Dan Sfera

Thoughts on this? >> Gene therapy trial shows promise in late-stage rare-disease test for Ultragenyx - The Business Journals >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #healthcare #competitivemarketing #pharma #biotech #pharmaceutical

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> #ADA24: Fractyl pitches potential of GLP-1 gene therapy, but pushes clinical trials back to 2025: A small biotech company is developing a one-time gene therapy that could turn the pancreas into its own GLP-1 drug factory and eliminate the need for frequent injections of medicines like Ozempic or Wegovy. Fractyl Health previously showed that an injection of its treatment, delivered in viral vectors injected directly into the liver, caused obese mice to lose nearly 25% of their body weight in just two weeks. At a scientific conference on Sunday, the company said that mice who got a GLP-1 drug for four weeks maintained their 22% weight loss four weeks later when they switched to the gene therapy. The updated data were presented at the American Diabetes Association’s scientific sessions in Florida. In an interview with Endpoints News ahead of the conference, Fractyl CEO Harith Rajagopalan said that the yo-yo-ing weight loss and gain seen in people who go on and off GLP-1 drugs emphasizes the need for one-and-done alternatives. “The question is shifting from how do you lose weight to how do you keep it off,” Rajagopalan said. “If the effects wear off, you are talking about limited benefit for society. But if you can make those effects permanent, you can imagine what a post-obesity society looks like.” Last year at the diabetes conference, Fractyl announced plans to begin testing its gene therapy in both diabetes and obesity patients in 2024. But that ambitious timeline has slipped — as has the company’s stock, which is down about 70% since its February IPO. Rajagopalan said the company is now planning a clinical trial in patients with diabetes for 2025. The results will inform a future trial testing a slightly different gene therapy design in people with obesity, but Rajagopalan wouldn’t say when that trial might be. Longer-term studies in animals are also underway, he said. While there’s been ongoing interest from drugmakers about how to decrease the frequency of dosing for weight loss drugs and to make their effects last longer, a permanent treatment would likely raise many of its own questions. The drugs have been associated with side effects such as muscle loss, which could become a particular issue as patients age and become more frail, for example. Novo Nordisk recently opened a research center outside of Boston focused on genetic medicines. Its leaders told Endpoints in February that it is working on genetic medicines that could complement its weight loss drugs but haven’t disclosed interest in making a gene therapy version of Wegovy. And Eli Lilly, which is also opening a genetics-focused research hub in Boston, has also remained mum on the prospects for a GLP-1 gene therapy. Rajagopalan says that the company’s endoscopic procedure for shuttling… #lucidquest #genetherapy #celltherapy

Thoughts on this? >> CMS Guidance on IRA Price Negotiation: Part 3 >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #productmarketing #healthcare #biotech #pharmaceutical