"Just a small fraction of our 20,000 genes can cause disease when disrupted yet that sliver accounts for thousands of rare disorders. The difficulty is: what can a doctor do to treat them? In a common condition such as type 2 diabetes, the underlying biology is similar for millions of patients. The doctor can prescribe metformin. But with a genetic disorder, the mutation might only affect a small number of people worldwide."
"Novel gene-editing breakthroughs are making headlines. But therapies are expensive and complex to develop. The cost of bringing any new drug to patients is now around $2bn, in part because, as Brian David Smith notes in New Drugs, Fair Prices, the success rate, from discovery to market, is tiny and there are approved treatments for less than 10% of the 8,000 diseases that affect humans."
A small fraction of the roughly 20,000 human genes can cause disease when disrupted, producing thousands of rare disorders. Many genetic mutations affect only a handful of patients, making diagnosis and targeted treatment difficult. Common conditions share biology across millions, enabling standard therapies like metformin for type 2 diabetes. Bringing new drugs to patients costs about $2bn and has a very low success rate, with approved treatments for under 10% of roughly 8,000 human diseases. Commercial incentives favor profitable oncology and chronic-condition drugs, leaving complex gene therapies for ultra-rare conditions underinvested. Pioneering edited T-cell therapies and prime-editing milestones show promise but face manufacturing, funding, and market-scalability barriers.
Read at www.theguardian.com
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