TRUST GROWS
WHEN WE
SHOW THE
WHOLE STORY.
Every contribution carries trust. We believe that trust deserves clarity — about the research, the funding, the progress, the challenges and the road ahead.
Transparency is not something we add later. It is part of the responsibility we accept with every donation.
THIS IS
PERSONAL.
GAND Foundation was created because one family was suddenly faced with a diagnosis that changed everything.
That personal beginning is also why openness matters so much. People supporting the Foundation are not simply funding research. They are placing trust in the people carrying the work forward.
People do not donate only money. They give us their trust.
THAT TRUST DESERVES ACCOUNTABILITY.
WHERE WE
STAND TODAY.
Gene therapy development takes place in defined stages. Each stage requires research, specialist expertise, testing, manufacturing and regulatory preparation.
raised toward the development of the GAND gene therapy program
Development costs are estimates. They may evolve as new scientific data, manufacturing requirements and regulatory needs become clearer.
PROGRESS
HAPPENS
STEP BY STEP.
Each phase exists for a reason.
Every stage must answer scientific and safety questions before the program can responsibly move forward.
DISCOVERY &
PROOF OF CONCEPT
Construct development, in-vitro research, disease-model development and the scientific foundation required for translational development.
PRECLINICAL
VALIDATION
Validation and efficacy work designed to determine whether the evidence supports progression into IND-enabling development.
IND-ENABLING
DEVELOPMENT
Manufacturing, toxicology, biodistribution and regulatory preparation required before potential first-in-human development.
Additional operational and early clinical activities form part of the wider development budget.
THERE IS
A CHILD
WAITING.
Scientific development takes time. Families experience that time differently.
WHAT HAS TO
HAPPEN NEXT.
Progress is not automatic.
Each development stage must produce enough evidence to justify moving into the next one.
PROGRAM INITIATION
Program launch, construct work and development of disease models.
PRECLINICAL DEVELOPMENT
Validation, efficacy studies and continued scientific development.
GO / NO-GO DECISION
Available data are reviewed before committing to IND-enabling development.
IND-ENABLING DEVELOPMENT
Manufacturing, toxicology, biodistribution and regulatory preparation.
REGULATORY READINESS
Preparation of the development package required for potential clinical progression.
WE DO NOT
PROMISE
WHAT SCIENCE
HAS NOT YET
PROVEN.
Before moving into the next major stage, the evidence has to support progression.
THE RULES
EXIST FOR
A REASON.
Gene therapies are advanced medicinal products.
Development requires evidence across product quality, non-clinical research, clinical development and regulatory review.
A WELL-DEFINED
PRODUCT
Manufacturing, characterisation, potency, purity and consistency form part of the development process.
EVIDENCE BEFORE
HUMAN USE
Laboratory and biological models help evaluate activity, distribution and safety.
SAFETY &
EFFICACY
Clinical development evaluates dose, therapeutic effect and safety.
INDEPENDENT
REVIEW
Advanced therapies undergo formal scientific and regulatory assessment before authorisation.
ACCOUNTABILITY
IS PERSONAL.
Every contribution should have a clear purpose and a visible story.
People supporting the Foundation deserve to understand what their contribution supports.
Our goal is to communicate the development process, funding needs, scientific milestones and challenges as openly as possible.
DON'T JUST
TAKE OUR
WORD FOR IT.
The original documents behind the information on this page are available for anyone who wants to look deeper.
Development roadmap and funding plan
Program phases, timeline, activities and projected development costs.
Guideline on gene therapy medicinal products
Quality, non-clinical and clinical considerations for gene therapy medicinal products.
Regulatory aspects for the approval of ATMPs
Overview of the European regulatory pathway for advanced therapy medicinal products.
THANK YOU
FOR TRUSTING
THE JOURNEY.
We will continue to share what we know, what we achieve and what still needs to happen.
Support the research