirty years in rare disease medicine. Owns the five-year long-term follow-up registry and every regulatory interaction on the VG-101 file.
Selected papersPublications
Our clinical claims live in journals, with the data attached. Six selected papers from the last four years — the complete bibliography runs to 74 entries and is available on request.
74
Peer-reviewed papers · 2019–2026
Authorship spans the whole team, not just the founders — 41 of the 74 papers carry a first author from the research group.
Request the full bibliography2026Phase I/II
n = 54
Osei A, Mehta A, Reinhardt C, Chen W-L, Ellis M, et al.
Nature Medicine · 32(4), 1181–1192
2026Registry
n = 291
Ellis M, Moreno R, Osei A, et al.
Science Translational Medicine · 18(735)
2025Platform
Chen W-L, Reinhardt C, et al.
Cell · 188(19), 5288–5304
2025First-in-human
n = 8
Osei A, Ellis M, Mehta A, et al.
New England Journal of Medicine · 393(21), 2044–2055
2024Immunology
n = 96
Moreno R, Mehta A, Chen W-L, et al.
Molecular Therapy · 32(11), 3877–3891
2023Platform
Reinhardt C, Chen W-L, Ellis M, et al.
Nature Biotechnology · 41(9), 1288–1299
Investor relations
Gene therapy pays back over a lifetime, not a quarter. VitaGene is funded into 2029, runs a 71% R&D ratio, and has four readouts inside the next eight quarters.
Cash, equivalents & investments
$486M
As of 30 Jun 2026
Cash runway
2029
Into H1, past VG-101 filing
Shares outstanding
92.4M
NASDAQ: VITA · no preferred overhang
R&D share of opex
71%
68 scientists in-house
Diligence packages, clinical data rooms and the KOL call schedule are released on request. Institutional enquiries only.
