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Home » Rare diseases » Individualised medicines offer hope for children with a rare disease

Julia Vitarello

Mila’s Mum and Co-Founder, EveryONE Medicines

A commercially viable pathway for individualised medicines could make treatments accessible to rare disease patients.

What started as a race against time for my daughter Mila, diagnosed at six with a rare, fatal condition, has turned into a movement to make individualised medicines a routine way of treating genetic diseases. Instead of one drug for tens of thousands, Mila’s story points to a future of tens of thousands of drugs for just one or a few. This offers an impactful solution for millions of children with rare and life-threatening genetic diseases and no effective treatments.  

Science is no longer the barrier to individualised medicine 

Today, we have the technology to find the underlying genetic cause of a patient’s disease and design a medicine to target it. Recent advancements in genomics are enabling earlier diagnosis and allowing maturing modalities like antisense oligonucleotides (ASOs), siRNA, gene therapies and other RNA therapeutics to precisely correct distinct mutations.  

A recent publication in the scientific journal ‘Nature’ by Dr Timothy Yu, pioneering designer of Mila’s medicine, indicates that individualised ASOs could potentially correct mutations in approximately 15% of children with ataxia-telangiectasia. If a similar statistic applies across genetic diseases and other programmable modalities come online, millions could benefit from this approach. 

We have the technology to find the underlying
genetic cause of a patient’s disease and
design a medicine to target it.

Despite these promising advancements, a clear, rapid and affordable pathway for this new paradigm is missing. Current regulatory requirements are disproportionately cumbersome and solely academic-focused, disincentivising companies from entering this field. It takes highly motivated families, clinicians and organisations with the ability to raise substantial philanthropic funds to develop these treatments — neither scalable nor sustainable. 

Building a commercially viable pathway 

To bring effective individualised medicines to rare disease patients at scale, we need investment. This was the motivation behind starting the US-based biotech, EveryONE Medicines. Like Novartis, which spearheaded the CAR-T industry, it will take one company to prove this model commercially viable to open up the field. With aligned regulators, health officials, clinicians and payers, we can build the infrastructure to ensure all children like Mila have rapid access to transformative medicines. The question is: will we? 

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