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 Phase III trial of the CRISPR-based therapy experienced non-lethal liver toxicity. In other news, a therapeutic plasma exchange regimen combined with intravenous immunoglobulin reduced some biomarkers of biological age in humans. Sanofi has agreed to acquire Blueprint Medicines for up to $9.5 billion, and a universal CAR T cell therapy was produced by applying CRISPR gene editing to cells donated by healthy individuals. 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 Danon Disease Patient Dies in Rocket Gene Therapy Trial By Alex Philippidis, GEN, May 27, 2025 Boltz-2 Released to Democratize AI Molecular Modeling for Drug Discovery By Fay Lin, PhD, GENEdge, June 6, 2025 StockWatch: Intellia Stumbles on News of Patient's Severe Liver Toxicity By Alex Philippidis, GEN, May 30, 2025 The State of CRISPR Summit Registration https://bit.ly/CRISPR25 Plasma Exchange in Humans Reduces Biological Age by Over Two Years GEN, June 1, 2025 Sanofi to Acquire Blueprint Medicines for Up to $9.5B By Alex Philippidis, GEN Edge, June 2, 2025 "Universal" CAR-T Immunotherapy Targets Relapsed/Refractory Blood Cancers GEN, May 30, 2025 Hosted on Acast. See acast.com/privacy for more information.

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 human cells. On the business front, 10x Genomics settles with Bruker and Vizgen while Illumina sues Element Biosciences for patent infringement. Also, trouble for Prime Medicine as the company pivots its pipeline and downsizes, and after months of speculation about its fate, 23andMe is acquired by Regeneron.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 BaseASGCT 2025: World's First Patient Treated with Personalized CRISPR Therapy By Fay Lin, PhD, GEN, May 15, 2025 EvoCAST Harnesses CRISPR-Linked Bacterial Transposases to Insert Genes Into Human Cells GEN, May 18, 2025 eePASSIGE Engineers Gene-Sized Edits in Human Cells GEN, June 10, 2024 Illumina Sues Element Biosciences, Alleging Infringement of Flow Cell, Imaging Patents By Alex Philippidis, GEN Edge, May 18, 202510x Settles Bruker, Vizgen Patent Lawsuits By Julianna LeMieux, PhD, and Alex Philippidis, GEN, May 18, 2025 Prime Medicine Chops 25% of Workforce, Pivots Pipeline as CEO Quits By Alex Philippidis, GEN Edge, May 19, 2025 Regeneron to Acquire 23andMe with Winning $256M Bid GEN, May 19, 2025 Hosted on Acast. See acast.com/privacy for more information.

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 infiltrating lymphocytes and a method for delivering protein therapies and gene editors using engineered vesicles. Lastly, in business news, Recursion rethinks its pipeline, the FDA gives Abeona's gene therapy a chance, and Bristol Myers Squibb execs open up on artificial intelligence.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 Base:AACR 2025: A Video Update from ChicagoBy Julianna LeMieux, PhD and Damian Doherty, GEN, April 29, 2025Hope and Headwinds at AACR in ChicagoBy Damian Doherty, GEN, April 29, 2025Senator Tammy Baldwin Supports Science in AACR SpeechBy Julianna LeMieux, PhD, GEN, April 29, 2025CRISPR-Edited TILs Fight Advanced Colorectal Cancer in PatientsBy GEN, May 4, 2025Engineered Extracellular Vesicles Could Deliver Gene Editors, Therapeutic Proteins to CellsBy GEN, April 30, 2025SARS-CoV-2 Likely Spread Through Wildlife Trade, Not Bat MigrationBy GEN, May 7, 2025Chatting with Author David Quammen about SARS-CoV-2 Will Leave You “Breathless”Originally aired: November 16, 2022Recursion Halts Four Pipeline Programs, Sharpening Cancer, Rare Disease FocusBy Alex Philippidis, GEN Edge, May 5, 2025StockWatch: Second Time's the Charm for Abeona's Gene TherapyBy Alex Philippidis, GEN Edge, May 4, 2025Predict First: BMS Executives Discuss Company's AI ApproachBy Alex Philippidis, GEN Edge, March 26, 2025 Hosted on Acast. See acast.com/privacy for more information.

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 come since the discovery of the structure of DNA and the completion of the Human Genome Project. We also talk about today's DNA-related advances that use machine learning to design tailored Cas9 proteins and multiple sequencing technologies to study mutation rates in four generations of the same family. 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:After Job Cuts, “We're Entering a Very New Territory for Biotech. By Alex Philippidis, GEN Edge, April 17, 2025 Roche Commits $50B to U.S. Manufacturing, R&D as Tariffs Loom By Alex Philippidis, GEN Edge, April 22, 2025 StockWatch: Investors Hungry for Lilly after Diabetes Pill Aces Phase III Trial By Alex Philippidis, GEN Edge, April 20, 2025 Machine Learning Engineers Bespoke Cas9 Enzymes for Gene EditingBy Fay Lin, PhD, GEN, April 22, 2025Multi-Platform Sequencing Study of Four Generations Sheds Light on Mutation RatesGEN, April 23, 2025 Hosted on Acast. See acast.com/privacy for more information.

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 layoffs and leadership departures at the FDA, CDC, and NIH. We also share news from recent scientific conferences and meetings that we've attended as well as an interesting study that used human cell maps to shed new light on pediatric bone cancer.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.Plex Research Partners with Ginkgo Bioworks to Apply AI-Based Search Engine to Drug DiscoveryBy Uduak Thomas, GEN, April 8, 2025 Danaher-IGI Beacon for CRISPR Cures Celebrates One-Year AnniversaryBy Fay Lin, PhD, GEN, March 28, 2025Human Cell Maps Uncover Insights in Pediatric Bone Cancer By Fay Lin, PhD, GEN, April 10, 2025StockWatch: Biopharma Funds Tumble with Wall Street as Industry Spared from Worst of Tariffs—for NowBy Alex Philippidis, GEN Edge, April 6, 2025 FDA, NIH, CDC Stagger as HHS Axe Falls, Eliminating 10,000 JobsBy Alex Philippidis, GEN Edge, April 1, 2025 Industry Voices Alarm as Peter Marks Departs FDABy Kevin Davies, PhD, and Alex Philippidis, GEN Edge, March 30, 2025The State of Multiomics & NGS 2025Broadcast Date: Wednesday, April 23, 2025 Resurrection Genomics: A Conversation with Colossal's Chief Biology Officer Eriona HysolliThe State of Omics 2024, April 17, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 Hosted on Acast. See acast.com/privacy for more information.

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 Hosted on Acast. See acast.com/privacy for more information.

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 Hosted on Acast. See acast.com/privacy for more information.

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. Hosted on Acast. See acast.com/privacy for more information.

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. Hosted on Acast. See acast.com/privacy for more information.

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 Hosted on Acast. See acast.com/privacy for more information.

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 Hosted on Acast. See acast.com/privacy for more information.

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. Hosted on Acast. See acast.com/privacy for more information.

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. Hosted on Acast. See acast.com/privacy for more information.

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 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: Nanoparticle Uptake Screen Informs Nanomedicine Design By GEN, March 19, 2024. mRNA Therapeutics May Act Longer If Given Multiple TailsBy GEN, March 22, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 stories, media, and other items discussed in this episode of Touching Base: The State of Omics 2024 Registration GEN Summit NanoString Agrees to be Acquired by Investment Firm for $220MBy GEN, March 11, 2024. Curio Begins Testing Commercial Spatial Barcoding Technology for Single Cell SequencingBy GEN, March 12, 2024 Cellares Unveils Clinically-Compliant Cell Therapy Manufacturing PlatformBy Jonathan D. Grinstein, PhD, GEN, March 12, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 Boston area, and how organoids derived from amniotic fluid show promise for understanding fetal diseases. Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: At Lost Women of Science, the Unsung Are CelebratedBy Julianna LeMieux, GEN, March 7, 2024. To Mark International Women's Day, C-Suite Women Share Leadership InsightsBy GEN, March 7, 2024. Lipid Nanoparticles Engineered to Target Lung Cells Reduce Tumor Size in MiceBy GEN, March 4, 2024. Novel Thio-lipids Developed Capable of Reaching Eyes and Lungs in AnimalsBy GEN, March 5, 2024. Novo Nordisk Expands Boston Area Research and Early DevelopmentBy Alex Philippidis, GEN, March 2, 2024.Metal-Organic Nanoparticles Enable Better Vaccine Delivery, Stronger Immune ResponseBy GEN, March 6, 2024. Organoids Originating from Cells in Amniotic Fluid Could Illiminate Congential ConditionsBy GEN, March 5, 2024. Hosted on Acast. See acast.com/privacy for more information.

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 payloads. Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: Rare Disease Day: Moderna Gets Back to Its RootsBy Jonathan D. Grinstein, GEN, February 28, 2024 Taming the Immune System When Rare Diseases StrikeBy Kathy Liszewski, GEN, February 27, 2024 Form Bio's AI Ensures Gene Therapies Pass Muster, with Ginkgo's HelpGEN, February 28, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 genome. Astellas Pharma and Kelonia announced a nearly $800 million research collaboration and license agreement to develop innovative universal, off-the-shelf in vivo CAR T cell therapies. Fourth-quarter earnings and revenue fell short of expectations as Biogen worked to eliminate 1,000 jobs (11.5% of its workforce), among other cost-cutting actions, and restructured its pipeline after a slow start. Plus, an interview with Andrea Choe, founder and chief executive officer at Holoclara. Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: Generation of Human Isogenic Induced Pluripotent Stem Cell Lines with CRISPR Prime EditingThe CRISPR Journal, February 14, 2024 Addressing Scientific Misconduct: An Interview with Elisabeth BikBy Uduak Thomas, GEN Biotechnology, February 15, 2024 StockWatch: Biogen's Q4 Results Disappoint AnalystsBy Alex Philippidis, GEN Edge, February 18, 2024 Astellas and Kelonia Partner for $800M In Vivo CAR T Cell TherapiesBy Jonathan D. Grinstein, PhD, GEN Edge, February 20, 2024 PRINT: Precise RNA-Mediated Insertion of TransgenesGEN, February 21, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 “inappropriate” relationships with two employees. Julianna LeMieux, PhD, Deputy Editor in Chief at GEN, provides a recap of her time at AGBT 2024.Plus, an interview with Christian Henry, president and chief executive officer at PacBio. Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: CRISPRed Pigs: Precision Porcine Gene Editing Combats PPRS Virus ThreatBy Uduak Thomas, GEN, February 14, 2024 Exscientia Fires CEO for “Inappropriate” Relationships with Two EmployeesGEN, February 13, 2024 Sun, Spatial, and Sequencing: A Report from the First Day of AGBTBy Julianna LeMieux, PhD, and Jonathan D. Grinstein, PhD, GEN, February 7, 2024NanoString CSO Joe Beechem Insists the Company “Not Going Anywhere”By Julianna LeMieux, PhD, GEN, February 8, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 for bankruptcy, blaming the $31 million jury award assessed against it last November in a patent infringement case filed by rival 10x Genomics. Plus, an interview with Simon Barnett, research director at Dimension. Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: Gene Therapy for Hereditary Angioedema Shows Success in Patients · GEN, February 5, 2024 Ultragenyx's Gene Therapy Ameliorates Pediatric Neurodegenerative Disorder · By Jonathan Grinstein, GEN Edge, February 7, 2024 CRISPR-Repaired T Cells May Treat Fatal Inflammatory Diseases · GEN, February 5, 2024 NanoString Files for Chapter 11 Bankruptcy, Launches Strategic Review · By Alex Philippidis, GEN, February 5, 2024 Novo Holdings Buys Catalent for $16.5B; Sells Three Sites to Novo Nordisk for $11B · By Alex Philippidis, GEN, February 5, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 Touching Base:Human Longevity Spin-Out Aims to Tap into Variant Interpretation MarketBy Uduak Thomas, GEN, January 31st, 2024Light My Fire: COUR Raises $105M Series A to Advance Tolerogenic Nanoparticles for AutoimmunityBy Jonathan Grinstein, GEN Edge, January 30th, 2024 Gene Replacement Therapy Halts Fatal Arrhythmogenic Condition in MiceGEN, January 30th, 2024 StockWatch: IPO Revival? CG Oncology, ArriVent Raise a Combined $555MBy Alex Philippidis, GEN Edge, January 28th, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 of Touching Base: Gene Therapy Restores Hearing in 11-Year-Old After Just One MonthGEN, January 24th, 2024 GSK and Elegen Team Up to Develop DNA-Based Medicines and VaccinesBy Jonathan Grinstein, GEN Edge, January 24th, 2024Blood Brothers: Kyverna Reinvents Autologous CAR T Manufacturing from Blood Draw By Jonathan Grinstein, GEN Edge, January 24th, 2024 StockWatch: Nvidia Jumps 24% on Recursion, Amgen AI Partnerships By Alex Philippidis, GEN Edge, January 21st, 2024 Five Deaf Children Have Hearing Restored by AAV-Based Gene TherapyGEN, January 25th, 2024 Hosted on Acast. See acast.com/privacy for more information.

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 RNAsGEN, January 18, 2024.· Rhesus Monkey Cloned via Somatic Cell Nuclear TransferGEN, January 16, 2024.· StockWatch: M&A Talk Fuels Cytokinetics Stock Roller CoasterBy Alex Philippidis, GEN Edge, January 16th, 2024. Hosted on Acast. See acast.com/privacy for more information.

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 By Alex Philippidis, GEN, January 10, 2024. JPM: Prime Medicine eyes 2024 IND for first prime editing therapy By Alex Philippidis, GEN, January 9, 2024 JPM 2024: Veracyte's C2i genomics acquisition adds AI readouts to liquid biopsy testing By Jonathan D. Grinstein, GEN, January 8, 2024 JPM: More M&A “firepower” in store for 2024, EY predicts By Alex Philippidis, GEN, January 8, 2024 Hosted on Acast. See acast.com/privacy for more information.

Episode 3 (January 5, 2024): Interview with Co-founder and CEO of Moonwalk Biosciences and former CTO of Illumina, Alex Aravanis; Neuro gene therapy with Novartis and Voyager; GEN reporters look forward to the 2024 J.P. Morgan Healthcare Conference Listed below are key references to the GEN stories, media and other items discussed in this episode of Touching Base:Novartis, Voyager Ink Up-to-$1.3B Expansion of Neuro Gene Therapy CollaborationBy Alex Philippidis, GEN, January 2, 2024.Moonwalk Biosciences Raises $57M to Edit the EpigenomeBy Uduak Thomas, GEN, January 4, 2024. Hosted on Acast. See acast.com/privacy for more information.

Episode 2 (December 22, 2023): Interview with President and CEO of Tome Biosciences, Rahul Kakkar; intelligent lab partner using LLMs; AI for antimicrobials; mRNA vaccine production in Africa; Moderna's positive cancer vaccine data.Listed below are key references to the GEN stories, media and other items discussed in this episode of Touching Base:The Next Chapter in Genome Editing? Tome Emerges from Stealth with $213M By Alex Philippidis, GEN, December 19, 2023.Artificial Intelligence-Based Lab Partner Designs, Runs Experiments in the Lab GEN, December 20, 2023.BioNTech Advances Plans for mRNA Vaccine Production in Africa by 2025 GEN, December 19, 2023.Explainable AI Directs Small Molecule Structural Class Drug Discovery By Jonathan Grinstein, GEN, December 20, 2023.StockWatch: Positive Cancer Vaccine Data Wows Moderna Investors By Alex Philippidis, GEN Edge, December 18, 2023. Hosted on Acast. See acast.com/privacy for more information.

Episode 1 (Week ending December 8, 2023): Nevan Krogan (UCSF) mapping autism pathways; Nvidia's latest AI partnership; CAR-T regulations; epigenome editing; mRNA therapeutics.References to the GEN stories, media and other items discussed in this episode of Touching Base:· Autism's Molecular Mechanisms Uncovered through Protein-Protein Interaction NetworkBy Julianna Lemieux, GEN, December 5, 2023.· Rhapsody in B: Tune Therapeutics Presents Data for Hepatitis B Epigenetic SilencerBy Jonathan Grinstein, GEN Edge, December 6, 2023.· Nvidia Looks to Genentech for its Next Leap in AI Drug DiscoveryBy Alex Philippidis, GEN Edge, November 28, 2023.· StockWatch: CAR-T Shares Mostly Flat as FDA Probes Risk of New CancersBy Alex Philippidis, GEN, December 1, 2023· Perfect Pitch: Genome Editing Pioneer Fyodor Urnov on Commercializing CRISPR Therapies and Epigenomic Tuning {VIDEO}GEN Edge, August 30, 2023· Press release: John Crowley appointed President & CEO of BIOBiotechnology Innovation Organization· BIO CEO Rachel King Outlines Industry's Challenges on “Close to the Edge”GEN multimedia, April 26, 2023 · The First CRISPR Drug: Vertex Pharmaceuticals' Casgevy Wins U.K. Approval for Sickle Cell DiseaseGEN, November 16, 2023 Hosted on Acast. See acast.com/privacy for more information.