Oxford-based SynaptixBio is advancing the development of an antisense oligonucleotide (ASO) aimed at inhibiting the expression of a single mutated gene. The company has identified a significant growth trajectory in the gene silencing market, driven by the advantages it offers over other genetic intervention methods.
SynaptixBio recently secured a £2 million BioMedical Catalyst grant from Innovate UK to back its initial clinical trials involving humans, focusing on a therapy for H-ABC. This follows an earlier allocation from Innovate UK in November 2023 to widen the company’s exploration of treatments for rare diseases.
According to a Global Market Insights (GMI) report from May 2024, the ASO market was valued at $4.4 billion in 2023 and is forecasted to grow at an 18% CAGR, reaching $19.7 billion by 2032.
ASOs are synthetic DNA strands that prevent mutated genes from producing harmful proteins, unlike gene therapies or gene editing, which involve replacing or altering genes—here, the mutated gene is “silenced.”
Dr. Dan Williams, CEO of SynaptixBio, stated, “We’ve observed the positive impact ASOs have brought to neurodegenerative diseases such as Duchenne muscular dystrophy, promising similar results for H-ABC, linked to a singular gene mutation. ASOs present numerous benefits, including targeted action and fewer side effects, compared to gene editing.”
According to GMI, factors such as the rising occurrence of neurodegenerative and genetic illnesses, increased research investments into gene expression/delivery technologies, and more regulatory endorsements for antisense treatments are key to this market expansion.
Patent activity further indicates market growth, with over 136,000 filings and grants in the past three years, as noted in GlobalData’s genomics report on antisense oligonucleotides in pharmaceuticals.
“Crucially, ASOs attack the molecular foundation of diseases instead of just managing symptoms, raising their potential for game-changing impact,” he added.
ASOs are being trialed for Alzheimer’s disease at University College London Hospital, showing early promise, with exploration also underway for potential treatments for Parkinson’s and motor neuron disorders.
Other market research bodies like Future Market Insights and Grand View Research corroborate the estimation that ASOs’ market will expand significantly in the coming 5-10 years, as highlighted by Dr. Dan Williams.
“The H-ABC Foundation in the UK has done exceptional work, rapidly increasing disease awareness among the public and medical professionals. Diagnosing these rare diseases early is crucial as missed or delayed diagnoses can lead to prolonged suffering,” Dr. Dan Williams remarked, advocating for enhanced efforts in light of the new government-initiated genetic screening program—recognizing that infants and children are primarily impacted by such conditions.