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

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

We partnered with KisoJi Biotechnology to humanise novel antibodies targeting the cancer target TROP-2. Using our humanisation expertise, we developed stable, potent antibodies that demonstrated tumour reduction in preclinical models. 🧬💥 t.ly/xEBFj

Proposals from 31 countries across 5 continents, narrowed to 4 groundbreaking research projects that we're supporting to explore whether existing medicines can be repurposed to treat motor neuron disease (MND), also known as amyotrophic lateral sclerosis (ALS). Meet the projects ⬇️

Living with motor neuron disease (MND) brings significant daily challenges. Our new report shares insights from people living with MND, their caregivers, and the wider community about the most pressing issues, and how technology could help: https://bit.ly/4pgDfYc

@ukdri.ac.uk researchers are testing an RNA-editing technique that could help prevent the expansion of the most common genetic cause of ALS and FTD. This fascinating research is one of 5 projects we’re funding as part of the UK DRI Translation Awards.

Today, we are pleased to welcome Dr Alessandra Gaeta as our new Head of Rare Disease. Alessandra has more than 20 years of experience in research and innovation, including senior roles at @nihr.bsky.social, the Medicines Discovery Catapult and most recently, Duchenne UK.

Motor neuron disease is a devastating condition without a cure. Our in-house induced pluripotent stem cell (iPSC) technology provides accurate disease models to support drug discovery projects and research in motor neuron disease. 📷: Our iPSC scientists in Stevenage

We believe the research community has a responsibility to make its findings accessible to those it aims to help. That's why we're supporting Readable Research, a website that offers lay summaries of scientific papers reviewed by people with lived experience. 📘Start reading: https://t.ly/-Zydr

Diagnosing chronic lung conditions can take years, partly due to a lack of tools in primary care. With @asthmaandlung.org.uk, we've developed a Target Product Profile (TPP) to guide developers in creating a simple diagnostic test that can be used in primary care. Read TPP: https://bit.ly/4rlIcQH

We are co-funding a new clinical trial to investigate if a one-off gene therapy to replace faulty genes in stem cells can bring hope to children with Hunter syndrome. Ollie is the first child to participate in this @manchester.ac.uk study: bit.ly/49BaCjt

Entries for the #LongitudePrizeOnALS close on 3 December. This global Prize supports AI-led approaches to validate new drug targets for ALS/MND. We're helping shape its impact by identifying promising therapeutic hypotheses. https://bit.ly/3HBz0VW @mndassoc.bsky.social @challengeworks.bsky.social

Antimicrobial resistance (AMR) is one of the greatest threats to public health, with the potential to undo decades of medical progress. This #WAAW2025, head to our LinkedIn to hear from world-leading experts on how we can tackle AMR and protect the future of medicine: t.ly/3Oxk1

UTIs are the second most common reason for antibiotic use globally and inappropriate use is contributing to the growing crisis of antimicrobial resistance (AMR). This #WAAW2025, we're spotlighting some of the projects we are supporting to address AMR. Today's spotlight - DOSA2: https://t.ly/Z-k9x

It was powerful to see the many stories of how antibiotics have changed people's lives as part of the Fleming Initiative's takeover of the Piccadilly Lights in London last night. #WAAW2025

The @who.int placed hospital-based antibiotic stewardship programmes at the core of its Global Action Plan. This #AMRAwarenessWeek, we're spotlighting projects we're supporting to help address AMR worldwide. Today: The ASPIRE program led by ReAct Africa: https://t.ly/iLrTi

We put lived experience at the heart of our work – and want to empower others to do the same. This new report shares insights from a workshop on advancing patient public involvement in translational research and 10 top priorities for action. 🔗 Read the report: https://t.ly/Z6Oap

The first patient has been treated in an early-phase clinical trial investigating whether a new medical device, Carbalive, can slow disease progression of a rare liver disease. We are proud to fund this trial through our Pathfinder Programme. 🔗 https://t.ly/0BYtw

Patient voices have been involved at every stage of establishing a UK-wide network of research hubs improving how lung infections are managed. Read more about how we work with patients: https://www.lifearc.org/work-with-us/patients-community/ @cysticfibrosis.org.uk

Yesterday, over 100 people working on the Translational Centres for Rare Disease gathered to share achievements from their first year of operation and upcoming plans, as well as discuss important themes that affect the rare disease ecosystem in the UK.

If you weren't able to attend #GAMRIC2025, or you want to catch up on sessions you missed, a comprehensive round-up is now live, covering each session of the conference in detail ➡️ https://www.gamric.org/about-gamric/conference-round-up/ @carb-x.bsky.social @escmid.bsky.social @gardp.bsky.social#GAMRIC2025

Researchers, philanthropists, industry and other partners working in childhood cancer -- join our international consortium, C-Further, with your expertise, capabilities, ideas and investment.

Just like yesterday's fireworks displays, this network of mature motor neurons is colourfully displaying their expression of MAP2 (red), ISL1 (orange), and SMI32 (green) with their blue nuclei. This image is courtesy of our team working on our iPSC platform. Photo: Will Stebbeds

What does patient engagement mean to us? Working with patients, carers and communities to gather insights that shape research and improve health outcomes. From setting priorities to guiding funding decisions, the patient voice is at the heart of our work. 🔗Learn more: https://t.ly/9e8yO

Rare disease research needs a more integrated and collaborative approach. Next week, experts from across our 4 Translational Centres for Rare Disease, will meet to discuss the year's progress, share knowledge, and discuss the challenges and opportunities to come.

We are pleased to see this new paper from the @mhragovuk.bsky.social, committing the UK to major reform in therapies for rare disease. The UK has the potential to be a hub for innovative rare disease research and care. t.ly/usXH6

Happy Halloween from some spooky A549 cells and their E3 ligase expression (or haunting lack of in the nucleus). Photo credit: Chloe Sanders

TROP-2 is an attractive cancer target that is overexpressed on solid tumours. Yet, developing effective safe therapies targeting it remains a challenge. With KisoJi, we've humanised the first unconjugated anti-TROP-2 antibody showing strong efficacy in vivo: https://t.ly/qRRSs

Science + precision + creativity: 3 of our chemical biologists recently learned to use a cutting-edge liquid-handling robot for high-throughput experimentation, and tested their skills by programming it to dispense coloured solutions into a 384-well plate to recreate our logo 🧪

Epidermolysis bullosa (EB) is a rare genetic condition causing extremely fragile skin that blisters and tears easily. With no cures currently available, we've teamed up with @charitydebra.bsky.social to fund a drug-repurposing clinical trial to help change that. 🔗 Learn more: t.ly/vUByj

In 2024, we held 4 workshops with our MND Insights Group, conducted 2 community surveys and published the MND Insights Report, sharing the findings with researchers and decision-makers. You can find out more about this group and the report here: https://www.lifearc.org/project/mnd-insights-group/

Monoclonal antibodies save lives — but high manufacturing costs keep them out of reach in low-resource settings. With the Gates Foundation, we've awarded over $5 million to 7 projects aimed at cutting manufacturing costs and making mAbs more affordable. Read about the projects: https://t.ly/mrjH-

Through our Childhood Cancer Translational Challenge, we're committed to helping deliver better treatments, better decisions and better trials for children with cancer. Hear from David Jenkinson on how we've begun delivering on that promise: https://t.ly/aycRf

@marcaurelbusche.bsky.social is exploring exactly that in one of the 5 projects we’re funding as part of the @ukdri.ac.uk Translation Awards: https://t.ly/C3vOl

With Professor Joanna Zakrzewska at @ucl.ac.uk, we analysed GP records to uncover patterns in trigeminal neuralgia diagnosis –building the evidence needed to establish centres of excellence for faster, earlier care.

ICYMI: A new round of funding for antimicrobial resistance innovators has opened through PACE. The round closes 5 November, supporting new therapeutics for serious bacterial infections that pose the greatest risk to human health. Link to apply in the comments! onenucleus.com/pace-announc...

🎓 This year, we have industrial placement students joining our teams across data science, molecular and cellular pharmacology, analytics, and more! We're proud to support the next generation of scientists, helping them develop their skills and make an impact early in their careers.