Reverse Rett Strategy 2021 - 2026 (Change Is Within Reach)

CHANGE IS WITHIN REACH

OUR STRATEGY 2021-2026

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ABOUT THE PLAN The 2021-2026 strategy begins a new

Reverse Rett has been at the forefront of

era in the development of Reverse

Rett Syndrome laboratory and clinical

Rett. It is a statement of how we are

research funding in the UK for more than

going to change over the next few

a decade. Now that change is in reach,

years, whilst remaining true to the

we need to do more. Our first principle is

original vision and mission we had

equality; that all patients with Rett

when the charity was founded.

Syndrome regardless of age or gender should have access to emerging

This strategy document begins with our

treatments and cures.

vision, which after 11 years of operations,

We will continue to seek out and register

remains the same. Our mission remains

as many UK patients as possible,

focused on the delivery of treatments and

wherever they are, so that we can reach

cures for Rett Syndrome but

them, when treatments which will

acknowledges that advocacy as well as

improve their lives become viable.

research will play a critical role in ensuring that emerging treatments are

We are proud of our past achievements

accessible to all who need them.

and positive about the future unfolding before us. We just might be the last

With new treatments for Rett on the

generation to live with Rett Syndrome as

horizon, those children and adults who

a disease which has no treatment. We

are already living with the symptoms of

will be bold in our expectations and

the disease to varying degrees, need

actions and collaborative moving

access to adequate clinical care. Specialist

forward, with the individual with Rett

centres with the expertise to treat the

Syndrome at the heart of everything we

complexities of this multi-system disorder

do.

are vital, as is the dissemination of knowledge, so that all patients with Rett Syndrome are able to benefit from what is learned through focused and innovative clinical practise.

RACHAEL STEVENSON CEO

JOHN H SHARPE CHAIR

2010

2021

2024

WHERE WE’VE COME FROM

WHAT WE HAVE NOW

WHERE WE'RE GOING

No approved Treatments

Reverse Rett has now delivered over £7M to

Enabling access to Gene Replacement

research.

Therapies for all identified eligible

No human clinical trials

patients We fund the CIPP Rett Centre, a UK clinical

Proven reversibility but no tangible way

research centre of excellence implementing

Ongoing identification of affected children

forward

pilot studies of treatments, gathering data to

and adults

support and inform the delivery of emerging No UK research focused organisation

treatments and managing the health of 125

Managing Rett Registry

complex UK patients with Rett Syndrome.

No UK Centre of excellence No active UK Registry or Database

Sharing/facilitating best practice leading We are custodians of the Rett Registry UK with

to strong medical management for all

over 400 registered patients.

patients before, after and those without Gene Replacement Therapies.

We have a tangible way to address the underlying cause of the disorder in human

Funding research focused on refining

trials (Gene Replacement Therapies).

treatments and clinical research and rehabilitation aimed at improving the

Three human clinical trials of GRTs are projected to file INDs (pre-cursor to human clinical trials) in late 2021.

lives of those with residual symptoms.

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NOW AND THEN Since Reverse Rett was founded in 2010, we’ve learned a lot. We’ve learned that the development of treatments and cures for a condition stretches all the way from the most basic laboratory research to the moment when a doctor can legitimately prescribe the medicine or treatment for a particular patient.

Overleaf, you’ll see Reverse Rett’s vision for the future. This has been our documented vision for seven years and it still stands today. We’re making good progress towards this goal. In this strategy document, we’re mapping out our priorities and the actions we need to take in order to keep moving forward as effectively and efficiently as possible.

To this end, we had to imagine the world, not as it is today but as we expect it to be in the coming years. We had to think about what our role will be once transformative treatments for Rett Syndrome begin to emerge and how we can ensure that these treatments are accessible to everyone who needs them.

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OUR VISION

OUR PRINCIPLES

We want a world where girls and boys with Rett

EQUALITY

Syndrome are diagnosed quicker, earlier and more effectively than they are right now. A world where much more is known about the condition and factors that affect progression. A world where at every stage of development, treatments are available to counteract its symptoms. A world where we ultimately deliver a cure and enable people with Rett Syndrome to lead free and healthy lives.

All patients with Rett Syndrome regardless of age or gender should have access to emerging treatments and cures.

COMPASSION Beyond treatments and a cure, all patients with Rett Syndrome are likely to require some level of holistic medical management, the extent of which will be based on whether they have been able to access and/or benefit from the treatments/cure or not and the age they were and condition they were in when this became available.

COLLABORATION

OUR MISSION

We believe in collaboration. Delivering treatments and a cure for Rett Syndrome is a big job and it needs all of us to bring about real change everywhere.

Reverse Rett is a patient advocacy and

DIVERSITY

research organisation focused on delivering

We value diversity. We want to ensure that no one is marginalised

treatments and a cure for Rett Syndrome to

as we move together towards our common goal.

everyone affected.

SCIENCE We are full members of the Association of Medical Research Charities and abide by all the rules therein associated.

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THE FOUR PILLARS OF OUR WORK AT REVERSE RETT The future for Reverse Rett is one where children and adults with Rett Syndrome are at the heart of everything we do in line with our mission to deliver treatments and a cure to everyone affected.

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RESEARCH

OUTREACH

To fund laboratory and clinical research projects that will lead to better outcomes for children and adults with Rett Syndrome both now and in the future.

Sending targeted Rett Registry UK campaigns out to GPs, paediatricians, neurologists, adult neurology departments, learning disability nurses, special schools, colleges, respite centres and care homes to locate unregistered patients.

ADVOCACY To advocate for access to treatments and therapies for all affected patients.

CLINICAL CARE To improve clinical care and outcomes for complex UK children and adults with Rett Syndrome whilst disseminating research and clinical best practice information to improve management of all those affected.

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RESEARCH We used to think that once a cure for Rett Syndrome was proven in clinical trials,the work of Reverse Rett would be done, we’d all be able to down tools and go home.

But it’s seldom the case that the first approved treatments for a condition become the standard treatment offered for a particular disease. Even if early attempts at transformative treatments are successful, gene therapies will need refining and improving.

We will do this by: Working in partnership with the US based RSRT to fund UK based Rett Syndrome research at Edinburgh University focused on improving and refining curative therapies.

Drugs to ameliorate particular symptoms also need to be developed and improved in aid of those with untreated or residual Rett symptoms.

Funding clinical research focused on establishing effective outcome measures for human trials, clinical care best practice and research updates to ensure strong medical management for all patients, whilst providing the necessary clinical infrastructure for transformative therapies and treatments to improve residual symptoms.

Our goal over the next five years is to champion new and emerging treatments for Rett Syndrome ensuring that lives can be lived to the fullest.

Continuing to promote MECP2 Duplication Funds and work with affected families to deliver funds to Cure MDS.

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ADVOCACY Emerging treatments and ultimately a cure for Rett Syndrome will be expensive.

In a country where we are dependent on a publicly funded health service to assess the broader value of emerging treatments, there is a way to go from successful clinical trials to the patient’s bedside.

After transformative treatments are shown to be effective, Reverse Rett will have a pivotal role in ensuring Gene Replacement Therapies (GRTs) and other treatments are funded by the NHS in England, Scotland, Northern Ireland and Wales.

As well as working to ensure that these emerging treatments are authorised for younger, less symptomatic patients, It will be incumbent upon Reverse Rett and the wider UK Rett community to advocate for older, symptomatic patients with Rett Syndrome to have the opportunity to access treatments and cures from the outset.

We will do this by:

Increasing the number of registered UK patients in order to demonstrate unmet need in terms of treatments and clinical care.

Continuing to fund the CIPP Rett Database which we’ve been seed funding since 2014. These online questionnaires are tailored to people with Rett Syndrome. Participation is free and open to UK patients of any age who have Rett Syndrome or a related condition.

Building a strong evidence base showing the typical progression of disease in patients with Rett, the economic impact and the economic benefit to the NHS of improvements in symptoms even in older patients who may not have the same optimal response to treatment as those who are pre-symptomatic.

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OUTREACH Change is coming for people with Rett Syndrome. As well as working to ensure that treatments for Rett become viable, we want to make sure that all UK patients have access to those treatments. Part of that is about the advocacy work we’ve already described. But we also need to know where each of those patients are.

Finding more patients will enable us to help more patients and families; providing them with accurate and up to date best practice research updates and resources through various platforms.

We’re prioritising reaching out to the general public to increase awareness of Rett Syndrome so that as many individuals as possible are correctly diagnosed, registered and contactable as treatments emerge and so that their health can be better managed until they can access them.

We will do this by: Re-developing the UK Rett Syndrome Registry to make it interactive, informational, user-directed and highly secure so that more people are motivated to join.

Running specific timed social media campaigns to grow/advertise the Rett Registry.

Running new and innovative public campaigns such as Stop the Creep and Free From Rett, to bring more general awareness of Rett Syndrome into the public psyche.

Sending targeted Rett Registry UK campaigns out to GPs, paediatricians, neurologists, adult neurology departments, learning disability nurses, special schools, colleges, respite centres and care homes to locate unregistered patients.

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Increasing UK CIPP Rett Database recruitment Ensuring that the Rett Disorders Health Checklist is kept up to date and accurate through regular annual revisions.

Running ongoing Rett Connect programs online to keep parents and carers connected, informed and empowered to manage the health of someone with Rett Syndrome including participation in clinical trials or research.

Publishing Reverse Rett Podcast episodes and short clips on YouTube to enable parents and carers to learn from and motivate each other.

Producing monthly digital mailouts with all the latest information and activities at Reverse Rett and learnings from the CIPP Rett Centre.

Distributing a quarterly hard copy/digital newsletter to share the latest research and health updates and wider activities of Reverse Rett in a varied format.

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CLINICAL CARE With a complex multi-system disorder like Rett Syndrome, an individualised and holistic approach to treatment is vital.

Having a UK centre of expertise which regularly manages and treats a large number of affected patients will help to deliver treatments more quickly and more effectively and bolsters the confidence of the funding bodies who want to see that the treatments they are funding are used to their maximum benefit.

Reverse Rett has provided three years of start-up funding for the creation of the Centre of Interventional Paediatric Psychopharmacology, (CIPP Rett Centre).

The centre has become the CIPP for clinical research and treatments in the UK. Through the CIPP Rett Centre, we are laying the foundation for human trials of transformative treatments to happen in the UK. We are learning as much as we can from the CIPP Rett Database and sharing that information widely, to improve the lives of children and adults with Rett Syndrome and related disorders everywhere

Critically, we are also ensuring that as many complex UK patients with Rett Syndrome as possible survive and have optimal quality of life until treatments and cures become available.

We will do this by: Disseminating information about the CIPP Rett Centre to help grow the CIPP Rett Database which in turn will help researchers learn more and share more best practice information to support the health of people with Rett Syndrome.

Raising the profile of the CIPP Rett Centre amongst UK clinicians, hospitals and CCGs so that as many patients as possible can access the service.

Facilitating connections between the CIPP Rett Centre and industry partners.

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MAKING IT HAPPEN BY INVESTING IN OUR FUTURE Our plans for the future are ambitious. To achieve all we set out to achieve, we must invest in our people.

We will do this by:

Ensuring that all members of the Reverse Rett team have the skills and resources they need Keep our systems updated and optimally effective Invest in resources to deliver this strategy and ensure long term growth Introduce annual disability awareness training for all staff Engage additional support to increase fundraising capacity, particularly through trusts and grants and corporate support.

Broaden our ability to generate sustainable income by diversifying our sources of funding.

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THROUGH ALL OUR WORK WE WILL UPHOLD OUR CORE VALUES PEOPLE FIRST People with Rett Syndrome will always be at the heart of all we do

SPEAK OUT We will always speak out on issues that affect our community

OPEN AND TRANSPARENT We will be clear in our communications and transparent about our work

TOGETHER, WE ARE MAKING CHANGE HAPPEN None of the work we are doing today would be possible without our amazing community. We need you now more than ever.

Please consider supporting us through a regular monthly donation, taking part in a challenge, getting involved in the annual gala or getting your company involved.

You can also help by spreading the word about Rett Syndrome and the treatment which is within reach, by joining the 365 club, or by adding a gift in your will.

CONTACT 0161 413 0585 info@reverserett.org.uk www.reverserett.org.uk Reverse Rett , Statham Link 1F, Lancastrian Office Centre, Talbot Road, Old Trafford, Manchester M32 0FP

Registered charity in England & Wales, charity number 1136809 and in Scotland, charity number SC046735. A company limited by guarantee number registered in England & Wales, company number 07278507