Send us a text Andy and JC get the lowdown from Robert Pacifici, CSO of CHDI Foundation, on the latest in Huntington's treatments from the recent 20th CHDI Therapeutics Conference in Palm Springs, California. Robert has spearheaded efforts at CHDI for the past 22 years, driving forward preclinical drug discovery programs and corralling efforts to develop bioassays, clinical biomarkers, patient-staging systems, clinical and regulatory consortia to galvanize industry efforts in this devastating...
All content for The Translational Mixer is the property of Andy Marshall and is served directly from their servers
with no modification, redirects, or rehosting. The podcast is not affiliated with or endorsed by Podjoint in any way.
Send us a text Andy and JC get the lowdown from Robert Pacifici, CSO of CHDI Foundation, on the latest in Huntington's treatments from the recent 20th CHDI Therapeutics Conference in Palm Springs, California. Robert has spearheaded efforts at CHDI for the past 22 years, driving forward preclinical drug discovery programs and corralling efforts to develop bioassays, clinical biomarkers, patient-staging systems, clinical and regulatory consortia to galvanize industry efforts in this devastating...
Send us a text Andy and JC get the lowdown from Robert Pacifici, CSO of CHDI Foundation, on the latest in Huntington's treatments from the recent 20th CHDI Therapeutics Conference in Palm Springs, California. Robert has spearheaded efforts at CHDI for the past 22 years, driving forward preclinical drug discovery programs and corralling efforts to develop bioassays, clinical biomarkers, patient-staging systems, clinical and regulatory consortia to galvanize industry efforts in this devastating...
Send us a text William Pao, CEO of Revelio Therapeutics, and former Chief Development Officer for Pfizer/Head of Roche pRED, sits down with JC and Andy to talk about his new book: 'Breakthrough: The quest for life-changing medicines'. By charting the ups and downs of eight different drugs and the challenges faced by those shepherding them through the complex drug approval process, the book provides a fascinating peek into the hidden world of pharmaceutical discovery and the tireless work of t...
Send us a text Sebastian Giwa, co-founder of the Organ Preservation Alliance and the Biostasis Research Institute and serial entrepreneur, talks to JC and Andy about the promise of organ storage research in transplantation. Seb obtained a PhD in Economics at the Stockholm School of Economics and was named a Baker Scholar at Harvard and worked at Bridgewater Associates, Bain and Goldman Sachs. Since spending time at Singularity University/NASA's Global Solutions Program and working with the Ob...
Send us a text Andy and JC explore the murky world of cyberthreats to biopharma and biomanufacturing with Charles Fracchia, CEO of a Boston startup Black Mesa, currently in stealth mode and co-founder of BIO-ISAC, a BIO-Information Sharing and Analysis Center to educate about threat intelligence, vulnerability identification and mitigation strategies. Charles was previously CEO of BioBright, one of the first life science companies providing end-to-end encrypted data collection and analysis in...
Send us a text https://bit.ly/3YxRltJ. Andrew Lo, Charles E. & Susan T. Harris Professor of Finance and director of the Laboratory for Financial Engineering at the MIT Sloan School of Management, gives JC and Andy the skinny on his progress in finding new commercialization models for rare diseases. He also reveals plans on implementing his debt securitization megafund model, first described over a decade ago. Finally, he gives us some tips on how to prepare a delicious refreshing sparklin...
Send us a text Anne Wyllie, principal investigator at the Yale School of Public Health and pioneer of the Saliva Direct initiative, talks to JC and Andy about the potential of open and collaborative models to transform infectious disease diagnostics and pandemic preparedness. 1) The Gin & Tonic 1 cup ice cubes 2 Oz gin ~4 Oz Schweppes Tonic Water 1 slice Lime/lemon DIRECTIONS: Pour 2 Oz gin into your favorite glass, ideally a large goblet full of ice, as shown. Top with ~4 Oz toni...
Send us a text Veronique Kiermer, Chief Scientific Officer and Executive Editor at the Public Library of Science, talks about the myriad ways in which open science is changing the face of research and some of the challenges it poses for AI and the translational arena. 01:55 What is open science? 03:55 What are barriers to openness? 07:28 Early adopters 10:30 Open challenges for AI 11:35 Registered reports and publication bias 14:20...
Send us a text Nathan Price, currently on leave from the Institute for Systems Biology in Seattle and Chief Scientific Officer at Thorne HealthTech, talks to JC and Andy about his data-driven approach to understanding health and predicting personal trajectories into disease as we age. 03:24 What is scientific wellness? 06:53 Correlates of scientific wellness 15:45. Generating hypotheses 18:40 Multimodal over unimodal data 22:04. Biomarkers and individual disease trajectories 28:00 How to int...
Send us a text UCSD's Dan Kaufman, an innovator in the field of induced pluripotent cell (iPSC)-derived natural killer cell therapies, talks to Andy and JC about the latest in allogeneic and autologous immune and regenerative cell therapies at the 2024 Keystone symposium on Emerging Cell Therapies. 02:55 Sourcing and expanding NK cells 05:59 Off-the-shelf versus self 09:26. Clinical trials and manufacturing 13:40. Stealth, immune cloaking and protein manufacture 16:51. Sa...
Send us a text UPenn's Kiran Musunuru, a human geneticist and practicing cardiologist who has pioneered the translation of gene- and base-editing approaches, talks to JC and Andy about the latest clinical results and modalities discussed at the 2024 Keystone symposium on Precision Genome Engineering. 4:07 Impacting patients 6:44 In vivo editing in different liver diseases 11:37 The FDA stance on programmable therapy 19:41 Base-editors march into the clinic 25:54 Multiplexi...
Send us a text Scripps' Eric Topol is a visionary in the application of artificial intelligence to medicine. He has a wide-ranging conversation with JC and Andy about : 02:52 Multimodal AI is coming 06:44 FDA-approved AI software 10:02 How to validate AI models? 12:50 Synthetic doctor’s notes and other early applications 15:38 Thinking about the model and its training 19:28 Dealing with hallucination and GPT5 24:13 Low-to-middle income countries 27:40 Uptake by the medical c...
Send us a text Pete Kirkpatrick, Chief Editor of Nature Reviews Drug Discovery, gives Andy and JC the lowdown on a Nature Conference on RNA therapeutics and what innovations he is seeing in the field of mRNA therapies. 01:44 Nature conference on RNA therapies 08:11 Differences between mRNA therapeutics and mRNA vaccines 14:24 mRNA chemistries 18:08 mRNA manufacturing 22:05 mRNA delivery 27:21 Delivering LNPs to organs other than liver 34:...
Send us a text Andy and JC get the lowdown from Robert Pacifici, CSO of CHDI Foundation, on the latest in Huntington's treatments from the recent 20th CHDI Therapeutics Conference in Palm Springs, California. Robert has spearheaded efforts at CHDI for the past 22 years, driving forward preclinical drug discovery programs and corralling efforts to develop bioassays, clinical biomarkers, patient-staging systems, clinical and regulatory consortia to galvanize industry efforts in this devastating...
