{"id":272963,"date":"2026-02-07T23:17:12","date_gmt":"2026-02-07T23:17:12","guid":{"rendered":"https:\/\/www.newsbeep.com\/nz\/272963\/"},"modified":"2026-02-07T23:17:12","modified_gmt":"2026-02-07T23:17:12","slug":"how-ai-is-helping-solve-the-labor-issue-in-treating-rare-diseases","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/nz\/272963\/","title":{"rendered":"How AI is helping solve the labor issue in treating rare diseases"},"content":{"rendered":"<p id=\"speakable-summary\" class=\"wp-block-paragraph\">Modern biotech has the tools to edit genes and design drugs, yet thousands of rare diseases remain untreated. According to executives from Insilico Medicine and GenEditBio, the missing ingredient for years has been finding enough smart people to continue the work. AI, they say, is becoming the force multiplier that lets scientists take on problems the industry has long left untouched.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">Speaking this week at Web Summit Qatar, Insilico\u2019s president, Alex Aliper, laid out his company\u2019s aim to develop \u201cpharmaceutical superintelligence.\u201d Insilico recently launched its <a rel=\"nofollow noopener\" href=\"https:\/\/fortune.com\/2026\/01\/22\/insilico-medicine-ai-mma-science-gym\/\" target=\"_blank\">\u201cMMAI Gym<\/a>\u201d that aims to train generalist large language models, like ChatGPT and Gemini, to perform as well as specialist models.<\/p>\n<p class=\"wp-block-paragraph\">The goal is to build a multimodal, multitask model that, Aliper says, can solve many different drug discovery tasks simultaneously with superhuman accuracy.<\/p>\n<p class=\"wp-block-paragraph\">\u201cWe really need this technology to increase the productivity of our pharmaceutical industry and tackle the shortage of labor and talent in that space, because there are still thousands of diseases without a cure, without any treatment options, and there are thousands of rare disorders which are neglected,\u201d Aliper said in an interview with TechCrunch. \u201cSo we need more intelligent systems to tackle that problem.\u201d<\/p>\n<p class=\"wp-block-paragraph\">Insilico\u2019s platform ingests biological, chemical, and clinical data to generate hypotheses about disease targets and candidate molecules. By automating steps that once required legions of chemists and biologists, Insilico says it can sift through vast design spaces, nominate high-quality therapeutic candidates, and even repurpose existing drugs \u2014 all at dramatically reduced cost and time.<\/p>\n<p class=\"wp-block-paragraph\">For example, the company recently used its AI models to identify whether existing drugs could be repurposed to treat ALS, a rare neurological disorder.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">But the labor bottleneck doesn\u2019t end at drug discovery. Even when AI can identify promising targets or therapies, many diseases require interventions at a more fundamental biological level.\u00a0<\/p>\n<p>Techcrunch event<\/p>\n<p>\n\t\t\t\t\t\t\t\t\tBoston, MA<br \/>\n\t\t\t\t\t\t\t\t\t\t\t\t\t|<br \/>\n\t\t\t\t\t\t\t\t\t\t\t\t\tJune 23, 2026\n\t\t\t\t\t\t\t<\/p>\n<p class=\"wp-block-paragraph\">GenEditBio is part of the \u201csecond wave\u201d of CRISPR gene editing, in which the process moves away from editing cells outside of the body (ex vivo) and toward precise delivery inside the body (in vivo). The company\u2019s goal is to make gene editing a one-and-done injection directly into the affected tissue.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">\u201cWe have developed a proprietary ePDV, or engineered protein delivery vehicle, and it\u2019s a virus-like particle,\u201d GenEditBio\u2019s co-founder and CEO, Tian Zhu, told TechCrunch. \u201cWe learn from nature and use AI machine learning methods to mine natural resources and find which kinds of viruses have an affinity to certain types of tissues.\u201d<\/p>\n<p class=\"wp-block-paragraph\">The \u201cnatural resources\u201d Zhu is referring to is GenEditBio\u2019s massive library of thousands of unique, nonviral, nonlipid polymer nanoparticles \u2014 essentially delivery vehicles designed to safely transport gene-editing tools into specific cells.<\/p>\n<p class=\"wp-block-paragraph\">The company says its NanoGalaxy platform uses AI to analyze data and identify how chemical structures correlate with specific tissue targets (like the eye, liver, or nervous system). The AI then predicts which tweaks to a delivery vehicle\u2019s chemistry will help it carry a payload without triggering an immune response.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">GenEditBio tests its ePDVs in vivo in wet labs, and the results are fed back into the AI to refine its predictive accuracy for the next round.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">Efficient, tissue-specific delivery is a prerequisite for in vivo gene editing, says Zhu. She argues that her company\u2019s approach reduces the cost of goods and standardizes a process that has historically been difficult to scale.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">\u201cIt\u2019s like getting an off-the-shelf drug [that works] for multiple patients, which makes the drugs more affordable and accessible to patients globally,\u201d Zhu said.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">Her company recently <a href=\"https:\/\/www.ddw-online.com\/geneditbio-gets-green-light-for-in-vivo-genome-editing-programme-39752-202601\/\" target=\"_blank\" rel=\"noreferrer noopener nofollow\">received FDA approval<\/a> to begin trials of CRISPR therapy for corneal dystrophy.<\/p>\n<p>Combating the persistent data problem<\/p>\n<p class=\"wp-block-paragraph\">As with many AI-driven systems, progress in biotech ultimately runs up against a data problem. Modeling the edge cases of human biology requires far more high-quality data than researchers currently can get.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">\u201cWe still need more ground truth data coming from patients,\u201d Aliper said. \u201cThe corpus of data is heavily biased over the Western world, where it is generated. I think we need to have more efforts locally, to have a more balanced set of original data, or ground truth data, so that our models will also be more capable of dealing with it.\u201d<\/p>\n<p class=\"wp-block-paragraph\">Aliper said Insilico\u2019s automated labs generate multi-layer biological data from disease samples at scale, without human intervention, which it then feeds into its AI-driven discovery platform.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">Zhu says the data AI needs already exists in the human body, shaped by thousands of years of evolution. Only a small fraction of DNA directly \u201ccodes\u201d for proteins, while the rest acts more like an instruction manual for how genes behave. That information has historically been difficult for humans to interpret but is increasingly accessible to AI models, including recent efforts like Google DeepMind\u2019s AlphaGenome.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">GenEditBio applies a similar approach in the lab, testing thousands of delivery nanoparticles in parallel rather than one at a time. The resulting datasets, which Zhu calls \u201cgold for AI systems,\u201d are used to train its models and, increasingly, to support collaborations with outside partners.\u00a0<\/p>\n<p class=\"wp-block-paragraph\">One of the next big efforts, according to Aliper, will be building digital twins of humans to run virtual clinical trials, a process that he says is \u201cstill in nascence.\u201d<\/p>\n<p class=\"wp-block-paragraph\">\u201cWe\u2019re in a plateau of around 50 drugs <a href=\"https:\/\/www.nature.com\/articles\/d41573-025-00001-5#:~:text=The%20FDA%20approved%2050%20new,NASH%20medicine%20and%20much%20more.&amp;text=You%20have%20full%20access%20to%20this%20article%20via%20your%20institution.&amp;text=The%20FDA&#039;s%20Center%20for%20Drug,Source:%20FDA.\" target=\"_blank\" rel=\"noreferrer noopener nofollow\">approved by the FDA<\/a> every year annually, and we need to see growth,\u201d Aliper said. \u201cThere is a rise in chronic disorders because we are aging as a global population\u00a0\u2026 My hope is in 10 to 20 years, we will have more therapeutic options for the personalized treatment of patients.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"Modern biotech has the tools to edit genes and design drugs, yet thousands of rare diseases remain untreated.&hellip;\n","protected":false},"author":2,"featured_media":272964,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[34],"tags":[17463,1017,10726,154116,134,527,154117,111,139,69,90809],"class_list":{"0":"post-272963","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-healthcare","8":"tag-ai-drug-discovery","9":"tag-exclusive","10":"tag-gene-editing","11":"tag-geneditbio","12":"tag-health","13":"tag-healthcare","14":"tag-insilico-medicine","15":"tag-new-zealand","16":"tag-newzealand","17":"tag-nz","18":"tag-web-summit"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts\/272963","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/comments?post=272963"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/posts\/272963\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/media\/272964"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/media?parent=272963"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/categories?post=272963"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/nz\/wp-json\/wp\/v2\/tags?post=272963"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}