Ciliopathy Alliance

What Ciliopathy Alliance does

The Ciliopathy Alliance is a UK-registered charity (Charity No. 1148034) and company limited by guarantee (Company No. 7842342) founded in March 2010 and headquartered at the UCL Institute of Child Health, London. It functions as the umbrella patient-advocacy organisation for ciliopathies — a group of over 20 identified rare genetic conditions caused by defective cilia and estimated to affect roughly 1 in 1,000 people. The Alliance convenes more than 30 ciliopathy patient support groups (founder members including Alström Syndrome UK, Bardet-Biedl Syndrome UK, PKD Charity UK, and PCD Support UK), researchers, clinicians, and allied health professionals, providing a unified voice and an interface between patient communities and the research community. Its principal activities are awareness-raising, policy advocacy (including participation in the UK Rare Diseases Framework delivery and Rare Disease Day events at the House of Commons), coordination of research participation, and convening of scientific meetings.

The Alliance's "products" are information, convening, and advocacy services rather than software. Its main surface is the ciliopathyalliance.org website (hosted by Softforge), which serves as an information portal listing ciliopathies, patient groups, research labs, news and events, and a PubMed research feed. Complementary channels include a MailChimp e-newsletter, Eventbrite-managed event registration, Zoom-based webinars (launched November 2023), and the biennial Cilia Conference series (Cilia2022 in Cologne attracted 600+ delegates from 35 countries; Cilia2024 was held in Dublin; Cilia2027 is in planning). The Alliance is the patient partner in major research consortia including TheRaCil (Horizon Europe, €7.4M of EC funding plus €540,520 of UKRI co-funding across 16 partners in 6 countries), SCilS (EU Marie Skłodowska-Curie ITN), PREDICT, CiliaREN, and BeatPCD, and is integrated into European Reference Networks ERN-LUNG and ERKNet. A Scientific Advisory Board of international researchers provides research and medical guidance.

The Alliance's revenue model is donation- and membership-driven, with no commercial product sales. Income sources comprise individual donations collected via PayPal (with associated Gift Aid declarations retained for HMRC), and a tiered membership structure (Full, Affiliate and Associate Organisation members, plus Associate Individual members, with acceptance decided by the Board). Operations are run by a Board of Trustees (currently 8 named trustees including an Interim Chair) with a stated headcount of 1-10, supplemented by structured Keele University internships and a broader volunteer base; financial controls are handled at Board level, and a Treasurer is actively being recruited as of May 2026.

Name

Ciliopathy Alliance

Legal name

Ciliopathy Alliance

Website

https://ciliopathyalliance.org

Company type

Private

Founded year

2010

Operating status

Operating

Headcount range

1–10 employees

Short description

The Ciliopathy Alliance is a UK-registered charity and umbrella patient-advocacy organisation that convenes more than 30 ciliopathy patient support groups, researchers, clinicians, and allied health professionals to improve outcomes for patients and families affected by rare ciliopathies across the UK and Europe.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy

NAICS

Individual and Family Services (6241), Social Advocacy Organizations (8133)

SIC

Services-Social Services (8300), Services-Membership Organizations (8600)

akta.pro industry

PRIMARYBPAGACAA

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS)

Business & Public Services/Nonprofit, NGO & Social Services/Health & Medical Nonprofits

SECONDARYEDAIABAC

Research Collaboration & Joint Labs (International Partnerships)

Education/International Education & Study Abroad/University/College Exchange Programs & Partnerships

Keywords

• Rare disease advocacy
• Patient support networks
• Medical research coordination
• Genetic disorder awareness
• Healthcare charity services

Headquarters

HQ city

London

HQ country

United Kingdom

HQ region

Europe

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Operations, Technology or R&D, Marketing or Sales, Others

Revenue model

1. Donations: The organization accepts donations through PayPal. The website features a prominent 'Donate' button linking to their PayPal fundraising account.
2. Gift Aid Declarations: The charity maintains Gift Aid declarations for HMRC to claim tax relief on eligible donations. Records are retained until the end of the tax year following 7 years from the last donation.
3. Membership fees: The organization has a membership structure including Full (Organization), Affiliate (Organization), Associate (Organization), and Associate (Individual) categories. Board decides on acceptance of members.

Go-to-market motion2 records

Distribution channels4 records

Marketing channels5 records

Core offering

The Ciliopathy Alliance is a UK-based patient advocacy charity (Reg Charity No 1148034) that serves as an umbrella organization connecting over 30 ciliopathy patient support groups, researchers, doctors, and allied health professionals. It coordinates patient communities across 20+ identified rare genetic ciliopathies affecting an estimated 1 in 1,000 people, facilitates research collaboration through networks such as TheRaCil and CiliaREN, organizes biennial international Cilia Conferences, runs educational webinars, and advocates for ciliopathy patients at UK policy level.

Product overview

Ciliopathy Alliance is a UK-based patient advocacy charity (registered charity no. 1148034) that serves as a coalition connecting patient support groups, researchers, doctors, and allied health professionals for individuals affected by ciliopathies—rare genetic disorders caused by dysfunctional cilia. The organization does not offer a traditional software product but provides a suite of services including a comprehensive information website and portal, coordination of patient group networks across 30+ rare conditions (such as Bardet-Biedl Syndrome, Alstrom Syndrome, Polycystic Kidney Disease, Primary Ciliary Dyskinesia, Retinitis Pigmentosa, and Usher Syndrome), research network facilitation through organizations like TheRaCil and CiliaREN, educational webinars, the biennial Cilia conference series, advocacy engagement with UK policy makers, a PubMed research literature feed, and newsletter communications via MailChimp. The organization functions primarily as a convening and information-sharing platform rather than a technology product company.

Differentiator

Problem solved

Functional benefit

Products and services

• Ciliopathy Alliance Information Portal and Website Main public-facing digital platform providing information about ciliopathies, patient support groups, research updates, news, events and educational resources for patients, families, clinicians and researchers worldwide.
• Ciliopathy Alliance Newsletter (eNews) Email newsletter service delivered via MailChimp to keep subscribers informed about ciliopathy research, events, policy updates and community news.
• Educational Webinars Online educational webinars delivered via Zoom covering ciliopathy research topics such as sight loss research, skeletal ciliopathies, and cilia biology, often featuring expert clinicians and researchers.
• Biennial Cilia Conference Series Major international scientific conferences (Cilia2022, Cilia2024, Cilia2027) bringing together researchers, clinicians, patients and industry partners to advance cilia and ciliopathy research; the Cologne 2022 edition was attended by 600+ delegates from 35 countries.
• Research Network Coordination (TheRaCil, SCilS, PREDICT, CiliaREN, BeatPCD) Facilitation of collaboration and information sharing across multiple ciliopathy research networks and European Reference Networks including Cilia Network, TheRaCil, BeatPCD, PREDICT, CiliaREN, ERKNet and ERN-LUNG.
• Patient Group Network Coordination Coordination and umbrella support of patient organisations for specific ciliopathies including Alström Syndrome UK, Bardet-Biedl Syndrome UK, PKD Charity UK, PCD Support UK, Retina UK, Cure Usher and Heterotaxy Foundation, providing a unified voice for the ciliopathy community.
• Scientific Advisory Board Service Research and medical advisory service provided to the Ciliopathy Alliance Board through an international panel of researchers and doctors involved in ciliopathy research and patient treatment.
• Advocacy and Policy Engagement Service Representation of the ciliopathy patient community in UK and European policy discussions, including participation in Rare Disease Day events at the House of Commons and consultations on the UK Rare Diseases Framework.
• PubMed Research Literature Feed Aggregated feed of the latest scientific publications on ciliopathies from PubMed, providing researchers and families with access to current literature in the field.
• Ciliopathy Alliance Membership Programme Formal membership programme with Full (Organization), Affiliate (Organization), Associate (Organization) and Associate (Individual) categories, providing formal affiliation with the Alliance for patient groups, research institutions and individuals.

Quantifiable outcome

• Supported establishment of NHS funding for diagnosis and management services for two patient groups
• +2 more outcomes

Named customers2 records

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Integration5 records

Partnerships

22 partnerships are on record, tiered core, major and supporting.

• TheRaCil (Therapies for Renal Ciliopathies)coreStrategic or Co-development PartnerA 4-year Horizon Europe research programme with European Commission funding of €7,425,446 and UKRI co-funding of €540,520. The Ciliopathy Alliance is the patient partner in this consortium of 16 partners (15 institutions in 6 countries) working to develop treatments for paediatric renal ciliopathies including nephronophthisis and ARPKD.
• CILIARENcoreStrategic or Co-development PartnerA collaborative network of clinical teams, research centres and patient groups around the UK working to improve the care and diagnosis of people with kidney ciliopathies. The Alliance contributes patient perspectives through Patient Advisory Groups.
• SCilS (Studying Ciliary Signalling in Development and Disease)coreStrategic or Co-development PartnerEU-funded Marie Skłodowska-Curie Innovative Training Network (ITN) training a pan-European cohort of students to become the next generation of independent cilia researchers. The Ciliopathy Alliance represents patient interests in this consortium.
• Genetic Alliance UKcoreStrategic or Co-development PartnerPartnership for policy advocacy and rare disease framework consultations. The Ciliopathy Alliance participates in Future for Rare campaign and UK Rare Diseases Framework activities organized by Genetic Alliance UK.
• UK Rare Disease Research PlatformcoreStrategic or Co-development PartnerA £14 million investment over 5 years for rare diseases. The Ciliopathy Alliance is involved with the UK Renal Ciliopathies National Network as part of this larger platform.
• PREDICT (PREcise DIagnosis in Ciliopathies)majorStrategic or Co-development PartnerEuropean Joint Programme on Rare Diseases project aimed at improving and accelerating diagnosis of ciliopathies. Part of EJP RD initiative.
• CiliaRENmajorStrategic or Co-development PartnerNetwork improving identification, diagnosis and management of renal ciliopathies in the UK.
• Cilia Network (UK Cilia and Centrosome Network)majorStrategic or Co-development PartnerNetwork of researchers and clinicians working on structure and function of motile and primary cilia and centrosomes in the UK. The Alliance provides support to run meetings and facilitates collaboration.
• BeatPCDmajorStrategic or Co-development PartnerERS-sponsored Clinical Research Consortium working to improve diagnosis and clinical care, and expand research for PCD patients. Includes patient advocacy representatives.
• ERN-LUNG (European Reference Network)majorStrategic or Co-development PartnerEuropean Reference Network for Rare Lung Diseases. The Ciliopathy Alliance participates through connections with PCD support activities.
• ERKNet (European Reference Network for Rare Kidney Diseases)majorStrategic or Co-development PartnerEuropean Reference Network for Rare Kidney Disease. The Alliance is a patient representative in this network.
• Rare Disease UKmajorStrategic or Co-development PartnerCampaign organization adding patient voice for genetic conditions to diagnosis, medical research, healthcare delivery, access to treatments. The Alliance participates in Rare Disease Day events at House of Commons.
• EURORDISmajorStrategic or Co-development PartnerEuropean umbrella organization empowering, partnering and advocating for people living with a rare disease in Europe.
• Heterotaxy FoundationcoreChannel Partner/ Reseller/ DistributorPatient organization that joined the Ciliopathy Alliance. Heterotaxy is a congenital condition caused by rotation of cilia in the developing embryo. The foundation brings expertise in this specific ciliopathy.
• Alström Syndrome UKcoreChannel Partner/ Reseller/ DistributorFounder member of the Ciliopathy Alliance. UK patient organization for Alström Syndrome.
• Bardet-Biedl Syndrome UK (BBS UK)coreChannel Partner/ Reseller/ DistributorFounder member of the Ciliopathy Alliance. UK patient organization for Bardet-Biedl Syndrome. BBS Global Day is coordinated internationally with BBS Family organisations in France, Italy, Netherlands, and North America.
• Polycystic Kidney Disease Charity UKcoreChannel Partner/ Reseller/ DistributorFounder member of the Ciliopathy Alliance. UK charity for PKD. Tess Harris served as CEO and represented PKD Charity.
• PCD Support Group UKcoreChannel Partner/ Reseller/ DistributorFounder member of the Ciliopathy Alliance. UK support group for Primary Ciliary Dyskinesia. Fiona Copeland previously served as chair.
• Retina UKmajorChannel Partner/ Reseller/ DistributorUK charity supporting people with inherited sight loss including retinitis pigmentosa, a ciliopathy. Collaborates on webinars about sight loss research in ciliopathies.
• Cure UshermajorChannel Partner/ Reseller/ DistributorOrganization focused on Usher syndrome. Participates in Ciliopathy Alliance events and represents patient perspective on sight loss research.
• Keele UniversitysupportingOthersUniversity that provides intern placements for the Ciliopathy Alliance. Students complete 75 hours of work over four months as part of their course requirements.
• Institute of Child Health (UCL)coreOthersLocated at the Institute of Child Health. Multiple trustees are affiliated with UCL including Prof Hannah Mitchison, Dr Roly Megaw, and Prof Phil Beales. Hosts PCD research labs visited by interns.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• National Organization for Rare Disorders (NORD): US-based umbrella for rare disease patient organizations with analogous advocacy, research facilitation, and policy functions. Most comparable US counterpart to the Ciliopathy Alliance model.
• Rare Disease UK: UK campaign alliance for rare diseases coordinated by Genetic Alliance UK. Direct collaborator with Ciliopathy Alliance on House of Commons Rare Disease Day events and UK Rare Diseases Framework activities.
• Genetic Alliance UK: UK umbrella organization for genetic and rare disease patient groups. Direct partner of the Ciliopathy Alliance and convenes it within the broader rare disease policy framework in the UK.
• Orphanet: European reference portal for rare diseases and orphan drugs. Comparable in providing information infrastructure and research coordination for rare disease communities, including ciliopathies.
• EURORDIS - Rare Diseases Europe: European umbrella for rare disease patient organizations. Listed partner of the Ciliopathy Alliance and operates at a comparable pan-European advocacy and convening scale.

Broad incumbents

• Kidney Research UK: UK-wide kidney disease charity encompassing renal ciliopathies such as PKD and nephronophthisis. Comparable as a broader disease-area umbrella intersecting with the Ciliopathy Alliance's renal ciliopathy work (CiliaREN, TheRaCil).
• British Lung Foundation (Asthma + Lung UK): UK respiratory charity overlapping with PCD (Primary Ciliary Dyskinesia) and BeatPCD Consortium activities. Comparable as a larger umbrella encompassing one of the Alliance's key ciliopathies.

Emerging players

• Retina UK: UK charity for inherited sight loss including retinitis pigmentosa (a ciliopathy). Direct partner of the Ciliopathy Alliance on webinars and sight loss research, comparable as a disease-specific patient advocacy group within the Alliance's network.
• PKD Charity UK: UK patient organization for Polycystic Kidney Disease and founder member of the Ciliopathy Alliance. Comparable as a single-disease ciliopathy patient group within the Alliance's umbrella.
• Bardet-Biedl Syndrome UK: UK patient organization for Bardet-Biedl Syndrome and founder member of the Ciliopathy Alliance. Comparable as a single-disease ciliopathy advocacy group under the Alliance umbrella.

Market position

Strengths1 record

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• YouTubeyoutube.com/channel/UCqL3QJz1WJq6nJ6tOlQuT1A
• LinkedInlinkedin.com/company/ciliopathy-alliance

Compliance3 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Ciliopathy Alliance