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PCD Research Contact information, map and directions, contact form, opening hours, services, ratings, photos, videos and announcements from PCD Research, Charitable organisation, The House of Commons, London.

Delighted to have been asked to contribute to this report https://researchbriefings.files.parliament.uk/documents/POST-P...
26/03/2026

Delighted to have been asked to contribute to this reporthttps://researchbriefings.files.parliament.uk/documents/POST-PN-0763/POST-PN-0763.pdf

It’s rare disease day. We are working to make change happen so that it’s will be possible to make treatments that functi...
28/02/2026

It’s rare disease day. We are working to make change happen so that it’s will be possible to make treatments that functionally restore genetic diseases at scale. Behind the scenes, things are moving and we can’t wait to share this news with you when we can.

24/11/2025

Sharing a video about The Rare Disease Summit 2025 that we hosted last month to set out how to solve the challenges of rare disease. Share and like to help us raise awareness

➡️ Transforming Lives Through Action
On 14 October together with Alexion we held a Rare Disease Summit, sponsored by GSK...
15/10/2025

➡️ Transforming Lives Through Action
On 14 October together with Alexion we held a Rare Disease Summit, sponsored by GSK, Eli Lily, Weatherden, LifeArc and Alexion. Here we brought together scientists, clinicians, industry leaders, and policymakers for one purpose, to change the future for people living with rare diseases.

From stories of years-long diagnostic journeys to breakthroughs in genetic research and new therapies, every voice reminded us: patients are the reason we do this.
The Rare Disease Summit was about actioning scientific breakthroughs - driving towards a shorter the path to genetic answers, unlocking access to genetic treatments, and building a future where nobody is left behind.

Founder and Exec Chair, Dr Harriet Holme in conversation with Lord David Cameron, Former Prime Minister and Chair, Advisory Council of the Oxfor-Harrington Rare Disease Centre.

Together, we’re transforming diagnosis and treatment.

🔗 Learn more: pcdresearch.org/transforming-diagnosis-and-treatment-rare-disease-summit/


Great to be back at the Houses of Parliament again for hosted by and the new chair of the APPG, Peter Dowd MP. A hig...
28/02/2025

Great to be back at the Houses of Parliament again for hosted by and the new chair of the APPG, Peter Dowd MP. A highlight of the day was speaking with Parliamentary Under-Secretary of State Ashley Dalton MP.

This year, as the sun shone over the Thames, there was a palpable sense of change—with a new government eager for and , the opportunity for a bolder, more ambitious vision for rare disease innovation and investment feels within reach.

2025 is a critical year for rare disease, as the Government’s Rare Disease Framework ends with no future commitment.

Yet the challenges facing people with rare disease are stark:
 
⏩ 4 million people in the UK live with a rare disease.
⏩ 95% of these patients lack approved treatments.
⏩ 30% of children with a rare disease die before the age of 5.
⏩ Families struggle for years to get a diagnosis, with the median age at 4.7 years.
 
The case for urgent, radical reform is strong. Despite major advances in genomics, science, and technology, rare disease patients too often remain overlooked because innovation in this space is not deemed commercially viable by the pharmaceutical industry.

Yet there is a solution that aligns with the government’s Plan for Change, building on the knowledge economy to unlock innovation, treatments for underserved patients while delivering capital growth and jobs. This requires minimal investment for a far-reaching return, which could position the UK as a world-leading innovator in rare diseases. The UK is uniquely positioned to lead, even more so with uncertainty in the USA.
 
We are at a pivotal moment where technological advances can lead to a paradigm shift for millions of people as a precursor for a new model of health.

Delighted to be able to announce on , PCD Research and NATA - Nucleic Acid Therapy Accelerator have agreed to jointly a...
28/02/2025

Delighted to be able to announce on , PCD Research and NATA - Nucleic Acid Therapy Accelerator have agreed to jointly award Prof Stephen Hart and colleagues at UCL, a £250k two-year research grant to develop mRNA therapy for primary ciliary dyskinesia (PCD). 
 
This grant targets the 3rd most common disease causing gene CCDC39, known for causing some of the most severe clinical outcomes. This project aims to restore the movement of cilia (tiny hair-like structures in the lungs) by delivering mRNA to replace the faulty CCDC39 protein. 
 
If successful, this method could be a significant advance in creating more effective, targeted treatments for PCD across many disease causing genes. 
 
This is a first not just for PCD Research, but also the first research towards developing a treatment to restore ciliary function that has been funded by a charity. Instrumental in this achievement were Gina Bicknell, Kiah York and their colleagues from the IP Transactions team at Pinsent Masons, who worked tirelessly to negotiate and finalise the contract with UCL, and without their help, this achievement would not have been possible. 
 
           

It’s and we are pleased to share a few posts and news.
28/02/2025

It’s and we are pleased to share a few posts and news.

At the start of PCD awareness month we hosted our Rare Disease Industry Accelerator day. The event looked to reframe the...
22/10/2024

At the start of PCD awareness month we hosted our Rare Disease Industry Accelerator day. The event looked to reframe the narrative of PCD as a mild disorder and to leverage interest and IP from cystic fibrosis to address the lack of much needed treatments.

The whole day was by the talented Jonny Glover from covering the important themes of the day: , , ,
, , , for rare disease, and the needs to change.

We are grateful for our sponsors who made this event possible: LifeArc, , Weatherden, Pinsent Masons, AXON, MRC National Mouse Genetics Network, Congenital Anomalies Cluster and the LifeArc Centre for Rare Respiratory Diseases.












Starting with PCD Research’s inaugural rare disease industry accelerator event - getting cilia moving. A huge thank yo...
03/10/2024

Starting with PCD Research’s inaugural rare disease industry accelerator event - getting cilia moving.

A huge thank you to the the faculty, sponsors and our tiny but mighty team of volunteers led by Florence Barkats who made this day possible.

I look forward to sharing some of the photos and insights from the day soon.

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