SynaptixBio, the only company licensed to commercialise a treatment for a rare, deadly disease, says the technology it uses will become widely adopted for the treatment of many diseases
Oxford-based SynaptixBio, which is developing an antisense oligonucleotide (ASO) to ‘silence’ the expression of a single mutated gene, says the ASO market is set to grow dramatically, largely because of the potential benefits compared to other genetic intervention technologies.
According to a report from May this year by Global Market Insights (GMI), the market for ASOs was worth $4.4 billion in 2023 and is predicted to grow at a CAGR off 18% to reach $19.7 billion in 2032.
ASOs are short strands of synthetic DNA that stop mutated genes from producing toxic proteins. This is very different from gene therapies, or gene editing, which replace or modify existing genes, as the mutated gene is simply ‘silenced’.
Dan Williams PhD, CEO at SynaptixBio, said; “We have seen the positive impact ASOs have made on neurodegenerative diseases such as Duchenne muscular dystrophy, so we are confident that our candidate ASO will produce similar effects on the single-gene mutation that causes H-ABC, the most severe form of TUBB4A-related leukodystrophy.
“ASOs offer a range of benefits compared to gene editing; they are highly targeted, generally produce much fewer side effects, and have broad applicability.”GMI cites the increasing prevalence of neurodegenerative and genetic disorders, growing investments in research related to gene expression and delivery technologies, and the growth in regulatory approvals for antisense therapeutics as the key drivers behind this growth.
Another indicator that the market is soaring is the level of patent activity. In the last three years alone, there have been over 136,000 filed and granted, according to GlobalData’s report on Genomics in pharma: anti-sense oligonucleotides.
Dan Williams added; “Perhaps most importantly, ASOs target the molecular causes of disease, rather than just treating the symptoms. This makes them potentially game changing.”
ASOs are currently in trials for Alzheimer’s disease (University College London Hospital), where they have already demonstrated promising results, and are being investigated as potential therapies for Parkinson’s and Motor Neuron Disease.Other industry analyses, including Future Market Insights, Grand View Research and Polaris Market Research, support the prediction that the market for ASOs will grow dramatically over the next 5-10 years.
Nucleotides are molecules that form the basic structure of DNA and RNA.
Oligonucleotides are short strands of synthetic DNA or RNA.
Messenger RNA (mRNA) is a copy of DNA that travels from the nucleus to another part of the cell where proteins are made. It is the ‘sense’ part of mRNA that results in a protein.
ASOs are called antisense because they bind to the sense part of mRNA in a complementary manner, preventing it from producing its associated protein.
Increased awareness of the diseases that ASOs are well-suited to address is also a factor in the growth of the market; patient advocacy groups have been effective in campaigning to make the public, medical community and governments aware of the nature and impacts of genetic disorders.
Dan Williams noted; “Here in the UK, the H-ABC Foundation has done a remarkable job in just a few years of raising awareness amongst the public and in the medical community of the devastating impacts this disease has on those affected and their families.“
It is particularly important that such rare diseases are identified as early as possible; delayed and misdiagnoses can lead to years of unnecessary suffering.
“The new genetic screening programme announced recently by the government is a huge step in the right direction, but when it is primarily babies and young children that are affected, then we must push for more to be done.”
SynaptixBio was recently awarded a £2 million BioMedical Catalyst grant from Innovate UK to support first-in-human clinical trials of its therapeutic targeting H-ABC.
This followed an earlier grant, in November 2023, from Innovate UK to expand the company’s search for rare disease therapies.
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About SynaptixBio
SynaptixBio is a developer of therapeutics to treat TUBB4A leukodystrophy.
At the time of press, the company’s therapeutic assets are at a pre-clinical stage of development.
The company is working in accordance with applicable GxP regulations, guidelines and standards as it works towards first-in-human clinical trials.
Such clinical trials shall be conducted in accordance with Good Clinical Practice (GCP), appropriate sponsor oversight by SynaptixBio, and approval(s) by relevant authorities – including FDA and/or MHRA.
