“This ODD is a huge boost to our efforts in tackling these devastating, life-limiting rare diseases. Our whole strategy is centered around achieving these prestigious designations, which make development of therapeutics easier and more cost-effective,” said Dan Williams, CEO of SynaptixBio.
Isolated Hypomyelination, a less understood variant, exhibits milder symptoms compared to H-ABC. SynaptixBio’s focus on addressing less common variants of the disease aligns with its recent receipt of a £490,000 BioMedical Catalyst grant from Innovate UK.
With funding secured through investments totaling £13.2 million and support from grants, SynaptixBio is poised to advance its research into human clinical trials later this year. The ODD status provides numerous benefits, including tax credits, grants, and exemption from certain regulatory requirements and fees, along with potential market exclusivity for seven years post-approval.
This latest designation follows the company’s receipt of a Rare Paediatric Disease Designation (RPDD) last year, which could lead to a Priority Review Voucher (PRV) upon product approval. The PRV expedites FDA review processes and can be sold or transferred to offset development costs.
Rare diseases, often genetic and affecting young individuals, pose significant challenges to affected families. SynaptixBio’s approach utilizes antisense oligonucleotide (ASO) technology, which has shown promise in treating other diseases, including Duchenne muscular dystrophy.
ASOs can alter the expression of genes, in this case a specific ASO molecule targets the mutated TUBB4A gene to stop it forming toxic proteins, which in turn help build the cells that form myelin sheaths surrounding nerve fibres in the brain. With the toxic protein suppressed, other proteins step in to help form normal myelin.
The technology has been proven in the treatment of other dystrophies, including Duchenne muscular dystrophy, and is quick and cost-effective to develop.
According to a European Commission report from 2020, “1 in 17 people will be affected by a rare disease at some point in their lives. This amounts to 3.5 million people in the UK”.
There are over 7,000 known rare diseases, with more emerging all the time.
The company’s research is supported by the Children’s Hospital of Philadelphia (CHOP), a leading center for leukodystrophy studies, under a sponsored research agreement. SynaptixBio holds a worldwide exclusive license to intellectual property from CHOP, enabling the commercialization of potential treatments.
As SynaptixBio continues its efforts, it underscores the critical need for advancements in treating rare diseases, given their profound impact on individuals and families worldwide.
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