Touching Base

AcastBIO 2025, Huge Virtual Cell Dataset, AI-Designed De Novo ProteinsBIO 2025 is off and running. GEN editors discuss the size and scope of the bustling conference hall and share excitement about the week’s talks and panels. News from BIO includes the announcement of Lilly’s acquisition of Verve Therapeutics. Editors discuss the implications of industry-backed therapeutics amid cautious investors and public sentiment. In the realm of AI, Xaira Therapeutics, a $1 billion-backed AI drug discovery unicorn, has capped its first year by releasing the largest publicly available Perturb-seq dataset to support its virtual cell initiative. Additionally, AlphaDesign, a new AI-driven framework, accelerates the creation of functional de novo proteins, moving protein design toward custom therapeutics and precision medicine. Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Uduak Thomas, and Fay Lin, PhD, for a discussion of the latest biotech and biopharma news. Listed below are links to the GEN stories referenced in this episode of Touching BaseLilly to Acquire Verve Therapeutics for Up to $1.3BBy Alex Philippidis, GEN Edge, June 17, 2025 Second DMD Patient Dies After Treatment with Sarepta Gene TherapyBy Alex Philippidis, GEN Edge, June 15, 2025Danon Disease Patient Dies in Rocket Gene Therapy TrialBy Alex Philippidis, GEN Edge, May 27, 2025Xaira Therapeutics Releases Largest Perturb-Seq Dataset to Power the Virtual CellBy Fay Lin, GEN Edge, June17, 2025AI-Designed Proteins Created De Novo with New AlphaDesignGEN, June 17, 2025

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 

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 

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

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 

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

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