PHARMExcel shortlisted for Most Impactful Industry Collaboration award for novel gene therapy for rare disease
Welwyn Garden City, UK — September 2025 — PHARMExcel is honoured to have been shortlisted for the Most Impactful Industry Collaboration of the Year at the prestigious OBN Awards 2025. The awards, now in their 17th year, aim to celebrate innovation and outstanding achievement across multiple areas of the Life Sciences industry, highlighting groundbreaking scientific advancements and contributions to unmet clinical needs from emerging startups to established firms.
PHARMExcel’s nomination at this year’s OBN Awards is for its ophthalmology and rare disease study partnership with Axovia Therapeutics, a University College London spin-out developing a world-first gene therapy for Bardet-Biedl Syndrome (BBS).
The collaboration between PHARMExcel and Axovia, brings together Axovia’s cutting-edge science with PHARMExcel’s proven expertise in ophthalmology study management, inherited retinal disease trials, and rare disease clinical trial delivery. By combining innovation with operational excellence, the partnership is setting a benchmark for how gene therapy trials in rare eye diseases can be prepared and delivered in the UK.
Award-worthy ophthalmology rare disease study
BBS is a rare genetic condition that can cause progressive vision loss, obesity, kidney failure, and developmental delays. It affects between one in 70,000 – one in 100,000 in Europe and North America, with up to 10 times that prevalence in certain populations in the Middle East. There is no curative treatment for BBS, therefore this first-in-human (Phase I) rare disease clinical trial, which PHARMExcel and Axovia Therapeutics have partnered on is particularly promising and represents new hope for patients and families.
Offering hope to patients and families is central to PHARMExcels purpose as an organisation, which approaches every clinical trial motivated to make a difference in the bigger picture of healthcare, which it feels honoured to play a small part in. PHARMExcel was founded by Yvanne Enever in 2009, based on the vision to deliver high-quality, personalised, and flexible clinical trial support that brings life changing treatments to patients faster. Of this ever-present opportunity to make a difference, Yvanne Enever says: “The fact that each trial we sponsor might result in a breakthrough that gives someone more time, improved health, or renewed hope is a very strong motivator.” This motivation in undoubtedly part of the reason why this collaboration between PHARMExcel and Axovia Therapeutics has been impactful and has subsequently been recognised as a finalist for the OBN Awards 2025
PHARMExcel, the CRO for vision loss research
While PHARMExcel is not therapeutic specific, this award winning CRO has worked extensively in delivering both clinical trials for ophthalmology therapies and rare disease clinical trials. With regard to ophthalmology clinical trials: A considerable amount of the experience accumulated in this area is the result of a long-term partnership which the company has stewarded with Moorfields Eye Hospital. PHARMExcel and Moorfields have collaborated for the past 16 years to push the boundaries of ophthalmology clinical research, improve patient outcomes, set new standards in eye care, and make significant strides in the fight against eye diseases and disorders.
It is well known that Moorfields Eye Hospital offers unparalleled advantages for clinical research in ophthalmology including: world-class expertise, a diverse patient population and cutting-edge infrastructure. As such, PHARMExcel was well positioned to partner with Axovia Therapeutics on this specific rare disease clinical trial for a world-first gene therapy for to treat BBS.
PHARMExcel, the CRO for rare diseases
When it comes to clinical trials for rare disease, PHARMExel has extensive experience in successfully delivering clinical trials for rare disease research, largely due to its UK network of specialist research sites which it has access to as a result of established partnerships with specialist NHS hospitals, UK Biomedical Research Centres(BRCs) and Clinical Research Facilities (CRF).
This network enables this UK CRO us to secure sites with the right experience in recruiting and retaining patients, which is one of the main challenges in rare disease clinical trials. Delivering clinical trials for rare conditions requires a nuanced, practical and integrated approach, which this hands-on UK CRO specialises in. PHARMExcel has been involved in clinical trials for rare diseases like Fabry disease, Behcet’s disease, Birdshot Uveitis, Myelodysplastic Syndrome (MDS) and Acute Myelocytic Leukemia – to name a few. For more information and specific rare disease case studies detailing PHARMExcel’s approach to rare disease clinical trials, visit its rare disease page on their website.
OBN Awards 2025
Of this year’s shortlisting, Yvanne Enever, CEO of PHARMExcel, says:
“We’re incredibly proud to be recognised again at the OBN Awards. Our collaboration with Axovia is a great example of the impact that can be achieved when a specialist CRO works closely with pioneering biotechs to accelerate innovative therapies for patients who urgently need them.”
This year’s shortlisting builds on PHARMExcel’s continued recognition at the OBN Awards, after being named a finalist in 2024 for Most Impactful CRO of the Year. PHARMExcel will be attending the OBN Awards on 26 November 2025 and looks forward to celebrating innovation across the life sciences sector — and, hopefully, bringing home a win.
