{"id":135568,"date":"2025-05-27T09:30:07","date_gmt":"2025-05-27T09:30:07","guid":{"rendered":"https:\/\/www.europesays.com\/uk\/135568\/"},"modified":"2025-05-27T09:30:07","modified_gmt":"2025-05-27T09:30:07","slug":"jim-wilson-left-upenn-but-hes-still-working-to-cure-rare-genetic-diseases","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/uk\/135568\/","title":{"rendered":"Jim Wilson left UPenn but he’s still working to cure rare genetic diseases"},"content":{"rendered":"

Jim Wilson\u2018s pioneering gene therapy explorations have <\/b>helped launch eight companies focused on translating his 40 years of <\/b>research, mostly at the University of Pennsylvania<\/a>, into treatments for rare diseases. <\/p>\n

During that time, he learned a formula that appealed to <\/b>investors: advance the company\u2019s science, make the company more valuable <\/b>to bring in more investors, and then sell it to a big pharmaceutical company. <\/p>\n

Then a couple years ago, Big Pharma lost interest in gene therapies<\/a> for rare diseases. \u201cWhen that avenue of liquidity evaporated, the whole thing shut down,\u201d he said.<\/p>\n

But Wilson, 69, wasn\u2018t ready to give up on his lifelong goal of developing gene therapies to cure patients with rare diseases. <\/p>\n

\u201cWhen we thought about our next step, what was critical to moving in this direction was not relying on the pharmaceutical industry to commercialize our products,\u201d he said. <\/p>\n

Wilson\u2018s answer was to leave Penn, his home for 33 years, to test a new business model for advancing gene therapy. He <\/b>formed Gemma Biotherapeutics<\/a> to <\/b>partner with countries in Latin America and the Middle East, not just to raise money but also to ensure that treatments reach populations where the need is great.<\/p>\n

He is now pitching what he calls a modular approach to getting treatments to patients: Bundle treatments for similar genetic diseases to reduce development costs, and focus on diseases for which there is a clear road map to reaching the marketplace. <\/b><\/p>\n

That spinout of Philadelphia-based Gemma from Wilson\u2018s lab at Penn happened Oct. 1. His other business interests include <\/b>Franklin Biolabs, a contract research organization that separately <\/b>spun out of Wilson\u2018s lab at the same time and set up operations in King of Prussia<\/a>.<\/p>\n

Wilson left academia at a tough time for the gene therapy sector<\/a>. Financially, investors are worried about limited applications for therapies with sticker prices that can climb into the millions, with ongoing safety questions. <\/p>\n

And the science itself is evolving. Many labs are diversifying techniques beyond the viruses Wilson discovered and has used to deliver healthy genes into the human body.<\/p>\n

He is looking to Brazil to start proving his concept. <\/b>Wilson struck his first deal with Brazil\u2018s Ministry of Health<\/a>, which agreed last year to spend up to $100 million to bring six treatments for rare central nervous system diseases to market over the next three to five years. <\/p>\n

Difficult investment landscape<\/p>\n

Wilson became interested in rare genetic diseases while in graduate school at the University of Michigan in the early 1980s. He studied a form of cerebral palsy<\/a> in boys called Lesch Nyhan Syndrome. <\/p>\n

The rare disease results in symptoms ranging from <\/b>impaired kidney function to harm-inducing <\/b>behaviors such as lip and finger biting. \u201cThey can\u2019t talk, but they\u2019re actually cognitively intact,\u201c Wilson said, noting that his five years studying these children led to his commitment to rare diseases.<\/p>\n

He has persisted, despite significant setbacks. <\/b>In 1999, an 18-year-old man named Jesse Gelsinger died after undergoing a gene therapy treatment<\/a> that Wilson developed, setting the field back by years. For about a decade, \u201cwe couldn\u2018t do anything but just hunker down and try to move our science forward.\u201d<\/p>\n

As the field entered another major downturn in investment support \u2014 in addition to Wilson becoming a grandfather, the researcher considered whether it was time to let others carry on his dream of treating rare genetic diseases. \u201cBut I don\u2018t think there\u2019s anyone but me that can really try to try to pull this off,\u201d he said.<\/p>\n

There\u2019s another factor. The notion of his retirement didn\u2018t make his wife of more than 40 years happy. \u201cCOVID didn\u2018t go well, if you know what I mean,\u201d he said.<\/p>\n

Still, Wilson\u2018s departure from Penn came amid a painful slowdown in venture capital investment in biotech. Gene therapy has been hit particularly hard. The amount of venture capital invested in gene therapy globally fell to $1.4 billion last year from a peak of $8.2 billion in 2021<\/a>, according to Reuters. <\/p>\n

The gene therapy section \u201cis in a dark place right now, and it\u2019s a shame, because the science, while not fully there, is on the cusp of some greatness,\u201d said John F. Crowley, CEO of the Biotechnology Innovation Organization, an advocacy group in Washington and a Bucks County<\/a> resident.<\/p>\n

There were signs after last fall\u2019s presidential election that private equity<\/a> and biotech investing would recover from the slump that started in 2022, but now political and regulatory turmoil under President Donald Trump<\/a> has made executives skittish about dealmaking.<\/p>\n

Pension funds, endowments, and others who poured money into funds that backed biotech startups are \u201chankering for exits,\u201d said Dean Miller, CEO of the Philadelphia Alliance for Capital and Technologies, a group that advocates for startups in the region. <\/p>\n

Despite the difficult environment, Wilson announced late last year that he had raised $34 million in seed capital<\/a> from four investment firms. Now he\u2019s out trying to raise enough to give Gemma three years of runway. He declined to say what his target is.<\/p>\n

Companies focused on <\/b>rare diseases have had an especially hard time finding financial success with gene therapies because the costs are high and the markets are small.<\/p>\n

Steven Altschuler, a biotech investor who helped found Philadelphia\u2019s Spark Therapeutics<\/a> a decade ago when he was CEO of Children\u2018s Hospital of Philadelphia, said he would not discount Wilson\u2018s ability to raise money.<\/p>\n

\u201cFor high profile people, there\u2019s still opportunity, but it\u2019s much less than it used to be,\u201d Altschuler said. <\/p>\n

Gemma\u2019s approach to the market<\/p>\n

Wilson has a two-pronged approach to addressing the business <\/b>challenges threatening the medical applications of his research. <\/p>\n

Gemma\u2019s plan is to partner with middle-income countries that have national healthcare systems to share in the cost of commercializing treatments. The company already has a deal with Brazil, and is seeking a partner in the Middle East, where Wilson said <\/b>genetic disease is more prevalent<\/a> than in other regions of the world. <\/p>\n

Gemma is also working on a bundle of treatments for similar genetic diseases to reduce development costs, with the goal of ultimately reaching $500 million in annual revenue. <\/p>\n

\u201cIf you just have one really rare disease as a company, it\u2019s not cost effective to develop a worldwide commercial enterprise to commercialize that,\u201d Wilson said. <\/p>\n

Another key to Gemma\u2019s strategy is targeting diseases that have no other treatment. Specifically, he looks for diseases and treatments with <\/b>straightforward ways to verify <\/b>that the treatment worked, even with a small number of patients in a trial.<\/p>\n

An advantage to working with national health systems is that they provide insurance coverage for life. That means the high cost of genetic cures makes sense financially in the long term because the insurer that pays for the treatment saves money over the patient\u2019s entire life.<\/p>\n

By contrast, in countries like the United States, the economics are more complicated<\/a>, experts said. <\/p>\n

\u201cThe reimbursement side of it is not really set up to absorb those significant upfront costs,\u201d Miller said of the U.S. system, because people frequently change insurance companies. That makes it hard for an insurer to pay a multimillion dollar tab for a gene therapy cure, only to see the patient leave for a different insurer.<\/p>\n

Wilson struck his first deal with a national health plan in Brazil, where he has experience with clinical trials. <\/p>\n

Gemma is on track to treat its first patients in Brazil this year, Wilson said.<\/p>\n

Brazil\u2019s health ministry will cover the cost of clinical trials involving an estimated 10 to 30 patients. That means Gemma doesn\u2018t have to spend investor money on this developmental stage, Wilson said. He estimated that trials are expected to cost from $300,000 to $1 million per participant.<\/p>\n

Wilson\u2018s approach to market and technical challenges<\/p>\n

Wilson built Gemma keeping in mind what he called a series of gates that all new treatments have to get though to succeed in the marketplace. <\/p>\n

He breaks down the steps <\/b>as: developing the right technology with the right dose, securing regulatory approval <\/b>for safety, negotiating <\/b>adequate pricing from insurers, and gaining acceptance by doctors. In other words, doctors have to prescribe the medication.<\/p>\n

Having the right dose is particularly important in gene therapies that his approaches deliver into the human body using modified <\/b>viruses, called vectors. \u201cIt\u2019s a convenient way to deliver the treatment, but it has its challenges,\u201d Wilson said.<\/p>\n

To ensure the vectors carrying the new gene reach their target, sometimes the doses have to be so high that they become toxic and potentially deadly. Plus, the cost of producing that much of the vector can be exorbitant, Wilson said.<\/p>\n

Gemma is trying to <\/b>avoid those risks by targeting treat diseases of the central nervous system, with the vector injected into the cerebral spinal fluid. \u201cYou use less of it. It\u2019s localized, more efficient, and you\u2019ll have far less toxicities,\u201d Wilson said<\/p>\n

He also aims to avoid the fate of some gene therapies, like those approved in <\/b>recent years for the blood disorder <\/b>hemophilia B, that <\/b>haven\u2018t met sales expectations<\/a> because there are cheaper, <\/b>existing treatments that work well. So doctors aren\u2018t prescribing them.<\/p>\n

Gemma\u2019s rare disease targets have no alternative treatments. That means Wilson expects to see <\/b>patients and their families line up. <\/p>\n

Wilson is targeting diseases that don\u2018t require as burdensome a treatment process as is the case for some gene therapies, which require families to move to be near an advanced care center for two months.<\/p>\n

\u201cTo get our product, you have to be on-site about three to four days,\u201d he said.<\/p>\n","protected":false},"excerpt":{"rendered":"Jim Wilson\u2018s pioneering gene therapy explorations have helped launch eight companies focused on translating his 40 years of…\n","protected":false},"author":2,"featured_media":135569,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[267,59415,70,16,15],"class_list":{"0":"post-135568","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-genetics","9":"tag-james-wilson-gemma-biotherapeutics-gene-therapy","10":"tag-science","11":"tag-uk","12":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114579101490979338","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/135568","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=135568"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/135568\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/135569"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=135568"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=135568"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=135568"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}