deaths linked to gene therapy<\/a> has caused additional investor angst. Yet, Edinburgh-based Trogenix, a clinical-stage genetic medicine company, announced a $95 million Series A financing to support the clinical development of its gene therapies for aggressive cancers. Trogenix\u2019s first products deliver an adeno-associated virus (AAV) directly into solid tumors, expressing a combination of immune payloads\u2014essentially a viral immunotherapy\u2014as opposed to the gene replacement or modification strategies often used to treat monogenic diseases.<\/p>\nTrogenix\u2019s secret sauce addresses a fundamental challenge to genetic medicine approaches\u2014safe and specific cell targeting. To do so, Trogenix uses synthetic super enhancers (SSEs), large clusters of enhancers densely occupied by regulatory proteins to drive robust expression of specific genes, often those that define cell identity. Trogenix CEO Ken Macnamara, PhD, explained to Inside Precision Medicine, \u201cThe unique precision comes from SSEs\u2014a genomic \u2018switch\u2019 that\u2019s selective not just for a cell type, but for the most aggressive cancer stem cell state.\u201d<\/p>\n
The company\u2019s Odysseus platform maps transcriptional networks defining disease states and engineers SSEs only 160 bp in length to turn on the right immune payloads only in those cells. By integrating AI-driven discovery with synthetic biology, Trogenix can rapidly design, test, and iterate candidate therapeutic AAVs.\u00a0 \u201cWe can build and test hundreds of vectors in a living tissue context, showing selectivity against patient-derived tumor samples,\u201d said Macnamara. \u201cWe\u2019re not just hitting the tumor core\u2014we\u2019re clearing infiltrating cells in the surrounding margin while sparing healthy neurons and astrocytes.\u201d<\/p>\n
That selectivity allows Trogenix to target the \u201capex of the tumor hierarchy\u201d\u2014the master transcriptional networks that drive cancer\u2019s self-renewal and resistance. \u201cTo truly achieve curative responses, we need to tackle multiple hallmarks of cancer simultaneously,\u201d Macbanara said. Trogenix\u2019s name reflects its philosophy: the \u201cTrojan horse\u201d strategy of slipping therapeutic genes into the tumor to trigger an immune assault from within. \u201cWe\u2019re not trying to kill every cancer cell directly,\u201d Macnamara said. \u201cWe\u2019re opening the gates to the immune system.\u201d<\/p>\n
Preclinical studies using Trogenix\u2019s SSEs have shown long-lasting immune memory, no off-target toxicity, and full tumor clearance at higher doses. \u201cAt low doses, we saw residual tumors surrounded by lymphocytes\u2014evidence that the immune system had been re-educated to keep the cancer in check,\u201d he said. \u201cAt higher doses, we saw complete responses.\u201d<\/p>\n
Trogenix\u2019s lead program targets glioblastoma (GBM), supported by extensive patient-derived cell lines developed through Cancer Research UK. A Phase I\/II clinical trial is already designed to identify an optimal biological dose and expand to newly diagnosed and recurrent GBM patients. \u201cBy 29 weeks, we expect registration-quality data,\u201d Macnamara said. \u201cIt\u2019s a model for how gene therapies could reach patients faster.\u201d<\/p>\n
Getting to the finish line<\/p>\n
When Alain Reine, MD, joined Prime Medicine in early 2024\u2014initially as CFO\u2014he wasn\u2019t seeking a young, preclinical platform company. But the science of prime editing changed his mind. \u201cAs I dug in, I was blown away,\u201d Reine told Inside Precision Medicine. \u201cThis technology isn\u2019t just another version of CRISPR\u2014it can fix mutations that no other editing tool can touch.\u201d<\/p>\n
Still, the company needed direction. When Reine arrived, Prime had roughly 18 active programs and was spread too thin. \u201cWe had brilliant scientists, but we needed focus,\u201d he said. Over his first four months, he led what he called a \u201cvalue framework exercise\u201d\u2014a deep review of every program across technical feasibility, clinical tractability, competition, and commercial potential.<\/p>\n
The result was a major refocus around liver-targeted diseases, where the biology, delivery, and regulatory precedent offer the best chance of near-term success. \u201cWe know we can safely and effectively dose the liver\u2014others have proven it,\u201d Reine explained. \u201cSo we\u2019re starting where the probability of both clinical and commercial success is highest.\u201d<\/p>\n
The company\u2019s lead program is for Wilson\u2019s disease, a rare genetic disorder of copper metabolism. \u201cThere\u2019s an unmet need here\u2014existing treatments are difficult, patients struggle with compliance, and life expectancy remains shortened,\u201d he said. \u201cWhat excites us is that prime editing is the only technology that can truly fix the transversion mutations behind this disease.\u201d With an estimated 20,000+ treatable patients worldwide, the market is meaningful by rare disease standards\u2014and potentially transformative for patients.<\/p>\n
Following Wilson\u2019s, Prime plans to move into alpha-1 antitrypsin deficiency (AATD) in 2025, leveraging shared delivery and manufacturing platforms to lower cost and accelerate development. \u201cIt\u2019s faster and cheaper because it\u2019s modular,\u201d Reine said. \u201cWe can reuse the same lipid nanoparticle system and much of the toxicology data.\u201d<\/p>\n
![\"Bob](\"https:\/\/www.newsbeep.com\/il\/wp-content\/uploads\/2025\/10\/MotM_2025_panel_1-1024x694.jpg\")
Bob Smith (left), Venture Partner, OrbiMed and Chairman, Alliance for Regenerative Medicine (ARM), and Allan Reine (right), MD, CEO, Prime Medicine, discuss how strategic funding and collaboration can accelerate gene editing technology development. [Jonathan D. Grinstein]The company also recently made a tough call to wind down its chronic granulomatous disease (CGD) program, despite strong early data. \u201cIt was an emotional decision,\u201d Reine said. \u201cWe may have effectively cured two patients, but the market is simply too small to sustain a company of our size. We\u2019re exploring regulatory paths to bring that data to patients without losing financial discipline.\u201d<\/p>\n
Prime continues to invest in cystic fibrosis, supported by the Cystic Fibrosis Foundation, and is exploring future opportunities in neurological disorders\u2014though Reine says the company will wait until delivery technologies mature. \u201cWe\u2019ll get there,\u201d he said. \u201cIt\u2019s not if, but when.\u201d<\/p>\n
Looking more broadly, Reine sees the gene-editing sector stabilizing after years of hype and volatility. \u201cFive years ago, valuations were unsustainable. Then everything crashed\u2014companies were trading below cash,\u201d he said. \u201cNow, sentiment is improving for the right reasons. We\u2019re seeing real clinical data, real approvals, and pharma partnerships that validate the space. The dream is becoming reality.\u201d<\/p>\n
Reine believes Prime\u2019s discipline\u2014focusing on tractable, high-impact diseases\u2014will keep it strong through the next wave of consolidation. \u201cThis sector is growing up,\u201d he said. \u201cIt\u2019s no longer about who has the flashiest platform, but who can turn groundbreaking science into real medicines. That\u2019s the mission we\u2019re executing on.\u201d<\/p>\n
No more excuses<\/p>\n
During a panel discussing the monumental Baby KJ story<\/a> that unfolded earlier this year, when it comes to making gene editing therapies a reality for patients with rare diseases, Fyodor Urnov, PhD, scientific director at UC Berkeley\u2019s Innovative Genomics Institute (IGI), said, \u201cThere are no more excuses.\u201d Not only has the field now demonstrated that developing an on-demand personalized gene editing therapeutic is possible, but Urnov believes that the Baby KJ story has led to a rare moment of alignment between science and regulation\u2014one that could accelerate a new era of personalized CRISPR-based cures<\/a>.\u00a0<\/p>\nLater in the afternoon in a one-on-one conversation, Urnov, describing what he sees as a transformative shift in how the agency approaches gene-editing therapies, told Inside Precision Medicine, \u201cWe have to leverage the momentum of the FDA. Urnov recalled hearing senior officials speak with unprecedented enthusiasm\u2014one declaring that \u201cCRISPR gives the gift of life,\u201d another assuring him, \u201cWe\u2019re on your team; we just want to get out of your way.\u201d Even the FDA\u2019s biologics division, he noted, is exploring new approval pathways for ultra-small, bespoke trials that could bring lifesaving treatments to children with rare genetic diseases far more quickly than before.<\/p>\n
That shift, Urnov argued, opens two powerful opportunities. The first lies with the biotech industry, where many companies have quietly shelved rare-disease programs because small patient populations made large pivotal trials financially unfeasible. With regulators signaling a willingness to rethink trial design, Urnov believes firms like Intellia, Editas, CRISPR Therapeutics, Beam, and Prime Medicine could \u201cde-mothball\u201d these programs and pursue accelerated routes to approval. \u201cAnyone not taking advantage of this regulatory moment,\u201d he warned, \u201cwill spend the rest of their lives kicking themselves.\u201d<\/p>\n
The second opportunity, he said, is to expand CRISPR-on-demand\u2014customized editing therapies designed for individual patients\u2014beyond the few centers currently pioneering the approach. Groups at St. Jude, Boston Children\u2019s, and Seattle Children\u2019s are well-positioned to join UCSF and the Innovative Genomics Institute in treating critically ill children. \u201cWe should aim for dozens of \u2018Kyle Juniors\u2019 across the country in the next few years,\u201d Urnov said, referencing the now-famous child who became one of the first to receive an FDA-cleared personalized CRISPR therapy.<\/p>\n
But the science can\u2019t move without manufacturing support. Urnov called on contract manufacturers like Danaher, Synthego, Agilent, and BioNTech to \u201craise their flags\u201d and enter the gene-editing supply chain. Scaling production of CRISPR-based medicines, he said, is just as vital as designing them.<\/p>\n
Ultimately, Urnov emphasized, data\u2014not theory\u2014must lead the way. Urnov applauded the guts and planning that it took for Becca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD<\/a>, to lead the teams at the Children\u2019s Hospital of Philadelphia and the University of Pennsylvania to be the first to try. He pointed to the bravery of Victoria Gray, the first patient to receive a CRISPR cure for sickle cell disease. \u201cNobody knew if her cells would survive,\u201d he said, \u201cand they did.\u201d The trial, run by Vertex, transformed uncertainty into evidence and set the benchmark for what gene editing can achieve.\u00a0<\/p>\nFor Urnov, the moral imperative is clear. \u201cChildren still die waiting for therapies we could already be testing,\u201d Urnov said. \u201cWe can\u2019t keep building safety assays for medicines they\u2019ll never receive.\u201d Urnov called on regulators, academics, and companies to share insights more freely\u2014a call to end the \u201csiloing of regulatory learning\u201d that slows progress.<\/p>\n
The science, he said, is ready. The question now is whether those in the cell and gene therapy field can move fast enough to match it.<\/p>\n","protected":false},"excerpt":{"rendered":"As gene editing enters a new era, leaders discuss Baby KJ’s implications for families, science, and industry. (Left…\n","protected":false},"author":2,"featured_media":76886,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[25],"tags":[377,85,46,54079,54080,3173,4712,7143,141,3171,3172,54081],"class_list":{"0":"post-76885","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-genetics","9":"tag-il","10":"tag-israel","11":"tag-issue-no-03","12":"tag-may-15-2014","13":"tag-news-features","14":"tag-oncology","15":"tag-precision-medicine","16":"tag-science","17":"tag-topics","18":"tag-translational-research","19":"tag-volume-01"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/76885","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/comments?post=76885"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/76885\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media\/76886"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media?parent=76885"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/categories?post=76885"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/tags?post=76885"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}
![\"2025](\"https:\/\/www.newsbeep.com\/il\/wp-content\/uploads\/2025\/10\/MotM_2025_panel_2-696x409.jpg\" "\\"MotM_2025_panel_2\\"\/")
As gene editing enters a new era, leaders discuss Baby KJ’s implications for families, science, and industry. (Left to right): Sadik Kassim, PhD, Chief Scientific Officer and Chief Technology Officer, Danaher Genomic Medicines; Maria Kefalas, PhD, Founder, MLD Voices and The Callope Joy Foundation; Devyn Smith, PhD, CEO, Arbor Biotechnologies; Fyodor Urnov, PhD, Director, Technology and Translation, Innovative Genomics Institute and Professor, MCB Department, University of California, Berkeley; Tom Wilton, Senior Vice President, Innovation Ventures, Children\u2019s Hospital of Philadelphia (Credit: Jonathan D. Grinstein)![\"Jonathan](\"https:\/\/www.newsbeep.com\/il\/wp-content\/uploads\/2025\/10\/IMG_2114-150x150.jpeg\")
Jonathan D. Grinstein, PhD, North American Editor, Inside Precision Medicine<\/p>\n