[S]tunning advances in genetic science have revealed the subtle, insidious culprits behind [rare genetic] diseases and have started paving the way for treatments. But patients with these exceedingly rare mutations have fewer options and poorer prospects than those with more typical forms of these diseases — and many are now pinning hopes on experimental gene therapies.
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It’s not just science that is working against these patients, it’s market forces. Drug companies are naturally going to look for medications that target the most common mutations.
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While there’s little chance of changing market dynamics, researchers said, one solution is to develop “mutation agnostic” gene therapies targeting all patients with a disease. This approach is being tried in diseases of the retina as well as cystic fibrosis.
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“There’s a huge push to develop these therapies,” said Dr. Garry Cutting of the Johns Hopkins Cystic Fibrosis Center.
