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Cure Blindness Australia is very proud to have allocated $40,000 to support
The Vision Initiative (TVI), a specialist unit within the Children’s Medical Research Institute at Westmead. The research is specifically targeted at Retinitis Pigmentosa (RP) and related conditions.

TVI has the ambitious goal of restoring vision to patients with genetically determined blinding retinal disorders (RP). To meet this ambitious goal, TVI will bring together researchers with international expertise to apply breakthroughs in genome engineering, sequencing, vector and stem cell technology; clinical,
social, and economic tools; and community and industry connections to create a new consortium that will address this currently insoluble problem leading to lifelong blindness.

There are significant problems to overcome to treat the blindness that occurs from RP. There is a need for improved methods of delivering treatments to the relevant retinal cells. In addition, an efficient strategy is needed to identify the most effective treatment mode such as knockdown or correction of genetic variants in disease models, which can be applied to human retinal tissues. The TVI team will address these problems, in combination with plans for clinical trials and assessment of the societal and economic value of the work, to maximise research outcomes and translate to therapies for blindness.

The eye is an ideal organ to apply new genome engineering, AAV, stem cell and other therapeutic technologies, due to its relatively small size (so less demand on the scale-up of the technology), and the ease of accessibility. The Chief Investigators (CIs) have collaborations in place with national and international leaders in AAV and stem cell technologies, as well as clinical trial collaborators, and social, ethics and economic researchers, which will contribute to this research program as the TVI progresses. Industry partnerships of the CIs in vector technology will facilitate scale-up and future commercial opportunities. The CIs have strong community relationships with Cure Blindness Australia Inc, and service groups committed to improved treatments for patients with blindness.

Specific outcomes in the
first 12-18 months will be:

1. Facilitation of a new collaborative approach to maximise research excellence
    and leadership in the area of treatment of blindness where no therapy is

    currently available

2. Pilot data and research outputs in areas including:

•  Establishment of methods of maximised delivery of treatment modality using AAV technology

•  Development of constructs for use in treatment pipeline

•  Establishment of health, economic and social impact data to address the economic and social issues
    related to RP diagnosis and treatment, and development of approaches to explore the ethical and          legal issues around novel diagnostics and therapies.

•  Investigator workshops to facilitate and coordinate the cross-disciplinary collaborative research
    to be undertaken in the TVI.

•  Workshops with patient support organisations
    e.g. Cure Blindness Australia, to facilitate consumer engagement and recruitment,

3. Future grant applications will be facilitated by the pilot data
    and will allow applications in the areas of:

•  the various types of RP, where the advances it makes in AAV, genome engineering,
    stem cell technologies, ethical and societal approaches will have specific applications.

•  clinical trials of new treatment applications in RP.

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