Developing 21st Century Medicines
NEUROLOGICAL DISEASES
Developing Targeted Medicines for Neurological Diseases
Neurological diseases are among the most complex and difficult conditions to treat. Many therapeutic agents do not reach disease-relevant cells in the brain and central nervous system at sufficient levels, limiting their ability to address underlying disease mechanisms.
SMDG is applying its proprietary peptide-based NanoLigand™ platforms, expertise in disease biology and nucleic acid therapeutic design to develop targeted, potentially disease-modifying medicines. Our approach is designed to support receptor-mediated transport across biological barriers and enable therapeutic agents to reach their intended tissues and cells.
Our Neurological Programs
SMDG is developing targeted therapeutic programs for Alzheimer’s disease, Motor Neurone Disease/ALS and Huntington’s disease—three progressive neurological disorders with significant unmet medical need.
Alzheimer's Disease
Alzheimer's disease (AD) is a progressive neurodegenerative disorder involving the loss of synaptic function, neuronal injury and cognitive decline. Its complex biology includes the accumulation of amyloid-beta and tau, neuroinflammation and disruption of communication across interconnected regions of the brain.
SMDG is developing targeted therapeutic approaches that combine our understanding of disease biology with our proprietary NanoLigand™ platforms. Our aim is to enable therapeutic agents, including nucleic acids, to cross biological barriers and reach disease-relevant cells in the brain—supporting the development of medicines designed to address underlying disease mechanisms rather than symptoms alone.
Our Published Preclinical Research in Alzheimer’s Disease
In our peer-reviewed study published in Nature Communications, NanoLigand Carriers™ transported BACE1-specific siRNA across the blood–brain barrier following intravenous administration. A single injection reduced BACE1 protein expression in the brain by more than 50%, demonstrating functional gene silencing within the brain—not simply delivery or tissue accumulation.
The NanoLigand Carriers™ reached neurons and microglia without observed toxicity or inflammation under the conditions studied. These findings provide direct preclinical evidence supporting the development of targeted nucleic acid therapeutics for Alzheimer’s disease.
Motor Neurone Disease (MND/ALS)
Motor neurone disease, also known as amyotrophic lateral sclerosis, is a progressive neurodegenerative condition that damages the motor neurons controlling movement, speech, swallowing and breathing. Its biological and genetic complexity—and the difficulty of reaching affected cells throughout the central nervous system—remain major barriers to developing effective treatments.
SMDG is advancing targeted approaches for genetically and biologically defined forms of MND/ALS. By combining disease-specific research with our proprietary NanoLigand™ platforms, we aim to enable nucleic acid therapeutics to reach motor neurons and other disease-relevant cells, supporting strategies designed to reduce harmful gene activity or restore essential cellular function.
Huntington’s Disease
Huntington’s disease is an inherited neurodegenerative disorder caused by an expanded CAG repeat in the HTT gene. The resulting mutant huntingtin protein progressively disrupts neuronal function, particularly within the striatum, leading to changes in movement, cognition and behavior.
Because Huntington’s disease has a defined genetic cause, it is an important candidate for nucleic acid therapeutics. SMDG is applying its proprietary NanoLigand™ platforms to support the systemic delivery of targeted genetic medicines to disease-relevant cells in the central nervous system, with the aim of reducing harmful mutant huntingtin expression while preserving essential neuronal function.
