Touching Base

A gene therapy clinical trial for Danon disease run by Rocket Pharmaceuticals has been suspended following the death of a patient. MIT and Recursion have released Boltz-2, an open-source tool that predicts molecular binding affinity at newfound speed and accuracy, aiming to democratize commercial drug discovery. Intellia Therapeutics saw its stock nosedive late last week after a patient in its Pha...

A baby, named KJ, has become the first patient to be treated with a personalized CRISPR therapy to address a severe metabolic disorder. We discuss this story—one of the biggest science/medical stories from ASGCT 2025—and talk about the family and scientists at its center. In other news, laboratory-evolved CRISPR-associated bacterial transposases are being used to insert healthy genes into hum...

The exact origins of SARS-CoV-2 are still something of a mystery, but new research may bring scientists one step closer to an explanation for how the virus reached Wuhan. Also in the episode, we dive into GEN’s coverage of the annual AACR meeting including how the community is navigating massive research budget cuts. Then we discuss a potential colorectal cancer therapy using CRISPR-edited tumor i...

The seismic changes made by the current administration in the United States continue to impact the scientific community. The business news segment of this week’s episode covers the effects of job cuts on biotech, Roche’s manufacturing and R&D plans amid tariff threats, and shares an update on Eli Lilly’s diabetes pill. Also, in honor of DNA Day, we reminisce about how far the field has co...

Are Dire Wolves really back? We give our take on Colossal Biosciences’ latest announcement about the de-extinction of Dire Wolves including some thoughts on their approach and some of the broader implications of their work. In other stories, we discuss the early impact of the Trump administration’s tariffs on biotech as well as the growing alarm in the scientific community in the wake of sweeping...

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...

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 location...

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,...

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.    &nbs...

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...

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 L...

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 i...

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...

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...

Episode 14 (March 22, 2024):  This week, the GEN editors cover identifying receptors and proteins for cellular uptake and intracellular processing of nanoparticles, and a new mRNA structure by adding multiple “tails” to the molecules that boosted mRNA activity levels in cells by 5 to 20 times.    Featuring Jonathan D. Grinstein, PhD, (Senior Editor, GEN ), Uduak Thomas (Senior Edito...

  Episode 13 (March 15, 2024): This week, the GEN editors discussed business news from NanoString, Curio, and Cellares.   Featuring Alex Philippidis (Senior Business Editor, GEN ), Uduak Thomas (Senior 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 s...

Episode 12 (March 8, 2024): Featuring Julianna LeMieux (Deputy Editor-in-Chief, GEN), Uduak Thomas (Senior Editor, GEN), and Alex Philippidis (Senior Business Editor, GEN), moderated by Fay Lin (Senior Editor, GEN Biotechnology)   This week, the GEN editors cover a recent interview with the Lost Women of Science, advances in delivery with lipid nanoparticles, Novo Nordisk’s expansion in the B...

Episode 11 (March 1, 2024): After being diverted by the SARS-CoV-2 pandemic to develop and manufacture COVID-19 vaccines, Moderna gets back to advancing rare disease mRNA therapeutics; a look at rare autoimmune diseases that fly below the public radar; Form Bio announced a partnership with Ginkgo Bioworks to bring their AI out of the computer and into gene therapy production by optimizing genetic...

Episode 10 (February 23, 2024): Researchers from Francis Collins’ lab developed an efficient CRISPR prime editing protocol to generate isogenic-induced pluripotent stem cell (iPSC) lines carrying heterozygous or homozygous alleles for putatively causal single nucleotide variants. An RNA-based system known as PRINT can create and introduce site-specific, safe, and stable transgenes into the human g...

Episode 9 (February 16, 2024): Using CRISPR-Cas9 technology, British animal genetics firm Genus has generated a population of pigs that are resistant to the deadly porcine reproductive and respiratory syndrome (PRRS) virus. Exscientia has fired its founder, CEO, and principal executive officer Andrew Hopkins, PhD, and removed him from the company’s board, after it concluded that he engaged in “ina...

Episode 8 (February 9th, 2024): Intellia and Ultragenyx gave updates on their clinical trials for gene therapies for inherited rare diseases. Novo Holdings, which manages the assets of the foundation that controls Novo Nordisk, agreed to buy Catalent for $16.5 billion to keep up with the high demand for its hit drugs Wegovy and Ozempic. Spatial biology tools developer NanoString Technologies filed...

Episode 7 (February 2, 2024): Simplify Genomics launches genome interpretation platform, COUR’s nanoparticles for autoimmunity, gene replacement therapy for heart condition, CG Oncology and ArriVent IPOs; an interview with Nautilus Biotechnology co-founders, Sujal Patel and Parag Mallick. Listed below are key references to the GEN stories, media and other items discussed in this episode of Touchin...

Episode 6 (January 26, 2024): 11-year-old regains hearing after gene therapy; Kyverna uses blood draw instead of apheresis for CAR T manufacturing; DNA production technology with GSK and Elegen, StockWatch with Nvidia and Recursion; an interview with renowned cardiologist, Eric Topol, MD   Listed below are key references to the GEN stories, media and other items discussed in this episode...

Episode 5 (January 19, 2024); RNAs to address Alzheimer’s disease; monkey cloned via somatic cell nuclear transfer; Cytokinetics stock roller coaster; an interview with founder and CEO of 1910 Genetics, Jen Nwankwo, PhD. Listed below are key references to the GEN stories, media and other items discussed in this episode of Touching Base : ·  Alzheimer’s Disease Linked to Toxic Short RNAs GEN ,...

Episode 4 (January 12, 2024): Takeaways from the J.P. Morgan Health Care conference: Illumina, Danaher, 10x, Empress Therapeutics; an interview with Liberate Bio’s Shawn Davis and Walter Strapps.  Listed below are references to relevant GEN stories discussed in this episode of Touching Base :  JPM: Illumina’s new CEO spells out 2024 priorities, presents preliminary Q4 2023 results  ...