{"id":259550,"date":"2026-08-05T10:08:07","date_gmt":"2026-08-05T10:08:07","guid":{"rendered":"https:\/\/www.newsbeep.com\/us-pa\/259550\/"},"modified":"2026-08-05T10:08:07","modified_gmt":"2026-08-05T10:08:07","slug":"philadelphia-labs-get-funds-to-treat-rare-diseases-in-5-years","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us-pa\/259550\/","title":{"rendered":"Philadelphia labs get funds to treat rare diseases in 5 years"},"content":{"rendered":"

From Philly and the Pa. suburbs to South Jersey and Delaware, what would you like WHYY News to cover? Let us know!<\/a>\n<\/p>\n

The federal government recently awarded millions of dollars in research funding to develop gene therapies for rare diseases, on an ambitious timeline that calls for human trials in three years.\n<\/p>\n

Two of the seven recipients<\/a> are based in Philadelphia. One of them went to scientists at the Children\u2019s Hospital of Philadelphia to continue their work on gene therapy that they recently used to treat a baby<\/a> with a rare genetic disorder. The other award was given to \u00a0GEMMABio<\/a>, the gene therapy company founded by pioneer Jim Wilson, who had a three-decade-long career<\/a> at the University of Pennsylvania.\n<\/p>\n

Wilson explained that patients with rare genetic diseases have problems or mutations in their genetic code that cause health problems, and the traditional approach has been to change the patient\u2019s genes. However, many patients with rare diseases have unique mutations, so each patient might need their own, individualized gene therapy.\n <\/p>\n

Wilson and his group will work with an AI gene editing company<\/a> to develop treatments that need not be unique to each patient.\n<\/p>\n

\u201cWhat we\u2019re trying to achieve in this program is to find a way to do that efficiently and do it at scale to ensure access,\u201d Wilson said.\n<\/p>\n

He added that he is \u201cdelighted\u201d the government decided to fund this research, because the pharmaceutical business will not fund these kinds of innovative treatments for rare diseases. This is where the federal government is stepping in.\n<\/p>\n

\u201cThey\u2019re basically filling a gap with the hope that if we show that we can do it and we can do it at scale, then we can pass the baton to more traditional drug developers.\u201d\n<\/p>\n

The award<\/a> from the government\u2019s Advanced Research Projects Agency for Health will fund Wilson\u2019s research on two diseases: Maple syrup urine disease<\/a>, a condition where the body cannot break down certain parts of proteins, causing these chemicals to build up in the blood, and requiring a restrictive and special diet; and homozygous familial hypercholesterolemia<\/a>, a genetic heart condition where cells are less able to remove cholesterol from the blood, which increases the risk of heart disease in childhood.\n<\/p>\n

Right now, these diseases can be treated, but not cured.\n<\/p>\n

Wilson will also tackle the problem of how to get a gene therapy into a patient\u2019s body, which he said \u201cdefines 40 years of my career.\u201d\n<\/p>\n

He said that while gene therapy is \u201cthe ultimate drug\u201d because it addresses the cause of the problem, it is much larger than other drugs so patients cannot just swallow them. Up until now, he said every gene therapy has used a virus to deliver the treatment to a patient\u2019s body, which works, but the patient\u2019s immune system will also respond to the virus, which makes it hard to deliver the same drug again.\n<\/p>\n

\u201cIf that dose was too low or it went away, you\u2019re essentially vaccinated against your own therapy,\u201d he said.\n<\/p>\n

Wilson\u2019s research under this award would use small fat-based molecules called lipid nanoparticles<\/a> to deliver gene therapies.\n <\/p>\n","protected":false},"excerpt":{"rendered":"From Philly and the Pa. suburbs to South Jersey and Delaware, what would you like WHYY News to…\n","protected":false},"author":2,"featured_media":259551,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[8],"tags":[69,71,70,110132],"class_list":["post-259550","post","type-post","status-publish","format-standard","has-post-thumbnail","category-philadelphia","tag-philadelphia","tag-philadelphia-headlines","tag-philadelphia-news","tag-rare-diseases"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/posts\/259550","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/comments?post=259550"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/posts\/259550\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/media\/259551"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/media?parent=259550"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/categories?post=259550"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/tags?post=259550"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}