Wave Life Sciences is a clinical-stage genetic medicines company focused on developing new treatments for debilitating diseases. The company harnesses genetic information to address the underlying causes of serious medical conditions, creating therapies designed to precisely interact with genetic material.
Unlocking Genetic Medicines with the PRISM Platform
Wave Life Sciences’ foundational technology is the PRISM platform, which enables the design of precisely engineered nucleic acid medicines. This platform creates molecules that specifically target and modulate RNA, a messenger molecule carrying instructions from DNA for making proteins. RNA is a compelling target because it offers an accessible way to influence protein production, often disrupted in various diseases.
The PRISM platform supports several distinct RNA-based therapeutic approaches: RNA editing, splicing modulation, RNA interference, and antisense silencing. RNA editing involves making precise, single-nucleotide changes to RNA sequences, correcting errors in the genetic code without altering DNA. This restores proper protein function or prevents harmful protein production.
RNA splicing modulation controls how RNA messages are cut and reassembled before becoming proteins. Many genes produce different protein versions through alternative splicing. By influencing this process, therapies can encourage beneficial protein forms or suppress disease-causing ones.
RNA interference (RNAi) is a natural cellular process where specific RNA molecules silence genes by preventing their messenger RNA from being translated into protein. Wave designs small interfering RNAs (siRNAs) that degrade problematic messenger RNA, reducing the production of disease-contributing proteins.
Antisense silencing uses short, synthetic nucleic acid strands called antisense oligonucleotides (ASOs) that bind to specific messenger RNA molecules. Once bound, ASOs can block messenger RNA translation into protein or trigger its degradation. This allows for reducing unwanted proteins or modifying splicing patterns.
Diverse Applications in Disease Treatment
Wave Life Sciences applies its PRISM platform to address a range of serious genetic disorders, including alpha-1 antitrypsin deficiency, obesity, Duchenne muscular dystrophy, and Huntington’s disease. Alpha-1 antitrypsin deficiency is a genetic disorder causing lung and liver disease. Individuals produce an abnormal alpha-1 antitrypsin protein, which accumulates in the liver and fails to protect the lungs. Wave’s approach aims to reduce this harmful protein variant.
Obesity, a complex metabolic disorder, is also a target. While many factors contribute, genetic predispositions influence metabolism and appetite regulation. Therapies aim to modulate specific RNA targets involved in metabolic pathways or appetite control, potentially offering a novel way to manage weight.
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle-wasting disease caused by mutations in the dystrophin gene. The absence or dysfunction of the dystrophin protein leads to muscle weakness. Wave’s strategies for DMD focus on RNA splicing, aiming to correct or bypass genetic mutations to produce a functional dystrophin protein.
Huntington’s disease is a neurodegenerative disorder caused by a genetic mutation leading to an abnormal, toxic huntingtin protein. Wave Life Sciences is developing therapies to reduce the production of this problematic protein. By targeting the messenger RNA responsible for its creation, these therapies aim to slow or halt neurodegeneration.
Partnerships and Advancing Research
Wave Life Sciences has established collaborative agreements with major pharmaceutical companies, including GlaxoSmithKline and Takeda Pharmaceutical Company. These partnerships accelerate the research, development, and potential commercialization of novel genetic medicines. Collaborations often involve sharing expertise, resources, and financial investments to advance drug candidates.
The alliance with GlaxoSmithKline focuses on developing oligonucleotide therapies, leveraging Wave’s PRISM platform. Such agreements provide Wave with additional funding and access to broader development and commercialization capabilities. Similarly, the partnership with Takeda Pharmaceutical Company explores new therapeutic avenues, demonstrating industry recognition of Wave’s scientific approach.