Governance

Charitable objects

Verbatim from our constitution (Clause 3). Registration does not mean the Charity Commission endorses our work.

Within our powers (not live programmes yet), the CIO may among other things fund or contribute to medical treatments, therapies, clinical trials, equipment, and travel for beneficiaries accessing treatment in the UK or overseas — always for public benefit under the objects.

Research grants

We have not yet awarded research grants. The sections below summarise trustee policy we will apply before any awards. They encode our Research Selection Criteria and Research Governance Framework. Family support remains available for all forms of Batten disease / NCL; research prioritisation has a particular focus on CLN2, without excluding other NCL research if trustees choose to fund it.

Research priorities

The charity will prioritise research that:

• Directly addresses Batten Disease (NCL), with particular focus on CLN2 disease
• Has potential to improve patient outcomes or quality of life
• Addresses gaps in current treatment options
• Includes gene therapy, enzyme replacement therapy, or other disease-modifying approaches
• Supports earlier diagnosis or improved disease management

Essential criteria (all must be met)

1. Scientific merit — sound scientific principles and methodology
2. Ethical approval — appropriate approval from a recognised body
3. Qualified researchers — appropriately qualified individuals at a recognised institution
4. Publication commitment — commitment to publish results in peer-reviewed journals
5. Relevance — directly relevant to Batten Disease / NCL

Desirable criteria

• Track record of the research team in NCL or related conditions
• Collaboration with established NCL research centres
• Potential for translational impact (bench to bedside)
• Leverage of additional funding from other sources
• Patient and family involvement in research design

Assessment process

1. Initial review — trustees assess applications against essential criteria
2. Expert review — applications meeting essential criteria are reviewed by independent scientific experts
3. Trustee decision — final funding decisions by trustees, based on expert recommendations and available funds
4. Feedback — all applicants receive feedback

Conflicts of interest

Any trustee with a personal or professional connection to a research application will declare the conflict and withdraw from assessment and decision-making for that application.

Governance principles for funded research

All research funded by Anisia's Hope must adhere to:

• The Declaration of Helsinki principles for medical research
• UK Research Integrity Office (UKRIO) Code of Practice
• Good Clinical Practice (GCP) guidelines where applicable
• Relevant data protection legislation (UK GDPR)

Approach to research funding

• Fund research at established academic and clinical institutions with proven track records
• Engage independent scientific reviewers to assess funding applications
• Aim to build a scientific advisory panel to guide research priorities
• Rely on institutional governance frameworks at host research organisations
• Focus on research with clear potential to benefit patients with Batten Disease

Pre-funding requirements

Before funding is released, researchers must provide:

1. Ethical approval — evidence of approval from an appropriate Research Ethics Committee
2. Institutional approval — confirmation from the host institution agreeing to sponsor the research
3. Protocol — detailed research protocol including methodology, timeline, and milestones
4. Budget — detailed budget breakdown with justification
5. Publication plan — commitment to publish results regardless of outcome

Monitoring and reporting

Funded researchers must provide:

• Regular progress reports (frequency agreed at funding)
• Financial / expenditure reports with progress reports
• A final report on completion
• Copies of any publications arising from the research
• Acknowledgment of charity funding in publications

Any significant changes to protocol or budget require written approval from trustees.

Publication requirements

Funded researchers must:

• Publish results in peer-reviewed journals, whether results are positive or negative
• Acknowledge funding from Anisia's Hope in all publications
• Provide the charity with copies of all publications arising from funded research
• Provide lay summaries suitable for sharing with patients and families

Review of this framework

Trustees will review this research framework annually and update it as necessary to reflect best practice.

How we keep education accurate

Educational materials and public communications should be accurate, evidence-based, and appropriate. Trustees will draw on recognised expert sources and quality checks. We do not give medical advice.

Evidence sources (examples)

• Medical literature: PubMed / MEDLINE, Orphanet, OMIM, GeneReviews
• Specialist centres and resources: Great Ormond Street Hospital (GOSH); UCL Great Ormond Street Institute of Child Health; UCL NCL Resource; University Medical Center Hamburg-Eppendorf (UKE); NCL Mutation Database
• Patient organisations (signposts): Batten Disease Family Association (BDFA) — UK charity 1084908; Batten Disease Support and Research Association (BDSRA); NCL Stiftung (NCL Foundation).
• Guidelines and regulatory information: NHS England Highly Specialised Services; European Medicines Agency (EMA); NICE where applicable; ClinicalTrials.gov

Professional consultation

Trustees will seek input from qualified professionals as needed — for example consultant paediatric neurologists with NCL experience, clinical geneticists, metabolic disease specialists, and NCL research scientists. We do not invent advisor names on this site.

Quality assurance for educational materials

1. Reviewed against peer-reviewed sources before publication
2. Checked by a medical advisor where clinical content is involved
3. Updated when new evidence or guidance becomes available
4. Clearly dated to indicate currency of information
5. Cross-referenced with BDFA materials where appropriate for consistency

What we will not claim

We do not claim cures or clinical results we cannot substantiate. We do not give medical advice. Family crowdfunding (including JustGiving campaigns for personalised medicine) is separate from this charity’s accounts unless a future appeal says otherwise in clear terms. Charity funds exist for public benefit under our objects — not as a private trial fund.