LifeArc

When Andy's daughter Olivia was diagnosed with rare Phelan-McDermid Syndrome, he made it his mission to make a clinical trial happen.

This is their story.

We’re funding an @ukdri.ac.uk Translation Awards project led by Prof Vincent Dion, testing whether gene editing techniques can lead to an improvement of symptoms.

We support our partners' therapeutic trials for new treatments and medications with the potential to transform lives.

Our wide-ranging experience, expertise and support help propel clinical trials from design and set-up through to delivery.

Learn more: http://bit.ly/4qwI2pk

Inclusion by Design shares practical recommendations to tackle structural and cultural barriers to #PPIE in academic research, with input from @rdrukhub.bsky.social and @lifearc.bsky.social Translational Centres for #RareDisease Research. Read the report here: geneticalliance.org.uk/wp-content/u...

Behind-the-scenes at The Brain House studio, our CEO, Sam Barrell discusses all things #BrainHealth with Niranjan Bose and Philip Scheltens.
#Davos2026

We're pleased to be supporting @lifearc.bsky.social and the Global Alzheimer's Platform Foundation (GAP) to launch a major research study, testing whether a finger prick-style blood test could be used to help diagnose Alzheimer’s 🩸

Find out more 👉 www.ukdri.ac.uk/news-and-eve...

We are pleased to welcome Professor Stuart Elborn CBE as our new Chair of Respiratory Disease. He will provide strategic leadership to support the translation of scientific discovery for people living with these conditions.

Today, our CEO, Sam Barrell, took to the stage at The Brain House in Davos, held alongside the @weforum.org Annual Meeting, for an impactful discussion on how to translate scientific breakthroughs in brain health into global, equitable impact.

#BrainHealth #Davos2026 #WEF26

Alzheimer's finger-prick blood test could transform diagnosis, experts say - BBC News An international trial involving 1,000 volunteers will aim to detect biomarkers associated with the condition.

We're proud to partner with Global Alzheimer's Platform Foundation and @ukdri.ac.uk on the Bio-Hermes-002 study, working towards an Alzheimer's test that's scalable, accessible and cost-effective.

t.ly/QWn1R

Professor Alfredo Iacoangeli and his team at King’s College London are exploring whether patterns of gene expression in blood could be used as an early diagnostic tool for ALS.

We’re funding this project to improve diagnosis of ALS in primary care: https://t.ly/KyRR4

We partnered with KisoJi to humanise a novel anti-TROP-2 antibody.

Now in trials with Cancer Research UK, this first-in-class therapy offers new hope for cancer. This was a global effort showcasing the power of our truly collaborative model.

Read more: https://t.ly/_cdM0

Lung conditions place a huge strain on people, health services and the UK economy. With @asthmaandlung.org.uk, we created guidance for a "simple lung test" to be used at GP appointments – cutting delays, improving referral precision and reducing costly hospital visits.

🔗Download: https://t.ly/53l1k

With @innovateuk.bsky.social and Medicines Discovery Catapult, we’re partnering on PACE to accelerate innovation in AMR research.

Read more about how we involve patients: https://www.lifearc.org/work-with-us/patients-community/

Dementia is not a sudden event. But how can we detect it earlier and, in some cases, prevent its onset?

In two weeks, we'll be at The Brain House during the World Economic Forum's Annual Meeting speaking to worldwide experts on how brain health strengthens societies and economies. #WEF26

@ukdri.ac.uk researcher Nathan Skene leads a LifeArc funded project which aims to develop a scalable test that could help inform how these variants influence disease development and progression.

We are pleased to welcome Matt Owens as our new General Counsel and Company Secretary.

Matt has a wealth of legal and governance expertise, including on fundraising, Phases II and III product development and commercialisation.

With EMBARC Network, we're supporting the AIR-NET trial. It is investigating if repurposed anti-inflammatory treatments can reduce neutrophilic inflammation and improve respiratory health outcomes in people with bronchiectasis.

@dundee.ac.uk

Learn more: https://www.lifearc.org/project/airnet/

Every month MND goes undiagnosed is a missed opportunity for treatment and planning.

Our Primer Fund is a dedicated funding stream to support early-stage, high-potential projects that could help speed up diagnosis of MND, particularly ALS, the most common form: https://t.ly/KyRR4

Through workshops, surveys and focus groups with patients, clinicians and healthcare professionals, we and @asthmaandlung.org.uk gathered insights from every angle.

These perspectives are now informing a new ‘Target Product Profile’ for diagnostics developers to use: https://t.ly/SGzIH

How could technology provide meaningful support to people with MND and their caregivers? Our recent report gives 5 recommendations, based on insights from people living with MND, their caregivers, and the wider community - https://bit.ly/4pgDfYc

Hunter syndrome: Boy with rare condition amazes doctors after world-first gene therapy Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

We’re supporting a @manchester.ac.uk trial, exploring the safety and potential of gene therapy to help reduce disease symptoms and prevent brain damage in children with Hunter syndrome.

Read about Ollie, the first child to receive this gene therapy, here: https://t.ly/nOQxK

Looking for a change in the New Year? Consider joining LifeArc and being part of a team that transforms lives.

Visit our careers page: https://www.lifearc.org/careers/

We wish U(2OS) a happy holiday season from these osteosarcoma cells showing red G3BP1 stress granules after chemical stress with sodium arsenite, used in an early validation assay for one of our MND projects.

Photo: Rebecca Girling

Involving patients in programmes like this can speed up and prioritise projects that will create effective, tolerable treatments for everyone affected by MND.

We’re funding a new clinical trial at @kingscollegelondon.bsky.social to test nasal insulin spray for Phelan-McDermid syndrome, which affects movement, speech, learning, sleep, digestion and mood. Co-designed with families, the trial aims to recruit 100 children and young adults.

2025 has been quite the year! It's nearly impossible to wrap up succinctly, with so many incredible projects underway! Our sincerest thanks to all of our partners and colleagues for making this year such an impactful one.

We have recently signed two new antibody development collaboration agreements with Laigo Bio, an innovative biotech company.

New drugs could selectively target Huntington’s disease-causing forms of the huntingtin gene – potentially preventing the expansion of protein that is toxic to cells.

We’re funding this drug development project led by Prof Sarah Tabrizi as part of the @ukdri.ac.uk Translation Awards.

We're co-funding the TIARA trial with Aplastic Anaemia Trust. The trial, conducted at @kingscollegenhs.bsky.social, is investigating the safety and optimal dosage of expanded autologous T regulatory cells to treat patients with aplastic anaemia.

https://t.ly/r2ECR

1 in 5 people will develop a chronic lung disease, yet asthma, COPD, ILD and bronchiectasis are often misdiagnosed. With @asthmaandlung.org.uk, we've shared guidance for developers to create a primary care test that helps people get the right treatment sooner.

🔗 Read the guide: https://t.ly/GkoS7