About Lifelong VISION

In Lifelong VISION, we will combat vision loss by developing effective therapeutic approaches for IRD and MD, and by matching patient characteristics with their optimal treatment(s) at the right time.

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About Lifelong VISION

In Lifelong VISION, we will combat vision loss by developing effective therapeutic approaches for IRD and MD, and by matching patient characteristics with their optimal treatment(s) at the right time.

Blindness is considered the worst possible health outcome by at least half of the world’s population. Two devastating eye disorders are disrupting the lives of the young and of the old: inherited retinal dystrophies (IRD) and age-related macular degeneration (AMD). In the last decade, enormous efforts have been made to disentangle disease pathomechanisms and develop revolutionary therapies such as gene therapy and regenerative medicine, but these advances are still far out of reach for most patients. There is a clear need for innovations in conceptual and computational technology that enables us to shift into a higher gear and develop therapies that are customized to the cause, disease stage, and patient profile. Such technology is instrumental to achieve lifelong vision for all.

A scientific architecture

Lifelong VISION will generate a pipeline of therapies applicable to the entire trajectory of retinal degeneration in five domains which form a scientific architecture:  

  • Pillar 1 will develop and demonstrate proof-of-concept of gene-based therapies targeting DNA or RNA for permanent or transient repair of the genetic defect.
  • Pillar 2 will develop and demonstrate proof-of-concept of pathway-based therapies targeting prominent disease pathways which cause retinal degeneration.
  • Pillar 3 will develop and demonstrate proof-of-concept of cell-based therapies replacing lost retinal cells.
  • Platform A will deliver model systems that closely mimic the back of the eye.
  • Platform B will develop data-driven patient profiles tailored to each intervention modus and a core outcome set for clinical trials. 

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Our people

Learn more about the people involved in Lifelong VISION.

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What, how, and why


Objectives and approach

The overarching aim of the program is to combat vision loss by developing effective therapeutic approaches for IRD and MD, and by matching patient characteristics with their optimal treatment(s) at the right time.

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Objectives and approach

The overarching aim of the Lifelong VISION program is to combat vision loss by developing effective therapeutic approaches for IRD and MD, and by matching patient characteristics with their optimal treatment(s) at the right time.

Key objectives

The key objectives for Lifelong VISION are to:

  1. Develop innovative therapeutic solutions using gene-based, pathway-based and cell-based therapies: Gene-based therapies will target DNA or RNA for either permanent or transient effects, by applying exciting new technologies such as PRIME editing and delivery of protective genetic variants to combat disease. Pathway-based therapies will target shared disease mechanisms and common drivers to protect retinal cells from degeneration and will introduce a revolutionary new concept to activate retinal regeneration in vivo from endogenous cell sources. Cell-based therapies will be developed by generating retinal cells and multicellular scaffolds for transplantation using cutting-edge innovative technologies, such as bioprinting of live cells, and implementing new supramolecular materials for tissue engineering.
  2. Test these highly innovative experimental treatments in newly developed, robust and scalable model systems.
  3. Create integrated patient profiles and match these to optimal treatment using structure-function correlates developed by AI frameworks.

Concept of Lifelong VISION

The overarching concept of Lifelong VISION is that only by integration of all required research fields, led by experts who are at the top of those fields, can the much sought-after goal to overcome blindness from retinal disease be reached. Our consortium harbours a wide range of disciplines: molecular and cell biology, clinical ophthalmology, genetic therapy development, biological engineering, epidemiology, organoid systems, regenerative medicine, advanced imaging, and artificial intelligence. We will pioneer innovative therapeutic approaches in the eye, test them in model systems that have closer resemblance to the human eye than ever before, define the window of opportunity for each intervention, and offer a test set to investigate efficacy.  

The therapies that we will develop will benefit patients at any given disease stage and at any age during the disease course. At cell-level, IRD and AMD follow a rather similar disease pattern, although the onset, retinal characteristics, and aetiology clearly differs (see figure). At the start of the disease, distinct disease features will have appeared and cells will have become dysfunctional, but they will still be alive. This stage is very suitable for repair of the causal problem, i.e. improve the gene defect or initial disease pathway. During disease progression, the disease is clearly on a path towards cell death but the integrity of the retina is still largely intact. This stage will be very appropriate for therapy that ultimately alters cell programming and keeps the cells alive. At the end stage of retinal disease, photoreceptors, retinal pigment epithelium and often neurons in neighbouring retinal layers will have died. Generation of new cells at locations where they are missing is then the ideal solution.

Consortium bodies

Curious about the consortium bodies working towards these objectives? Learn more at Lifelong VISION Bodies and organization.


Results and impact

The consortium will generate a medical toolbox of solutions and approaches to combat vision loss.

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Results and impact

The Lifelong VISION consortium will generate a medical toolbox of gene-based and pathway-based therapeutic solutions and innovative regenerative approaches to combat vision loss. By designing such targeted therapies and defining patient profiles to determine appropriate, effective, and accessible therapy, this research program has enormous potential for exploitation and clinical translation. Technologies and strategies pioneered in the Lifelong VISION consortium will serve as a blueprint for development of therapeutic solutions to other devastating disorders. Thus, the strategy of the Lifelong VISION program has the potential to radically improve prospects for patients suffering from blindness, and can serve as a show case for the entire biomedical community.

Publications

Curious to see what results the consortium has already been able to publish? Please take a look at our collection of publications (bestaat nog niet, anders hier linken).


Consortium and program

Learn more about the organizational structure in which the consortium operates.

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Events

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