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...

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 wellness15:45. Generating hypotheses18:40 Multimodal over unimodal data22:04. Biomarkers and individual disease trajectories28:00 How to intervene to maintain wellness?30:01 A new era for supplements?32:26 Single interventions versus combinations37:59 Racial background and lifestyle41:00 Digital twins, trial design and recruitment43:30 Mocktails and mojitosThe Mojito10–12 mint leaves and mint sprig 1 Oz simple syrup (50% sugar solution)2 Oz white rum 0.75 Oz freshly squeezed lime juice. ~1–2 Oz club sodaDIRECTIONS: Place the mint leaves in a shaker tin, add the syrup and gently muddle the leaves 10–12 times. Add the rum and freshly squeezed lime juice. Shake over ice for 15 seconds and double strain over fresh ice into a Collins glass. Top up with the club soda and mix gently. Slap a mint sprig to release the aromatic oils and add it as garnish. Sources mentioned in the podcastThe supposed Native American 'diabetes gene'. Newman, AS. Peace Rev. 12, 517-524 (2010)Todd Rose. The End of Average (HarperCollins, 2016). Can a biologist fix a radio? Lazebnik, Y. Cancer Cell 2, 172-182 (2002) https://www.cell.com/cancer-cell/pdf/S1535-6108(02)00133-2.pdfLancet Commission on risk factors for dementia: Livingston, G et al. Lancet 396, 413-446 (2020) https://www.thelancet.com/article/S0140-6736(20)30367-6/fulltextPrecision Medicine Approaches for Developing Combination Therapies for the Treatment and Prevention of Alzheimer's Disease (AD) and AD-Related Dementia, National Institute of Aging, December 4-5, 2023. Khullar, D How to die in good health. New Yorker (April 15, 2024)The Mixer music “Pour Me Another” courtesy of Smooth Moves!

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 self09:26. Clinical trials and manufacturing13:40. Stealth, immune cloaking and protein manufacture16:51. Safety, cost, availability and standardization  22:00. Stampede into autoimmune disease28:59. Neurological cell therapies and beyond33:30. Off the shelf and in vivo engineering 38:13. Commercialization headaches39:51. Dan’s drink The Manhattan:2 Oz rye whiskey, 1 Oz sweet vermouth, 2 dashes aromatic bitters. DIRECTIONS: Stir on ice for 45 sec and strain over a coupe glass. Garnish with a maraschino cherry.The Mixer music “Pour Me Another” courtesy of Smooth Moves!

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 patients6:44 In vivo editing in different liver diseases11:37 The FDA stance on programmable therapy19:41 Base-editors march into the clinic25:54 Multiplexing with base editors28:57 Reaching broader patient populations33:32 Investigator-initiated trials39:27 Prime and epigenetic editing44:34 Excitement around Bridge RNAs 47:15 Kiran’s mocktailBloody Mary 3 Ways4oz (120 ml) tomato juice1/2oz (15 ml) fresh lemon juice1/4oz (7 ml) Worcestershire sauce1/2 barspoon (3 ml) prepared horseradish, or to taste2 dashes Tabasco, or to tasteCelery stick, for garnishSalt and freshly ground pepperYour choice of pickled vegetables, skewered on a cocktail pick, for garnishDIRECTIONS: Add the tomato juice, lemon juice, Worcestershire, horseradish, and Tabasco to a shaker tin with ice and gently shake for 5 seconds.  Strain into a chilled double rocks glass over a large ice cube. Garnish with the celery stick, salt, pepper, and pickled vegetables and serve. For alcoholophiles, add 2oz (60 ml) vodka to the tomato juice, lemon, Worcestershire, horseradish and Tabasco. Enjoy!Refs:Gilmore et al. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. N Engl J Med 385, 493-502 (2021) DOI: 10.1056/NEJMoa2107454Chiesa et al . Base-edited CAR7 T cells for relapsed T-cell acute lymphoblastic leukemia.  N Engl J Med 389, 899-910 (2023) DOI: 10.1056/NEJMoa2300709 Longhurst et al. CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema N Engl J Med 390, 432-441 (2024) DOI: 10.1056/NEJMoa2309149Durrant et al. Bridge RNAs direct modular and programmable recombination of target and donor DNA.  https://www.biorxiv.org/content/10.1101/2024.01.24.577089v1Keystone Meeting on Precision Genome EngineeringSomatic Cell Genome Editing ConsortiumThe Mixer music “Pour Me Another” courtesy of Smooth Moves!