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2024-03-31-accounts

Trustees’ Annual Report for the period

From 31/03/2023 To 31/03/2024

Charity name: Cure DHDDS

Charity registration number: 1202643

Objectives and Activities

SORP reference
Summary of the purposes of
the charity as set out in its
governing document		 Para 1.17 The objects of the CIO are to relieve the needs
of those affected by DHDDS Disease to support
families affected by DHDDS mutations and
drive research into them
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		 Families Support/Information Services
The charity provides a support group for
individuals and families affected by the
condition. We aim to provide the latest
information on the condition to families and
health professionals via our website, e-news
and social media.
Educational Activities
We continue to work on raising both public
awareness and education amongst key
medical professionals throughout the world
so they can relate to the signs, symptoms
and medical diagnosis of a DHDDS gene
mutation.
Speaking at and/or hosting exhibition
stands (in-person or virtually) at medical
and scientific conferences provides
opportunities to fulfil our aims and
objectives whilst developing strategic
partnerships with research facilities,
medical and pharmaceutical institutions,
and like- minded charitable bodies.
Research
The charity has raised funds to pay for
research to be undertaken by Perlara, Sainte
Justine Hospital, UCL and new research with
Cardiff and Aston University’s is agreed with
the intention of funding the research in
2024/25..
Statement confirming
whether the trustees have		 Para 1.18 The Trustees consider the charity’s activities

had regard to the guidance issued by the Charity Commission on public benefit

are wholly in the public interest and designed to benefit all those who suffer from DHDDS, their families and medical professionals dealing with this debilitating condition, in line with the Charity’s Commission guidance on ‘public benefit’.

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 In the first year since the charity was formed
several aims were achieved:
Funding/Fundraising
We have been overwhelmed by the
generosity that we have received from
donors in the first year.
Notable member fundraising events
included the 50km walk from Windsor to
Mortlake, Will Gore’s Royal Park run, London
Scottish bucket shake, Zoe Dyer’s lunch in
Marbella, Susie Braam’s coffee morning plus
several independent fundraisers across the
globe.
Research
Perlara, San Francisco – We funded a drug
repurposing screen undertaken by Perlara. The
Z prime experiment was successfully
completed, and we have drug hits due to be
tested in brain organoids at Mount Sinai
Hospital.
Sainte Justine Hospital, Montreal – We funded
Mouse model research with Alexey Pchejetski.
University College London (UCL) – We funded
preliminary research into an RNA therapy and
subsequently Dr Raj Karda and Dr Ozge Cetin
have applied for a grant to undertake AI-Based
Preclinical Studies UtilizingAAV9-Mediated

DHDDS-Specific Gene Therapy for Ultra-Rare DHHDS Disease. Cardiff University - Prof. Emyr Lloyd Evans and Dr Hannah Best – we have agreed to fund a 19 month research program focused on small molecule testing and NPC2 protein work. At a cost of £55,000 Conferences/Events attended + speaker opportunities: We have hosted two incredibly successful Cure DHDDS conferences in conjunction with Costello Medical. The first of which has been nominated for The Communique Awards 2024. There were 30 attendees at the first Conference in September 2023 and then 18 at the second smaller roundtable meeting held in April 2024. Mel has presented at a number of events to raise the awareness of Cure DHDDS and the research we are undertaking, including: Beacon CAM Rare Festival of Genomics Orphan Drugs Congress 2024 Europe UPNAT symposium Genomics England Research Summit Cure DHDDS has set up a patient registry with Cords at Sanford We have also worked closely with Cure DHDDS USA on current and future research projects in the US including the development of Brain Organoids at Mount Sinai Hospital and novel IPSC cell line experiments at The Ready Centre UCSF. Cure DHDDS benefits from services provided by Beacon for rare diseases, CamRARE including mentoring, training, and networking opportunities.

Financial Review

Review of the
charity’s
financial
position at the
end of the
period		 Para
1.21		 The charity is in a very fortunate position at the end of the first
financial year as a result of the generosity of many individuals and
companies.
To the year end (31stMarch 2023) the charity had receipts of
£236,221.
We have made three grants thus far as detailed under research, with
significant future commitments to Cardiff, Dr Felix Chan and Sainte
Justine as detailed below:
-
5,000
10,000
15,000
20,000
25,000
30,000
35,000
Grants and Costs 23/24
£0.00
£10,000.00
£20,000.00
£30,000.00
£40,000.00
£50,000.00
£60,000.00
Sainte Justine
Cardiff University
Felix Chan's Studies
2024/25 Research Grants
Statement
explaining the
policy for
holding
reserves
stating why
they are held		 Para
1.22		 The purpose of the reserves is:
 To fund working capital and time lags in the receipt of
promised funds
 To fund unexpected expenditure, e.g., when projects
overrun, or unplanned events occur
 Manage economic or regulatory changes which may
have unforeseen financial consequences
We assess the level of general reserve needed by looking forward
and considering the risks to our funding balanced against our
expenditure commitments. To ensure we can continue to operate
in accordance with our plans, we would look to hold a general
reserve in the range £10,000 - £12,000 to cover unfunded
committed costs for the next 6 months.
Amount of
reserves held		 Para
1.22		 Our reserves as of 31 March 2024 were £159,738
Reasons for
holding zero
reserves		 Para
1.22
Details of fund
materially in
deficit		 Para
1.24
Explanation of
any
uncertainties
about the
charity
continuing as a
going concern		 Para
1.23

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

----- Start of picture text -----
Receipts through 2023/24
The charity’s
principal sources Para 1.47
of funds (including £200,000.00
any fundraising) £150,000.00
£100,000.00
£50,000.00
£0.00
Corporate Individual Bank Interest
Donations Donations
----- End of picture text -----

Structure, Governance and Management

Description of charity’s
trusts:
Type of governing document
(trust deed, royal charter)
Para 1.25		 CIO association constitution
How is the charity
constituted?
(e.g unincorporated
association, CIO)		 Para 1.25 CIO association constitution (formed 06 April
2023)
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 By appointment of registered members at
general meetings and by Trustees

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 New trustees are asked to abide by a
Code of Conduct and are issued with
Essential Information for Trustees from
the Charities Commission, sign a Trustee
Declaration and complete a Fit and
Proper persons form.
The charity’s organisational
structure and any wider
network with which the
charity works		 Para 1.51 Cure DHDDS is run on a voluntary basis.
Trustees consult on all significant matters
of policy formulation, family support and
methods of communication about Cure
DHDDS information services to medical
professionals.
The Scientific Advisory Board remains in
place with 10 leading scientific advisors
helping us understand the research and
determine the most relevant use of
funding.
The charity operates the following
policies: child protection, data privacy,
risk management and volunteer
management, published on our website.
Relationship with any
related parties		 Para 1.51 The charity is a member of:
Cure DHDDS and NUS 1
Community
Genetic Alliance UK (GAUK)
Rare X
UK Rare Epilepsies Together
network (UKRET)
CDG Global Alliance
CDG UK
• The Charity works closely with Cure
DHDDS USA

Other

Reference and Administrative details

Charity name Cure DHDDS
Other name the charity uses
Registered charitynumber 1202643
Charity’s principal address 6 Park Avenue
London
SW14 8AT

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(if any)
Melanie Dixon
Charles Dixon
Victoria Allan
PennyBrogan
Louise Townley
Katie McCombe

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

Director name

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 held in this capacity

Name and objects of the charity on whose behalf the assets are held and how this falls within the custodian charity’s objects Details of arrangements for 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

The Cure DHDDS Trustees would like to thank all their donors and volunteers for their support throughout the year.

Special mention should go to our DHDDS fundraising heroes during the period:

Freddie Tidbury

Sophie Clegg

Will Gore

Zoe Dyer

Susie Braam

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) Position (eg Secretary, Chair, etc) Date

Charity Name

Cure DHDDS
 Charity Name

Cure DHDDS
 Charity Name

Cure DHDDS
 No (if any)
1202643		 CC16a
For the period
from		 Period start date
4/1/2023		 To Period end date
3/31/2024
Section A Receipts and payments
A1 Receipts
Donations & Fundraisingactivties
235,972
Bank Interest
249
-
-
-
-
-
-
236,221
-
-
Sub total -
Total receipts 236,221
A3 Payments
Bank fees
50
Sainte Justine
28,620
PERLARA
30,570
FX Charges
175
UCL
16,000
Office,Subscriptions and Events
1,568
-
-
Sub total 76,983
-
-
Sub total -
Total payments 76,983
Net of receipts/(payments) 159,238
A5 Transfers between funds
-
A6 Cash funds last year end
-
Cash funds this year end 159,238
Unrestricted
funds
to the nearest £
Sub total(Gross income for AR)
A2 Asset and investment sales,
(see table).
A4 Asset and investment
purchases, (see table)		 to the nearest £
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
Restricted
funds		 to the nearest £
Endowment
funds		 Total funds
to the nearest £
235,972
249
-
-
-
-
-
-
236,221
-
-
-
236,221
50
28,620
30,570
175
16,000
1,568
-
-
-
76,983
-
-
-
76,983
159,238
-
-
159,238		 Last year
to the nearest £
- 235,972 -
- 249 -
- - -
- - -
- - -
- - -
- - -
- - -
- 236,221 -
- -
- - -
- - -
- 236,221 -
- 50 -
- 28,620 -
- 30,570 -
- 175 -
- 16,000 -
- 1,568 -
- - -
- - -
- - -
- 76,983 -
- -
- -
- - -
- 76,983 -
- - 159,238 -
- - - -
- - - -
- - 159,238 -
Section B Statement of assets and liabilities at the end of the period Section B Statement of assets and liabilities at the end of the period Section B Statement of assets and liabilities at the end of the period Section B Statement of assets and liabilities at the end of the period
Categories
B1 Cash funds
B2 Other monetary assets
B3 Investment assets
B5 Liabilities
B4 Assets retained for the
charity’s own use		 Details
CAF account
Details
Details
Details
Details
Total cash funds
(agree balances with receipts and payments
account(s))		 to nearest £
159,738
-
-
159,738
Agreement Error
to nearest £
-
-
-
-
-
-
Unrestricted
funds
Unrestricted
funds
Fund to which
asset belongs
Fund to which
asset belongs
Fund to which
liability relates		 to nearest £
-
-
-
-
OK
to nearest £
-
-
-
-
-
-
Cost (optional)
-
-
-
-
-
Cost (optional)
-
-
-
-
-
-
-
-
-
-
-
-
-
-
Restricted
funds
Restricted
funds
Amount due
(optional)		 to nearest £
Endowment
funds
-
-
-
-
OK
to nearest £
Endowment
funds
-
-
-
-
-
-
Current value
(optional)
-
-
-
-
-
Current value
(optional)
-
-
-
-
-
-
-
-
-
When due
(optional)
-
-
-
-
-

Signed by one or two trustees on behalf of all the trustees

Signature

Print Name

Date of approval

Independent examiner's report on the accounts

Section A Independent Examiner’s Report Report to the trustees Charity Name CURE DHDDS On accounts for the year Charity no 31 March 2024 ended (if any) 1202643 Set out on pages (remember to include the page numbers of additional sheets) I report to the trustees on my examination of the accounts of the above charity (“the Trust”) for the year ended DD / MM / YYYY 31 03 2024 . Responsibilities and As the charity's trustees, you are responsible for the preparation of the basis of report accounts in accordance with the requirements of the Charities Act 2011 (“the Act”).

I report in respect of my examination of the Trust’s accounts carried out under section 145 of the 2011 Act and in carrying out my examination, I have followed all the applicable Directions given by the Charity Commission under section 145(5)(b) of the Act.

Independent [The charity’s gross income exceeded £250,000 and I am qualified to examiner's statement undertake the examination by being a qualified member of [insert name of applicable listed body]]. Delete [ ] if not applicable.

I have completed my examination. I confirm that no material matters have come to my attention in connection with the examination (other than that disclosed below *) which gives me cause to believe that in, any material respect:

I have no concerns 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.

Signed:
Name:
Relevant professional
qualification(s) or body		 Date:
23 August 2024
Georgia Cragg
ICAEW

Oct 2018

1

IER

(if any):

Address: Loughrigg, Sunnyside Close, Lancaster, LA1 5NH

Section B Disclosure

Only complete if the examiner needs to highlight material matters of concern (see CC32, Independent examination of charity accounts: directions and guidance for examiners).

Give here brief details of any items that the examiner wishes to disclose .

Oct 2018

2

IER