\n<\/p>\n
J<\/span>ust a small fraction of our 20,000 genes can cause disease when disrupted \u2013 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. In many cases, doctors won\u2019t even know which mutation is responsible, let alone how to fix it.<\/p>\n 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\u00a0David\u00a0Smith notes in New Drugs, Fair Prices, the\u00a0\u201csuccess rate, from discovery to market, is tiny\u201d and there are approved treatments for \u201cless than 10% of the 8,000\u00a0diseases that affect humans\u201d. Commercial\u00a0incentives, he argues, skew innovation towards lucrative cancer drugs and long-term treatments for large populations. Complex gene therapies for very rare conditions are seen as too costly\u00a0to develop and too small to profit from.<\/p>\n Take 2022\u2019s revolutionary gene therapy to treat a young patient with relapsed T-cell leukaemia at Great\u00a0Ormond Street hospital (GOSH) in London. Waseem Qasim, a paediatric immunologist leading that\u00a0work, warned earlier this year that \u201cwe are going\u00a0to\u00a0end up with treatments that work, but that nobody wants to pay for\u201d. Sustaining pioneering work cannot rely on philanthropy alone. His technological advance \u2013 \u201coff\u2011the-shelf\u201d edited T-cells \u2013 would require industrial-scale manufacturing to become widely available, and the\u00a0market alone will not build it. GOSH\u2019s research hints at the promise \u2013 and limits \u2013 of the therapy.<\/p>\n Biotech\u2019s boom-time cash has moved on to silicon chips and artificial intelligence. That is why a milestone in prime editing \u2013 a DNA \u201csearch and replace\u201d technology \u2013 was announced this May with a warning. Prime Medicine, the company behind the therapy, had successfully treated a teenager with a rare immune disorder but said it would halt future work. The geopolitics of big pharma is also changing. Firms are shifting research and manufacturing toward nations with scale and long-term policy support, such as China and the US. Britain looks exposed. Donald Trump has already forced the UK\u00a0to\u00a0accept higher prices for NHS medicines under\u00a0threat of tariffs. This means less cash to support patients and for breakthrough therapies.<\/p>\n Per-patient costs of gene therapies can go up to six\u00a0or seven figures. If the cure exists and the business model does not, the latter must change. Britain needs state-backed manufacturing for medical sovereignty \u2013 though it must not ignore the reality that China can already produce some gene therapies considerably more cheaply. What\u2019s needed is a renewed social contract in which innovation is shared, not hoarded. Rare-disease gene therapy could be treated like dialysis or transplants: an NHS service funded collectively, not a market good.<\/p>\n Many experimental treatments could remain in long-running NHS research programmes, offered at cost while the science matures. Only a universal, publicly funded health system \u2013 with equitable access and lifelong follow-up \u2013 can make gene therapy medicine, rather than merchandise. Without the NHS\u00a0and charities, the breakthroughs remain in the lab. With it, they become part of the common good.<\/p>\n","protected":false},"excerpt":{"rendered":" Just a small fraction of our 20,000 genes can cause disease when disrupted \u2013 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<\/p>\n","protected":false},"author":1,"featured_media":38541,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[58],"tags":[5694,1571,16774,1516,1406,1510,204,1511],"class_list":{"0":"post-38540","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-science","8":"tag-breakthroughs","9":"tag-contract","10":"tag-editing","11":"tag-editorial","12":"tag-gene","13":"tag-guardian","14":"tag-social","15":"tag-view"},"_links":{"self":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts\/38540","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=38540"}],"version-history":[{"count":0,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/posts\/38540\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=\/wp\/v2\/media\/38541"}],"wp:attachment":[{"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=38540"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=38540"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/naijaglobalnews.org\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=38540"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}