2024-12-31-accounts

Trustees’ Annual Report for the period

From 1st January 2024 Period start date To 31[st] December 2024 Period end date

Charity name: Cure CLCN4 LTD

Charity registration number: 1190344

Objectives and Activities

	SORP reference
Summary of the purposes of the charity as set out in its governing document	Para 1.17	Cure CLCN4 Limited is a not-for-profit organisation constituted with charitable purposes in perpetuity. The company is limited by guarantee and is governed by a memorandum and articles of association. The objectives of Cure CLCN4 Limited are: a) The advancement of health, relief of sickness and saving of lives by such means as the trustees see fit including but not exclusively through: i) Providing relief and assistance to people suffering from pathogenic variants of clcn4; and ii) The funding of medical research anywhere in the world for the purpose of discovering, developing and delivering treatment for patients with pathogenic variants of clcn4; and B) The furtherance of such other charitable purpose or charitable purposes as the trustees in their absolute discretion from time-to-time think fit, in each case for thepublic benefit.
Summary of the main activities in relation to those purposes for the public benefit, in particular, the activities, projects or services identified in the accounts.	Para 1.17 and 1.19	The Charity's objects are such exclusively charitable objects and purposes for the public benefit throughout England and Wales as the trustees may in their discretion think fit. The Charity furthers its charitable purposes for the benefit of the public by funding or undertaking scientific research for the advancement of knowledge of CLCN4 related condition and by supporting those affected by it.
Statement confirming whether the trustees have had regard to the guidance issued by the Charity Commission on public benefit	Para 1.18	The trustees have due regard to the Charity Commission's guidance as to operation of the public benefit requirement when exercising powers or duties to which the guidance is relevant and in their opinion the charity's activities are wholly for the benefit of the public.

Additional information (optional) You may choose to include further statements where relevant about:

SORP reference Para 1.38 Policy on grant making Para 1.38 Policy on social investment

including program related investment Para 1.38 Contribution made by volunteers Other

Achievements and Performance

	SORP reference
Summary of the main achievements of the charity, identifying the difference the charity’s work has made to the circumstances of its beneficiaries and any wider benefits to society as a whole.	Para 1.20	During 2024 Cure CLCN4 LTD awarded the first research grants, supporting three projects focused on better understanding the biology of CLCN4-related condition. We have also continued to support efforts aimed at developing important research tools, such as a ClC-4 antibody for use in basic research. We successfully organised the 3rdAnnual Cure CLCLN4 Scientific Conference, held in London. We have continued to expand and strengthen our research network. We also continue to support families and individuals living with CLCN4-related condition. In 2024 we organised the second online family meeting, which brought together over 50 families. Families had the chance to ask question to a panel of clinical and research experts. We have also continued to offer support in the form of accessible informational resources, opportunities to participate in research and to connect with the rest of the community.

Additional information (optional) You may choose to include further statements where relevant about:

Achievements against objectives set	Para 1.41
Performance of fundraising activities against objectives set	Para 1.41
Investment performance against objectives	Para 1.41

Other

Financial Review

Financial Review
Review of the charity’s financial position at the end of the period	Para 1.21	The charity’s financial position at the end of 2024 is healthy with a balance of 3,401GBP. This is the fourth year that the charity has an income. This revenue has allowed us to support the charity’s projects in the furtherance of our objectives including research and support of affected families.
Statement explaining the policy for holding reserves stating why they are held	Para 1.22	The trustees aim to maintain reserves at a level which equates to approximately five to six months of charitable expenditure. The trustees consider that this level will provide sufficient funds for operational needs and ensure that support and governance costs are covered. The trustees consider that a level of 5-6 months is sufficient. Although the reserves at 31 December 2024 fell below the level desired by the trustees this will be replenished in early 2024 by a cash donation from Peter Trill,a charitytrustee.
Amount of reserves held	Para 1.22	3,401GBP
Reasons for holding zero reserves	Para 1.22	N/A
Details of fund materially in deficit	Para 1.24	N/A
Explanation of any uncertainties about the charity continuing as a going concern	Para 1.23	No uncertainties.

Additional information (optional) You may choose to include further statements	Additional information (optional) You may choose to include further statements	where relevant about:
The charity’s principal sources of funds (including any fundraising)	Para 1.47
Investment policy and objectives including any social investment policy adopted	Para 1.46
A description of the principal risks facing the charity	Para 1.46
Other

Structure, Governance and Management

Description of charity’s trusts:
Type of governing document (trust deed, royal charter)	Para 1.25	Memorandum and Articles Incorporated 06 Feb 2020
How is the charity constituted? (e.g unincorporated association, CIO)	Para 1.25	Charitable company (limited by guarantee)
Trustee selection methods including details of any constitutional provisions e.g. election to post or name of any person or body entitled to appoint one or more trustees	Para 1.25	The Board shall be permitted to appoint by majority decision any individual who is eligible as a Trustee to fill a vacancy in their number or as an additional Trustee.

Additional information (optional) You may choose to include further statements where relevant about:

Policies and procedures adopted for the induction and training of trustees	Para 1.51
The charity’s organisational structure and any wider network with which the charity works	Para 1.51
Relationship with any related parties	Para 1.51
Other

Reference and Administrative details

Charity name	Cure CLCN4 LTD
Other name the charity uses
Registered charity number	1190344
Charity’s principal address	2 Duke Street London W1U 3EH

Names of the charity trustees who manage the charity

1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20	Trustee name	Office (if any)	Dates acted if not for whole **year **	Name of person (or body) entitled to appoint trustee (ifany)
	Peter James Alistair Trill
	Larry Louison
	Alan Wise
	Wang Sin Tan
	Alexis Karim
	Paul McGuinness

– Corporate trustees names of the directors at the date the report was approved

Director name Peter James Alistair Trill Larry Louison Alan Wise Wang Sin Tan Alexis Karim Paul McGuinness

Name of trustees holding title to property belonging to the charity

Trustee name Dates acted if not for whole year

Funds held as custodian trustees on behalf of others

Description of the assets N/A held in this capacity Name and objects of the N/A charity on whose behalf the assets are held and how this falls within the custodian charity’s objects Details of arrangements for N/A safe custody and segregation of such assets from the charity’s own assets

Additional information (optional)

Names and addresses of advisers (Optional information)

Type of Name Address adviser

Name of chief executive or names of senior staff members (Optional information)

Exemptions from disclosure

Reason for non-disclosure of key personnel details

Other optional information

Declarations

The trustees declare that they have approved the trustees’ report above.

Signed on behalf of the charity’s trustees

Signature(s) Full name(s) Peter James Alistair Trill Position (eg Secretary, Trustee, Chair Chair, etc) Date 28/09/2025

2024

Re ort & Financial statement p

(a company limited by guarantee)

Financial Statements for the period ended 31 December 2024

Charity registration number: 1190344

Company Number: 12447230

Table Of Contents

About Us...............................................................................p.3 Meet Our Founders...............................................................p.4 Meet Our Trustees................................................................p.5 Vision And Mission................................................................p.6 Legal & Administrative Information......................................p.7 Director’s Report................................................................p.8-9 Statement of financial activities for the period ended 31 December 2024....................................................................p.10 Balance sheet as at 31 December 2024..................................p.11 Notes to the accounts......................................................p.12-14 Our Activities. Highlights...............................................p.15-18 Contact Us..........................................................................p.19

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About Us

We are a registered charity aimed at providing support , raising awareness and funding medical and scientific research for effective treatments for CLCN4 -related condition.

CLCN4 is a rare genetic condition that causes intellectual disability and other problems such as epilepsy, behavioural disorders, movement disorders and microcephaly.

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Meet Our Founders

Cure CLCN4 was co-founded by Peter Trill and Gina Tan. Their journey began with the birth of their youngest child, Daphne, who was diagnosed with CLCN4 -related condition. Faced with limited knowledge and a lack of treatment options, they began connecting with other affected families and experts in the field. Recognising the urgent need for greater awareness and scientific progress, they established Cure CLCN4 to help drive research and support families impacted by the condition. Their vision continues to guide the charity’s mission to promote basic and translational research aimed at understanding CLCN4 and identifying effective treatments.

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Meet Our Trustees

PETER TRILL (CO-FOUNDER & TRUSTEE)

Peter is a co-founder of the UK registered Charity, Cure CLCN4. He is a father of three awesome children, the youngest of whom is affected by CLCN4. He is a company founder and board member working in the biotechnology sector. He is also a seed investor and mentor to founders of early-stage healthcare and technology companies

GINA TRILL (CO-FOUNDER & TRUSTEE)

Gina is a co-founder of Cure CLCN4, a medical clinician and mother to two girls, the younger being affected by CLCN4. She studied medicine at King’s College London and trained in hospitals in London and Bath, subspecialising in Rheumatology. She undertook clinical research training at Queen Mary University London.

ALAN WISE

(TRUSTEE)

Alan is a Trustee at Cure CLCN4. He is an experienced and successful biotech entrepreneur with in-depth knowledge of drug discovery. He is currently CEO of Duke Street Bio. Previous roles include CEO of the successful biotech, IOmet Pharma which was acquired by MSD in 2016 and senior drug discovery roles in large Pharma (GlaxoSmithKline).

ALEXIS KARIM (TRUSTEE)

Alexis is a Trustee at Cure CLCN4. He is also a Corporate Partner at Charles Russell Speechlys. He holds a strong interest in science and medicine and often seeks to combine this with his role as a lawyer.

LARRY LOUSION (TRUSTEE)

Larry is a Trustee at Cure CLCN4 and a partner at TC Group, based in the London office. He is a fellow of the Association of Chartered Certified Accountants with over 20 years experience in general practice. Being very passionate about the goals of CureCLCN4, Larry is committed to using his skills and experience to build a strong financial infrastructure within the company.

PAUL MCGUINNESS (TRUSTEE)

Paul began his career as a technology consultant at Apple at the launch of the original iPhone and App Store. He has founded and been involved in various tech start-ups and is passionate about the power of technology to improve the way we interact, work and live. Paul aims to bring his experience with technology platforms to grow awareness about CLCN4 and make a long term positive impact for families who have a loved one with the condition.

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Vision & Mission

Mission

Our mission is to enhance the understanding and treatment of CLCN4 -related condition through research, in addition to supporting affected individuals and their families, and driving awareness initiatives. We are committed to fostering collaboration among scientists, healthcare professionals, and communities to discover effective treatments and improve the lives of those impacted by CLCN4 -related condition.

Vision

Our vision is a world where a diagnosis of a CLCN4 -related condition comes with hope and support, not uncertainty. We aim to transform the landscape of CLCN4 research, ensuring that every affected family has access to precise, comprehensive information, effective disease-specific therapies, and a supportive community. By connecting families with each other and with leading experts, we aim to foster a strong network of advocacy and aid, empowering individuals and their loved ones to face this challenge with knowledge and collective strength.

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CURE CLCN4 (a company limited by guarantee)

Legal and administrative information

Governed by

Memorandum and Articles of Association dated 6 February 2020.

Directors

P Trill A Karim W Tan

A Wise

L Louison

P McGuinness

Registered Office

2 Duke Street London W1U 3EH

Accountants

TC Group 6th Floor, Kings House 9-10 Haymarket London United Kingdom SW1Y 4BP

Registered charity number

1190344

Company Number

12447230

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CURE CLCN4 (a company limited by guarantee)

Director’s re ort p

The Directors’ submit the organisations report for the period ended 31 December 2024.

STRUCTURE, GOVERNANCE AND MANAGEMENT

Governing document

The organisation is a charitable company limited by guarantee. The company was established under a memorandum of Association which sets out the objects and powers of the charitable company and is governed under its Articles of Association.

Recruitment, Appointment and Training of Trustees

Trustees can be nominated by any member of the Board of Trustees to replace a trustee stepping down.

Appointment of newly nominated Trustees requires unanimous approval of all Trustees.

The new Trustee is provided with details of Trustees’ roles and responsibilities. Training as such is not provided as no one has felt it to be necessary. At meetings, there is always room for questions and explanations.

OBJECTIVES AND ACTIVITIES

Charitable Objectives

Cure CLCN4 Limited is a not-for-profit organization constituted with charitable purposes in perpetuity. The company is limited by guarantee and is governed by a memorandum and articles of association. The objectives of Cure CLCN4 Limited are:

A) The advancement of health, relief of sickness, and saving of lives by such means as the trustees see fit including but not exclusively through:

i) Providing relief and assistance to people suffering from pathogenic variants of clcn4;

ii) The funding of medical research anywhere in the world for the purpose of discovering, developing, and delivering treatment for patients with pathogenic variants of clcn4;

B) The furtherance of such other charitable purpose or charitable purposes as the trustees in their absolute discretion from time to time think fit, in each case for the public benefit.

FINANCIAL REVIEW

This is our fifth year of operations, during which we have continued to facilitate the development of research tools, including the generation of patient-derived cell lines, as well as an antibody for use in basic research. Additionally, we have given out our first research grants; we are currently funding three research projects focused on better understanding the biology of CLCN 4-related condition. We also celebrated our third annual scientific conference, which has continued to grow in size and engagement. Our community of families has also grown and we continue to provide them with connection, information and resources, such as the recently launched Cure CLCN4 Welcome Pack, which contains up-to-date and accessible information on CLCN4-related condition.

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CURE CLCN4 (a company limited by guarantee)

Director’s report continued

STATEMENT OF DIRECTORS’ RESPONSIBILITIES

The Directors’ are required under company law to prepare financial statements for each financial year. Under that law the directors have elected to prepare the financial statements in accordance with United Kingdom Generally Accepted Accounting Practice (United Kingdom Accounting Standards and applicable law). Under company law Directors’ must not approve the financial statements unless they are satisfied that they give a true and fair view of the company and of the surplus or deficit of the company for that period. In preparing those financial statements the Directors’ are required to:

a) Select suitable accounting policies and then apply them consistently;

b) Observe the methods and principles in the Charities SORP;

c) Make judgements and estimates that are reasonable and prudent;

d) State whether applicable UK accounting standards have been followed, subject to any material departures disclosed and explained in the financial statements;

e) Prepare the financial statements on a going concern basis unless it is inappropriate to presume that the company will continue in operation.

The Directors’ are responsible for keeping adequate accounting records that are sufficient to show and explain the company’s transactions and disclose with reasonable accuracy at any time the financial position of the Company and enable them to ensure that the financial statements comply with the requirements of the Companies Act 2006. They are also responsible for safeguarding the assets of the Company and hence for taking reasonable steps for the prevention and detection of fraud and other irregularities.

The above report has been prepared in accordance with the special provisions of Part 15 of the Companies Act 2006 relating to small companies.

On behalf of the Directors

............................

Mr P Trill Director

Date: 31/07/2025

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CURE CLCN4 (a company limited by guarantee)

Combined Statement of Financial Activities and Income Statement for the period ended 31 December 2024.

The statement of financial activities includes all gains and losses in the year. All incoming resources and resources expended derive from continuing activities.

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CURE CLCN4 (a company limited by guarantee)

Balance sheet as at 31 December 2024.

For the financial period ended 31 December 2024 the company was entitled to exemption from audit under section 477 of the Companies Act 2006. The members have not required the company to obtain an audit of these financial statements pursuant to section 476 of the Companies Act 2006.

The directors acknowledge their responsibilities for ensuring that the company keeps accounting records which comply with Section 368 of the Act and for preparing financial statements which give a true and fair view of the state of affairs of the company as at the end of the financial period and of its surplus or deficit for the financial period in accordance with the requirements of Sections 394 and 395 and which otherwise comply with the requirements of the Companies Act 2006 relating to accounts, so far as applicable to the company.

These accounts have been prepared in accordance with the provisions applicable to small companies subject to the small companies’ regime and in accordance with FRS102 SORP.

Approved on and signed on behalf of all directors by:

Mr P Trill Director

Company Number: 12447230

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31/07/2025
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CURE CLCN4 (a company limited by guarantee)

Notes forming part of the accounts for the year ended 31 December 2024

1. BASIS OF PREPARATION

Cure CLCN4 is a company limited by guarantee incorporated in England and Wales.

The financial statements have been prepared in accordance with the Statement of Recommended Practice: Accounting and Reporting by Charities preparing their accounts in accordance with the Financial Reporting Standard applicable in the UK (FRS 102) (effective 1 January 2015), the Financial Reporting Standard applicable in the UK (FRS 102), the Companies Act 2006 and the Charities Act 2011.

The Charity constitutes a public benefit entity as defined by FRS 102.

The accounts have been prepared under the historical cost convention with items recognised at cost or transaction value unless otherwise stated in the relevant notes to these accounts.

2 . ACCOUNTING POLICIES

2.1 Incoming resources

All income is recognised once the charity has entitlement to the income, there is sufficient certainty of receipt and so it is probable that the income will be received, and the amount of income received can be measured reliably..

2.2 Offsetting

There has been no offsetting of assets and liabilities, or income and expenses, unless required or permitted by the FRS 102 SORP or FRS 102..

2.3 Grants and donations

Grants and donations are only included in the SoFA when the general income recognition criteria are met (5.10 to 5.12 FRS102 SORP).

2.4 Direct charitable resources expended

Direct charitable expenditure includes the direct costs in furtherance of the charity’s objectives and are recognised as expenditure as soon as there is a legal or constructive obligation committing the charity to that expenditure, it is probable that settlement will be required and the amount of obligation can be measured reliably. Expenditure is accounted for under the accruals concept.

2.5 Administration resources expended

Administration costs represent the running costs of the charity and are recognised as expenditure as soon as there is a legal or constructive obligation committing the charity to that expenditure, it is probable that settlement will be required and the amount of obligation can be measured reliably. Expenditure is accounted for under the accruals concept.

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CURE CLCN4 (a company limited by guarantee)

Notes forming part of the accounts for the period ended 31 December 2024 (continued)

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CURE CLCN4 (a company limited by guarantee)

Notes forming part of the accounts for the period ended 31 December 2024 (continued)

7. Funds of the Company

All funds held by the company are unrestricted and are to be used in the furtherance of the company’s objectives as outlined in the directors’ report on pages 8 and 9.

8. Related party transactions and Trustees’ Remuneration and expenses

Donations (Note 3) include a £50,347 contribution made by P Trill, a director of the company.

At 31 December 2024, £18,046 (2023 - £18,046) was owed to P Trill in relation to expense payments made on behalf of the company.

The trustees all give freely their time and expertise without any form of remuneration or other benefit in cash or kind. None of the trustees were paid expenses during the year.

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CURE CLCN4 (a company limited by guarantee)

Our Activities Scientific Research

Scientific research remains the central priority of Cure CLCN4. Our efforts are guided by the belief that meaningful progress for individuals affected by CLCN4 -related condition depends on advancing our understanding of the underlying biology and developing effective treatments. Throughout 2024, we remained committed to supporting high-quality research and fostering collaboration across the scientific community.

Cure CLCN4 Pilot Grant

We launched our first competitive funding initiative, the Cure CLCN4 Pilot Grant Programme, £150,000 aimed at supporting research projects with the potential to improve understanding and identify therapeutic strategies for CLCN4 . As part of this grant we are funding the following projects:

“Deciphering the consequences of CLCN4 loss-of-function & gain-of-function in rats to better understand CLCN4 -related neurodevelopmental condition” - Dr. Yann Herault & Dr. Tania Sorg (IGBMC, France).

“Understanding the functional consequences of novel CLCN4 variants using multifunctional analyses – from biophysics to subcellular trafficking” -

Dr. Michael Pusch (Istituto di Biofisica, Italy).

“Electrophysiological examination of neuronal electrical activity in rat Clcn4 KO and Clcn4 A549V hippocampal cells” -

Dr. Raul Guzman (FZ Julich, Germany).

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CURE CLCN4 (a company limited by guarantee)

Development of research tools

In 2024, we continued to invest in the development of key research tools to support the global scientific community studying CLCN4-related conditions.

Generation of a ClC-4 antibody

Cure CLCN4 is collaborating with Dr. Raul Guzman (FZ Juelich), Dr. Stefanie Bungert (FZ Juelich), Dr. Heinrich Flaswinkel (Ludwig Maximilian University of Munich), and Dr. Vera Kalscheuer (Max Planck Institute) to develop a ClC-4 antibody for use in basic research.

Induced pluripotent stem cells (iPSCs)

CLCN4 patient-derived induced pluripotent stem cells are being generated by the Simons Foundation, using samples from patients in the Simons Searchlight patient registry. In addition, Cure CLCN4 is also collaborating with Dr. Marlen Lauffer (Dutch Centre for RNA Therapeutics) to establish a biobank of CLCN4-patient derived iPSCs, which will also be made available to the scientific community.

Annual Scientific Conference

We hosted our third Cure CLCN4 Scientific Conference, bringing together researchers, clinicians, and families to foster collaboration and accelerate progress. The conference was held on 7th & 8th June 2024, in Central London (UK) and the program included 22 speakers and the event was attended by 75 participants, in person and online. The event also featured a poster session, which provided an opportunity for PhD students and early career researchers to participate.

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CURE CLCN4 (a company limited by guarantee)

Family support

Family-focused events

On 6th June we celebrated our second online Family Meeting, attended by over 40 families from over 9 countries. The meeting featured an Expert Panel Q&A, during which families had the chance to ask questions to a group of clinical specialists, including a clinical geneticist, a neurologist, psychiatrist, and gastroenterologist, among others.

Resources

We released the Cure CLCN4 Welcome Pack, a comprehensive booklet filled with essential resources for families navigating life with CLCN4 -related condition. It contains up-to-date information on the biology, diagnosis, and symptoms of CLCN4 as well as frequently asked questions, resources, and ways to get involved!

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CURE CLCN4 (a company limited by guarantee)

Fundraising

Luca and Marco Run the 2024 Ibiza Marathon!

After a previous fundraiser in 2023, where they raised over £15,000, Luca and Marco took on an even tougher challenge! Inspired by Luca’s youngest sister, Daphne, who lives with a CLCN4 -related condition, they decided to tackle the Ibiza 2024 Marathon. Despite the grueling training, they pushed through for this brilliant cause. Every donation went directly towards research to help advance our understanding and treatment of CLCN4. Thanks to their hard work and the incredible support from the community, they managed to make a big impact for Cure CLCN4!

Caroline’s Colour Run for Cure CLCN4!

In 2024, Caroline and her brother took part in the vibrant Colour Run held in France, channeling every colourful step into meaningful support for Cure CLCN4. Through their energy and enthusiasm, they rallied friends and family to sponsor their run, collectively raising vital funds for research and awareness.

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CURE CLCN4 (a company limited by guarantee)

Contact Us

[e-mail] www.cureclcn4.org

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Lewin Accounts Ltd Chartered Accountant Independent examinerfs report to the Trustees of Cure CLCN4 Ltd Registered Charity England and Wales. No. 1190344. Company number 12447230 I report to the charitytrustees on my examination of the accounts of the Company forthe year ended 31 December 2024. Responsibilities and basis of report As the charity trustees of the Company (who are also the directors of the company forthe purposes of company lawl, you are responsible for the preparation of the accounts in accordance with the requirements of the Companies Act 20061.the 2006 Act"). Having satisfied myself that the accounts of the Company are not required to be audited for this year under Part 16 of the 2006 Act and are eligible for independent examination. I report in respect of my examination of your charitvs accounts as carried out under section 145 of the Charities Act 20111"the 2011 Act"). In carrying out my examination, I have followed the Directions given by the Charity Commission (under section 14515)Ibl of the 2011 Act. Independent examinerfs statement I have completed my examination. I confirm that no material matters have come to my attention which gives me cause to believe that: accounting records were not kept in accordance with section 386 of the Companies Act 2006: or the accounts do not accord with such records; or the accounts do not comply with relevant accounting requirements under section 396 of the Companies Act 2006 other than any requirement that the accounts give a "true and fair" view which is not a matter considered as part of the independent examination- or the account5 have not been prepared in accordance with the Charities SORP IFRS 1021 I have no concern5 and have come across no other matters in connection with the examination to which attention should be drawn in this report in order to enable a proper understanding of the accounts to be reached. Colin Lewin, FCA Mercury House 19-21 Chapel Street Marlow Bucks. SL7 3HN 27 June 2025 Phone: [phone] Email.. [e-mail] Registered office: Mercury House 19-21 Chapel St Marlow Bucks SL7 3HN Registered in England No 7197950