← The Business Brew6 Mar · 1 h 35 min
Current Events - uniQure and the FDA
<p>uniQure is a business that develops gene therapies. uniQure's AMT-130 is currently in the headlines. This podcast episode attempts to present the Huntington's Disease community's arguments for why the FDA should work collaboratively to design a palatable drug trial. </p><p><br></p><p>Last week the FDA told uniQure that AMT-130 would be subject to a double blind, Phase 3, randomized controlled study. While the scientific basis for that request is sound in most cases, the Huntington's Disease community argues that path is overly burdensome; perhaps impossible. </p><p><br></p><p>This episode cites:</p><p>Dr. Sung - <a href="https://www.youtube.com/watch?si=VhMQaFAZRvQA5NRj&t=62&v=mwEEJ91LeJY&feature=youtu.be" target="_blank" rel="ugc noopener noreferrer">https://www.youtube.com/watch?si=VhMQaFAZRvQA5NRj&t=62&v=mwEEJ91LeJY&feature=youtu.be</a></p><p>Sr. Sarah Tabrizi - <a href="https://www.youtube.com/watch?v=wKj1JRfJBRE&t=356s" target="_blank" rel="ugc noopener noreferrer">https://www.youtube.com/watch?v=wKj1JRfJBRE&t=356s</a></p><p>Dr. Ed Wild - <a href="https://www.spreaker.com/episode/uniqure-update-with-dr-ed-wild--67900959" target="_blank" rel="ugc noopener noreferrer">https://www.spreaker.com/episode/uniqure-update-with-dr-ed-wild--67900959</a></p><p>FDA papers - External Control Guidance <a href="https://www.fda.gov/media/122425/download" target="_blank" rel="ugc noopener noreferrer">https://www.fda.gov/media/122425/download</a>Use of Bayesian Statistics - <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents/use-bayesian-methodology-clinical-trials-drug-and-biological-products" target="_blank" rel="ugc noopener noreferrer">https://www.fda.gov/regulatory-information/search-fda-guidance-documents/use-bayesian-methodology-clinical-trials-drug-and-biological-products</a></p><p>Senate Committee on Aging - <a href="https://www.youtube.com/watch?v=yLm1SltbRxw&t=2702s" target="_blank" rel="ugc noopener noreferrer">https://www.youtube.com/watch?v=yLm1SltbRxw&t=2702s</a></p><p><br></p><p><br></p><p><strong>Sponsorship Information</strong></p><p>Thank you to <a href="https://www.trytrata.com/" target="_blank" rel="ugc noopener noreferrer">Trata</a> for sponsoring the show.</p><p>If you're listening to this podcast, you'll like Trata. Trata is buyside to buyside conversations on individual stocks. Trata makes finding a bull or bear on any stock as easy as clicking two buttons. Over 125 funds globally contribute that collectively cover 2000+ tickers. Trata raised over $3mm coming out of Y Combinator. Before you would track 13Fs, now you can understand what funds are actually thinking. You can join as a lurker or you can join as a contributor and Trata will pay you hundreds of dollars per call. For a free trial, go to <a href="http://trytrata.com/BREWSTER" target="_blank" rel="ugc noopener noreferrer">trytrata.com/brew</a> </p><p><br></p><p>OG Sponsor Shoutout!</p><p>Thank you to <a href="fiscal.ai/brew" target="_blank" rel="ugc noopener noreferrer">Fiscal.ai</a> for sponsoring the show. </p><p><br></p><p><strong>DISCOUNT INFO:</strong> If you use the affiliate link <a href="fiscal.ai/brew" target="_blank" rel="ugc noopener noreferrer">fiscal.ai/brew</a>, you will automatically get 2 weeks of Fiscal Pro for Free and if you find that you want to upgrade, my link will get you 15% off any paid plans. <strong>About </strong><a href="fiscal.ai/brew" target="_blank" rel="ugc noopener noreferrer"><strong>Fiscal.ai</strong></a></p><p><a href="fiscal.ai/brew" target="_blank" rel="ugc noopener noreferrer">Fiscal.ai</a> is the complete modern data terminal for global equities.</p><p>The <a href="http://fiscal.ai/brew" target="_blank" rel="ugc noopener noreferrer">Fiscal.ai</a> platform combines a powerful user experience with all the financial data
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