Controlled gene therapy to reverse Rett syndrome
First controlled gene therapy for Rett syndrome using next-generation CNS delivery.
Our therapy
Our therapy


Indication

Our therapy
A best-in-class therapy that controls the dose, matches the cell type, and reaches the whole brain
Rett syndrome traces to a single gene, MECP2. Reverta restores it the way a healthy body would.
Full-length gene
The complete MECP2 gene, not a shortened version, so it can do its full job.
Controlled dose
Built-in regulation holds expression in the correct therapeutic window and avoids the overexpression that has limited other approaches.
Whole-brain delivery
One intravenous infusion carries the gene across the whole brain. More effective, minimally invasive, no neurosurgery.
Cell-type matching
Expression matches how each brain cell naturally uses the gene, instead of one dose for all.
Where we are
Our science works and the data is strong. Now it is all about execution
We have finished the research phase and confirmed the approach in disease models. The program is now in preclinical development, with a defined path to first-in-human trials.
Partners

RESEARCH PARTNERSHIP
The largest worldwide funder of Rett research and leading partner for genetic medicine scientists and companies.

INVESTOR
Extend is a multidisciplinary technology transfer platform dedicated to transforming life sciences research into impactful biotech startups.

Team
A purpose-built team of world class researchers and serial entrepreneurs
More than 60 years of combined gene therapy experience across science, clinical development, regulatory affairs, and manufacturing.

Jeffery S. Vick
CO-FOUNDER & CEO
Co-founder of four biotechs and a former biotech CEO, with deep experience scaling gene therapy companies.

Vania Broccoli
CCO-FOUNDER & SCIENTIFIC ADVISOR
Head of Stem Cells and Neurogenesis at the San Raffaele Scientific Institute and a leading MECP2 researcher.

Carlo Bertozzi
CO-FOUNDER & CHAIRMAN
Founder and chief executive of multiple biotechnology companies across Europe.

Mary Newman
ADVISOR, CLINICAL & REGULATORY
Former chief development officer and senior regulatory leader in gene therapy.

Matthias Hebben
ADVISOR, CMC & MANUFACTURING
Gene therapy technology and manufacturing leader, formerly head of AAV technology and CMC at a leading institute.
Our vision

We are currently raising our Series A to finally develop a cure for Rett.
If you are interested to invest or partner with us, don't hesitate to reach out.

