Touching Base

Gene therapies take center stage in this week’s episode. We discuss a potential gene replacement therapy for people with Dravet Syndrome that scientists have tested in mice with good results and highlight some recent progress from two editing companies. We also break down the recent news from Sarepta about the death of a patient after treatment with its gene therapy for Duchenne muscular dystrophy. In other stories, new data reveals a nonviral method for introducing transgenes into human primates and AstraZeneca steps up R&D in China with new drug, vaccine, and healthcare partnerships in the billions of dollars.Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Julianna LeMieux, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news. Listed below are links to the GEN stories referenced in this episode of Touching BaseGene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome By Uduak Thomas, GEN, March 19, 2025 DMD Patient Dies After Treatment with Sarepta Gene TherapyBy Alex Philippidis, GEN Edge, March 18, 2025 PiggyBac Transposon System Creates Transgenic Cynomolgus MonkeysBy Julianna LeMieux, PhD, GEN, March 26, 2025 From Tools to Trials: Editing Therapy Companies Pivot to DevelopmentBy Alex Philippidis, PhD, GEN Edge, March 20, 2025 AstraZeneca Commits up to $11B+ to Chinese Collaborations, Beijing R&D Hub By Alex Philippidis, PhD, GEN Edge, March 21, 2025 

GEN editors discuss both the news and history of DNA and genetic engineering. We present a recap of the 50th anniversary Asilomar conference, delving into some of the history from the 1975 Asilomar conference and relating to the current discussions around genetic engineering. Science has been under fire recently and many people attended the Stand Up for Science Rally at one of a few dozen locations across the United States. One of the speakers at the rally in Washington, DC, was former NIH director Francis Collins, MD, PhD, a key member of the Human Genome Project. We discuss some of his career in this episode.  Join GEN's managing editor Corinna Singleman, PhD, editor in chief John Sterling, and editorial director Kevin Davies for a discussion of DNA news and history.   Listed below are links to the GEN stories referenced in this episode of Touching Base:  Scientists in NYC Rally to Defend and Stand Up for Science   By Corinna Singleman, PhD, GEN, March 10, 2025  Former NIH Director Francis Collins Praises the Institution as He Abruptly Departs After Three Decades By Kevin Davies, PhD, GEN, March 3, 2025 

In honor of Rare Disease Day, we discuss news from Healx, a clinical-stage biotech, which has dosed the first patient in a Phase II trial of its new drug for neurofibromatosis type 1. In the world of AI, the Arc Institute in collaboration with Nvidia has dropped Evo 2, the “largest publicly accessible AI model for biology to date,” for designing genomes. Additionally, new work from Nobel Laureate, David Baker, uses AI to generate enzymes from scratch. We also dig into the big announcements from Illumina and Roche that got the community buzzing ahead of this year’s AGBT meeting. Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news. Listed below are links to the GEN stories referenced in this episode of Touching Base: Healx Candidate, SpringWorks Therapy Expand NF1 Treatment OptionsBy Alex Philippidis, GEN Edge, February 25, 2025 Arc Institute’s AI Model Evo 2 Designs the Genetic Code Across All Domains of Life By Fay Lin, PhD, GEN Edge, February 19, 2025 AI-Driven Protein Design Produces Enzyme that Mimics Natural Hydrolase Activity By Corinna Singleman, PhD, GEN, February 13, 2025 Illumina Unveils Spatial Technology Days Before AGBT Meeting By Julianna LeMieux, PhD, GEN, February 19, 2025 StockWatch: Illumina Tumbles on Q4 Results, China Retaliation By Alex Philippidis, GEN Edge, February 8, 2025 Roche Announces SBX Technology, Creates Sequencing Buzz By Julianna LeMieux, PhD, GEN, February 21, 2025 StockWatch: NIH Indirect Cost Cuts Shake Tools Stocks By Alex Philippidis, GEN Edge, February 17, 2025 

Season 2 Episode 1 (February 14, 2025): This week, the GEN editors discussed some interesting and developing stories around AI, including an in depth dialogue about what Recursion has been doing lately. They also discussed gene editing updates in delivery systems, Cas12 preclinical work, and AgBio. The discussion was rounded out with a look forward into takeover targets for 2025.   Featuring Alex Philippidis (Senior Business Editor, GEN), Fay Lin, PhD, (Editor, GEN Biotechnology), Uduak Thomas (Senior Editor, GEN), and moderated by Corinna Singleman, PhD, (Managing Editor, GEN and IPM)  Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: As Pipeline Advances, Recursion Expands AI Focus to Clinical TrialsBy Alex Phillipidis, GEN, Jan 30, 2025. Recursion Announces Promising Clinical Data on Lead AI-Based Drug Candidate for Brain DiseaseBy Fay Lin, PhD, GEN Edge, Feb 5, 2025. AAV Delivered NanoCas CRISPR System Edits Muscle in Non-Human PrimatesBy GEN, Feb 3, 2025. Next-Gen Cas12a System Enables Precise Single and Multiplexed Gene Editing in CancerBy Corinna Singleman, PhD, GEN, Jan 30, 2025. AgBio Companies Embrace Gene Editing for Stronger Food FutureBy Uduak Thomas, GEN Feb issue, Feb 3, 2025. Top 10 Takeover Targets of 2025By Alex Phillippidis, GEN, Feb 3, 2025.

Episode 19 (April 26, 2024): This week, the GEN editors celebrated DNA Day by sharing some stories about their favorite observations and applications of DNA research. Featuring Kevin Davies, PhD (Editorial Director, GEN), John Sterling (Editor in Chief, GEN), Uduak Thomas (Senior Editor, GEN), Alex Philippidis (Senior Business Editor, GEN), Fay Lin, PhD (Senior Editor, GEN Biotechnology), Jonathan Grinstein, PhD (Senior Editor, GEN), and moderated by Corinna Singleman, PhD (Managing Editor, GEN and IPM). Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: The Life and Times of Rosalind FranklinGEN Webinar presented by Matthew Cobb, PhD, Oct 16, 2020. "Life Story" BBC film (1987). Nobel Winner Svante Pӓӓbo Discovered the Neandertal in Our GenesBy Daniela Mocker, Scientific American, Oct 4, 2022. “Svante Pӓӓbo, reader of the Neanderthal genome”Wielgus, Danielewski, and Walkowiak, Acta Physiol, Nov 23, 2022. Colossal’s Woolly Mammoth Project Advances as Elephant iPSCs Clear MilestonesGEN, Mar 7, 2024. The State of Omics 2024GEN Summit. “Genetic Ancestry Testing What is it and Why is it Important?”Jorde and Bamshad, JAMA, Mar 27, 2020. A Leading Cause of Autism Spectrum Disorders May Be Prevented by Novel ApproachGEN, May 21, 2023. AI Finds Autism-Causing Mutations in “Junk” DNAGEN, May 28, 2024. Pollution and Stress Alter Brain Development and Social Behavior in Male MiceGEN, Aug 3, 2022. “Mechanistic Basis of Resistance to PCBs in Atlantic Tomcod from the Hudson River”Wirgin, et al., Science, Mar 11, 2011.

Episode 18 (April 19, 2024):    Biology’s exploding data streams continue to leverage artificial intelligence (AI) tools to streamline drug development. How do we define AI in drug discovery? Who are notable players in the space? How do humans fit into these growing computational pipelines?   This week, Jonathan Grinstein, PhD (Senior Editor, GEN) and Fay Lin, PhD (Senior Editor, GEN Biotechnology) unpack one of the fastest growing topics in the biotech field, AI in drug discovery.  Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base:  The Difference Is the Data: Drug Discovery’s AI Revolution By Fay Lin, PhD, GEN Biotechnology, April 18, 2024  Archimedes’ Box: Diagonal Therapeutics Raises $128 Million to Discover Agonist Antibodies By Jonathan Grinstein, PhD, GEN Edge, April 3, 2024  Generate:Biomedicines Raises Largest Biotech Series C of 2023 By Jonathan Grinstein, PhD, GEN Edge, September 14, 2023  Alex Zhavoronkov Aims to Take Over the Drug Development World with AI By Jonathan Grinstein, PhD, GEN Edge, June 20, 2023  Diffusion Evolution: New Artificial intelligence Models Break Barriers in Protein DesignBy Fay Lin, PhD, GEN Biotechnology, October 16, 2023

Episode 17 (April 12, 2024):    This week, the GEN editors discuss the launch of Nvelop Therapeutics, a new start-up leveraging approaches developed by gene editing pioneers David Liu, PhD, and J. Keith Joung, MD, PhD, to advance delivery of genetic cargo. The GEN editors also recap highlights from the American Association for Cancer Research (AACR) annual meeting held in San Diego, California.  Featuring Uduak Thomas (Senior Editor, GEN), Alex Philippidis (Senior Business Editor, GEN), Julianna LeMieux, PhD (Deputy Editor-in-Chief, GEN), and Jonathan Grinstein, PhD (Senior Editor, GEN), and moderated by Fay Lin, PhD (Senior Editor, GEN Biotechnology)  Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base:   The State of Omics 2024 Registration  GEN Summit   Cloaking Device: Liu, Joung Launch $100M Nvelop Therapeutics to Advance Delivery of Genetic Cargo By Alex Philippidis, GEN Edge, April 9, 2024  Bertozzi, Regev, and More Inspire During the Opening Plenary Session of AACR By Julianna LeMieux, PhD, GEN, April 8, 2024  AACR 2024: Aviv Regev Shows How Single-Cell Atlases Foster New Axis to Genentech’s Drug Discovery By Jonathan Grinstein, PhD, GEN, April 8, 2024  AACR 2024: A Video Update from San Diego By Julianna LeMieux, PhD, and Jonathan Grinstein, PhD, GEN, April 8, 2024  A Video Update from Day Two of the AACR Meeting By Julianna LeMieux, PhD, and Jonathan Grinstein, PhD, GEN, April 9, 2024  AACR 2024: EpiBiologics Advances Degraders of Membrane-Bound Proteins By Jonathan Grinstein, PhD, GEN, April 8, 2024  Celebrating National Robotics WeekBy Uduak Thomas, GEN, April 12, 2024

Episode 16 (April 5, 2024): This week, the GEN editors discussed deep proteome profiling of home-sampled dried blood spots to assess the effects of SARS-CoV-2 in mildly symptomatic or asymptomatic, rapid liquid nitrogen treatment for converting tumor cells into carriers for gene editing tools that target cancer in vivo, Verve Therapeutics’ pausing of a clinical trial due to a serious adverse event, Iambic Therapeutics’ advancement of its first AI-designed candidate into the clinic, and the launch of Diagonal Therapeutics to find agonist antibodies for heteromeric receptor complexes. Featuring Uduak Thomas (Senior Editor, GEN), Alex Philippidis (Senior Business Editor, GEN), Jonathan Grinstein, PhD, (Senior Editor, GEN), and moderated by Corinna Singleman, PhD, (Managing Editor, GEN and IPM) Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: The State of Omics 2024 Registration GEN Summit COVID-19 Infections Detected in Dried Blood Spots via At-Home Proteomic ProfilingBy GEN, April 2, 2024. CRISPR-Cas9 Targets Lung Cancer Using Cryo-Shocked Tumor CellsBy GEN, March 31, 2024. Iambic Rhythm: AI Drug Developer Enters the Clinic, Targeting HER2 CancersBy Alex Philipidis, GEN, April 3, 2024. Archimedes' Box: Diagonal Therapeutics Raises $128 Million to Discover Agonist AntibodiesBy Jonathan D. Grinstein, PhD, GEN Edge, April 3, 2024.

Episode 15 (March 29, 2024): From the expansion of Nvidia's generative artificial intelligence (AI) platform for drug discovery to AI notetakers that coach doctors to be more human, the GEN editors discuss the latest news in AI from two conferences in California: Nvidia GTC (San Jose) and Precision Med TRI-CON (San Diego). Featuring Alex Philippidis (Senior Business Editor, GEN), Fay Lin, PhD (Senior Editor, GEN Biotechnology), Jonathan D. Grinstein, PhD (Senior Editor, GEN), moderated by Corinna Singleman, PhD (Managing Editor, GEN and IPM). Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: GTC 2024: Nvidia Highlights AI ‘Revolution’ in Drug Discovery, GenomicsBy Alex Philippidis, GEN, March 19, 2024. GTC 2024: Nvidia’s Quantum Expansion Runs through the CloudBy Alex Philippidis, GEN, March 20, 2024. Machines Bring Efficiency…and Empathy? Eric Topol Talks AI in Precision MedicineBy Fay Lin, PhD, GEN Edge, March 28, 2024.