LIFE bioCEEd invests into a potentially first-in-class therapeutic platform for Amyotrophic Lateral Sclerosis (ALS) from Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico in Milan, Italy, one of Europe's foremost academic medical centres. The platform, developed in the Neural Stem Cell Laboratory of Professor Stefania Corti, is based on a novel anti-miR morpholino antisense oligonucleotide (ASO) that targets the microRNA-129-1 (miR-129-1) pathway, with the aim of achieving genuine disease modification across a broad population of ALS patients, regardless of their genetic background. The agreement was signed on 1 July 2026 and expands LIFE bioCEEd's growing portfolio of innovative biomedical programmes across Europe.

THE UNMET NEED: ALS REMAINS WITHOUT A CURE

Amyotrophic Lateral Sclerosis is a rapidly progressive neurodegenerative disease in which the motor neurons controlling voluntary movement are selectively and irreversibly destroyed. Patients experience progressive muscle weakness, loss of speech, inability to swallow, and ultimately respiratory failure, typically within 3 to 5 years of the first symptoms appearing. ALS is universally fatal, affecting up to approximately 330,000 people worldwide.

Approximately 90% of cases are sporadic, arising with no known genetic cause. The remaining 10% carry inherited mutations in genes such as SOD1, C9orf72, TARDBP, and FUS. The disease is diagnosed on average 9 to 15 months after symptoms first appear, and current therapies offer only modest palliative benefit: riluzole extends survival by 2–3 months; edaravone provides limited functional slowing in a narrow subset of early-stage patients; and tofersen, the most recently approved ASO therapy, is applicable only to approximately 2% of patients carrying the SOD1 mutation. No therapy stops the progression of the disease. No therapy is capable of restoring lost motor neuron function. This profound therapeutic vakuum, spanning the vast majority of ALS patients, regardless of disease form is precisely the gap that the miR-129-1 platform is designed to address.

THE SCIENCE: TARGETING THE UPSTREAM ROOT CAUSE

The scientific insight at the heart of this programme is that miR-129-1, a small regulatory RNA molecule is abnormally upregulated in ALS motor neurons across both sporadic and genetic forms of the disease. When overexpressed, miR-129-1 is associated with reduced levels of two proteins that are critical for neuronal health: ELAVL4 (also known as HuD), a key regulator of neuronal RNA stability, and stathmin-2 (STMN2), a protein essential for axonal maintenance and regeneration. The loss of these proteins triggers a cascade of neuronal dysfunction leading to motor neuron death.

The therapeutic approach developed by Professor Corti's laboratory uses a morpholino antisense oligonucleotide (ASO), a synthetic molecule that physically blocks miR-129-1 from binding to its targets. By inhibiting miR-129-1 activity, the ASO restores ELAVL4/HuD expression and increases STMN2, an effect observed following treatment, normalising RNA regulatory networks within motor neurons and thereby protecting them from degeneration. Because miR-129-1 dysregulation is observed across both sporadic ALS (approximately 90% of all cases) and multiple genetic forms, the therapy has the potential to benefit a broad patient population, in stark contrast to mutation-specific therapies such as tofersen, which are limited to a small genetic subgroup. The drug is administered intrathecally, directly into the cerebrospinal fluid surrounding the spinal cord, enabling targeted delivery to the motor neurons of the spinal cord and brainstem. This delivery route is already clinically established in other approved CNS ASO therapies, providing meaningful regulatory precedent.

PRECLINICAL EVIDENCE

The platform has generated a coherent body of preclinical evidence across complementary experimental systems. In a transgenic ALS mouse model (SOD1-G93A), a single dose of the anti-miR-129-1 morpholino produced meaningful survival extension alongside improvements in motor function and neuromuscular junction preservation, a notable result for a single-agent intervention in this difficult disease model. Molecular validation confirmed that the therapy is engaging its intended target and producing the desired biological response in motor neurons. Critically, the therapy has also demonstrated robust target engagement in ALS patient-derived human motor neurons carrying multiple disease-relevant mutations, with restoration of the downstream proteins lost in ALS and no detectable toxicity in the assays performed. The convergence of animal model efficacy and human cellular validation places this programme on a strong translational footing as it enters the next phase of development.

DEVELOPMENT PLAN: TOWARDS FIRST-IN-HUMAN

LIFE bioCEEd is entering the programme at the transition from advanced preclinical validation into formal IND-enabling development. The roadmap ahead encompasses the full suite of studies required to bring the therapy to first-in-human clinical testing, including regulatory toxicology, GMP manufacturing of clinical-grade material, and proactive regulatory engagement with the FDA and EMA. The programme will pursue orphan drug designation in both the US and EU, leveraging the regulatory incentives that ALS's orphan disease status confers and the precedent established by previously approved CNS antisense therapies. Total investment into the programme is anticipated to reach tens of millions of euros. LIFE bioCEEd will lead the clinical development strategy, drawing on its expertise in navigating complex regulatory pathways, to advance this therapy as efficiently as possible toward the patients who need it.

THE INSTITUTION: FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE POLICLINICO

The miR-129-1 ALS programme originated at Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico in Milan, commonly known as the Policlinico di Milano, one of Italy's oldest, largest, and most internationally recognised university medical centres and public research hospitals. As an IRCCS (Istituto di Ricovero e Cura a Carattere Scientifico), a designation conferred by the Italian Ministry of Health on institutions that stand out for their exceptional excellence in clinical and translational research. The Policlinico is part of a select group of Italian hospitals authorised to conduct biomedical research at the highest level, while simultaneously providing excellent patient care.

The institute's Neuromuscular and Rare Diseases Unit, led by Professor Stefania Corti, is one of Europe's leading centres for the study and treatment of motor neuron diseases. It offers access to cohorts of patients with ALS for clinical research, state-of-the-art infrastructure in the field of molecular biology, and extensive experience in the development of advanced RNA-based therapies.

SCIENTIFIC LEADERSHIP

The project is led by Professor Stefania Corti, Full Professor of Neurology at the University of Milan and Director of the Neuromuscular and Rare Diseases Unit at Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico. She is Principal Investigator of the Neural Stem Cell Laboratory — the scientific group that conceived and developed the miR-129-1 ALS therapy.

A physician-scientist with nearly three decades of dedicated research in neurodegenerative disease, Professor Corti has focused her career on understanding the pathogenesis of motor neuron diseases, in particular ALS, Spinal Muscular Atrophy (SMA), and SMARD1 and on developing stem cell and molecular therapies for their treatment. After completing her Medical Degree with distinction in 1997 and Board Certification in Neurology, she obtained her PhD in Molecular Medicine from the University of Milan. She has worked at the University of Milan since 1994, progressing from researcher to Associate Professor (2015) and Full Professor (2023). Professor Corti is a member of the Board of the PhD School in Molecular Medicine at the University of Milan, of the Dino Ferrari Center Scientific Committee, and of the scientific committee of the Policlinico. Her scientific contributions — spanning basic and clinical research — have been published in high-impact peer-reviewed journals, establishing her as one of Europe's foremost authorities in RNA-based neurological therapeutics.

STATEMENTS

"ALS is a disease that takes everything from a patient, progressively and without exception, while every existing treatment can do nothing to stop it. What makes this programme extraordinary is that it targets the disease at its root: an upstream RNA regulatory failure that is present across a broad range of ALS forms. The preclinical data are genuinely compelling, the scientific team behind this work is world-class, and the institution it comes from is among the most prestigious in European neuroscience. We are proud to have exclusively licensed this technology and to take responsibility for bringing it through clinical development. We believe this could be a genuine turning point for ALS patients." — Stefan Savić, CEO, LIFE bioCEEd

"This research is the result of many years of work, driven by the conviction that patients with ALS deserve a treatment that does more than simply delay the inevitable by a few weeks or months. The miR-129-1 signalling pathway represents a scientifically valid and biologically compelling target, and the preclinical evidence we have gathered constitutes, in our view, a truly solid foundation for clinical development. We are delighted to partner with LIFE bioCEEd, which brings both the strategic expertise and the commitment to patient outcomes that this programme requires. Together, we are ready to take this project from the lab to the clinic." — Professor Stefania Corti, Full Professor of Neurology, University of Milan; Director, Neuromuscular and Rare Diseases Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico