Healx, the AI-powered, patient-inspired technology company accelerating the discovery and development of rare disease treatments at scale, today announced that it would be collaborating with the PKD Charity to identify and progress novel treatments for Autosomal Dominant Polycystic Kidney Disease (ADPKD) and Autosomal Recessive Polycystic Kidney Disease (ARPKD).
The project will leverage Healx’s extensive expertise in tech-driven drug repurposing for rare diseases and combine it with the PKD Charity’s unparalleled patient and disease insight to progress novel therapies for the conditions towards the clinic.
ADPKD is the most commonly inherited kidney disorder in the world, affecting over 12 million people worldwide. The condition is dominantly inherited, meaning there is a 1 in 2 chance of passing it on to children, and in the UK alone, there are estimated to be around 60,000 people currently living with the condition. ADPKD is caused by a genetic fault that disrupts the normal development of some of the cells in the kidneys and other organs - such as the liver and pancreas - and is characterised by the formation of multiple fluid-filled cysts on these organs. In the case of the kidneys, the cysts grow and multiply over time, replacing normal healthy tissue to the point that the kidneys start to fail. As these cysts grow, kidneys can enlarge up to four times their regular size and can weigh up to 100 times more. Alongside kidney failure, this can cause a range of problems including large cystic livers (which can result in transplantation), chronic back and abdominal pain, urinary and cyst infections, high blood pressure, brain aneurysms and kidney stones.
ARPKD, the rarer form of PKD, also affects the kidneys and liver. It is most commonly diagnosed during pregnancy and in babies and young children. ARPKD occurs in roughly one in 20,000 live births and is caused by an alteration in the gene PKHD1, which is passed on to a child through recessive inheritance by both parents. Parents of children with ARPKD don’t have the disease themselves but are ‘carriers’ of the disease and critically, around one in every 70 people is an ARPKD carrier.
Both ADPKD and ARPKD are currently incurable, and treatments vary in their effectiveness. In the case of ARPKD, there are no proven treatments to slow the progression of the disease but there are treatments to address other symptoms of the condition. For ADPKD, there are treatments available that can both slow kidney function decline and reduce the symptoms, but some of these are associated with side effects that can affect patients’ willingness to take the drugs long term. Moreover, many patients are ineligible for the only licensed therapy available worldwide. By combining the PKD Charity’s patient and disease knowledge with Healx’s extensive drug discovery expertise and AI technology, this partnership aims to identify new therapies for the conditions that are not only more effective, but also more tolerable and accessible for patients.
This tech-driven repurposing approach is central to Healx’s work and means patients can get access to life-changing treatments more quickly. AI enables incomparable speed and lateral thinking on a scale that is impossible for humans to achieve alone and, in the case of drug discovery and development, can make the process quicker, cheaper and more efficient. In this project, Healx will be using Healnet - its AI-powered drug discovery platform - to interrogate drug-disease data and quickly predict the most effective drugs that could be repurposed into mono- and combination therapies for the condition. By focusing on known drugs, the team is able to further accelerate and de-risk the progression to clinical trials and reduce the likely side effects of treatments since the safety profile of the drug is already known.
Commenting on the partnership, Tess Harris, CEO at the PKD Charity, said: “We are delighted to be collaborating with Healx in this pioneering partnership which has the potential to accelerate the development of much-needed treatments for both forms of PKD. We know that the patients and families we support will hugely welcome this initiative. They are eager and willing to contribute their insights and experiences in the hope of new therapies that can slow down the relentless progression towards kidney failure in ADPKD and kidney and liver failure in ARPKD, as well as reduce the symptom burden and improve overall quality of life – not just for themselves but for future generations.”
Bruce Bloom, Chief Collaboration Officer at Healx, says: “We are incredibly proud to be partnering with the PKD Charity, the UK’s leading charity for polycystic kidney disease awareness and research, to find repurposed therapies for these life-altering conditions. Putting patients at the heart of the drug discovery and development process is central to all of our work at Healx, and we are looking forward to working with the PKD Charity to integrate their expertise and insight right from the start.”
The partnership with the PKD Charity marks the latest in a growing number of collaborations between Healx and patient groups across the globe, and the team is eager to extend its expertise in the area by collaborating with other groups working on polycystic kidney diseases. The partnership also extends the number of disease areas within Healx’s portfolio. Earlier this year, Healx announced that it would be working with Mission: Cure to identify the world’s first treatments for chronic pancreatitis, and in 2020 the team launched partnerships with the Foundation of Angelman Syndrome Therapeutics, Muscular Dystrophy UK and The Children’s Tumor Foundation.
Patient, industry and academic groups interested in collaborating with Healx to unlock the power of repurposed drugs for rare diseases are encouraged to apply to Healx’s Rare Treatment Accelerator (RTA). The RTA is Healx’s partnering programme for groups and organisations with promising drug repurposing opportunities for rare diseases who are looking for financial or clinical support to take the drugs to trial. Working together with selected applicants, Healx will leverage its AI, drug discovery and clinical expertise to accelerate novel therapies towards clinical trials in a typical timeframe of 6-12 months. To learn more about the RTA, please go to https//healx.io/rta.
Healx is an AI-powered, patient-inspired technology company, accelerating the discovery and development of rare disease treatments at scale. There are 7,000 known rare diseases that affect 400 million people across the globe but only 5% of those conditions have approved treatments. Healx’s mission is to identify and progress novel therapies for the 95% of rare diseases without approved treatment.
To do this, Healx combines AI technology with drug discovery expertise and patient insight to identify existing drugs that may be repurposed and combined to treat rare diseases. This innovative approach means treatments can be found more quickly, efficiently and cost-effectively than traditional drug discovery methods allow.
Healx was founded in 2014 in Cambridge, UK, by Dr Tim Guilliams, a Biochemical Engineer and Tech Entrepreneur, and Dr David Brown, co-inventor of Viagra and former Global Head of Drug Discovery at Roche. For more information, please visit www.healx.io.
The PKD Charity was founded in 2000 by an ADPKD patient and a genetics doctor. It is patient/family led and its mission is to transform the quality of life of all those affected by PKD through providing support, raising awareness and funding research.
Over the past 20 years, the Charity has supported thousands of patients and their families coping with the often devastating impact of a PKD diagnosis and its progression. The Charity provides information written by medical experts, organises educational events (in-person and online) around the UK, moderates large online communities, and gives personalised peer support via trained volunteers and staff. The Charity also seeks to raise awareness amongst the Government, NHS and the public about the clinical, economic and psychological disease burden. It has created strong relationships with stakeholders in the kidney, liver, genetics and rare disease communities in the UK and internationally.
The Charity funds capacity-building research in the UK and funds a vital bio-resource bank of human PKD and age-matched normal control tissues, cell lines and animal models that faithfully recapitulate the genotypic and phenotypic characteristics of PKD. The Charity regularly contributes to national and international research projects and studies. For more information, please follow us on Twitter and LinkedIn.
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