Sarepta has temporarily paused shipments of Elevidys, its FDA-approved treatment for Duchenne muscular dystrophy. In this episode of GEN's Touching Base, we discuss the company’s response to the tragedies associated with its DMD therapy as well as with a new therapy for limb-girdle muscular dystrophy. Also in this episode, big updates from AstraZeneca including a $50 billion investment in U.S. manufacturing and R&D, a heartwarming story about preventing mitochondrial disease involving eight babies from the U.K., and from the lab of Nobel Prize winner David Baker, PhD, AI that designs drugs for previously “undruggable” proteins.

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:

About Face: Sarepta to Pause Elevidys Shipments Temporarily

By Alex Philippidis, GEN Edge, July 21, 2025

StockWatch: Sarepta Shares Nosedive after LGMD Gene Therapy Patient Dies

By Alex Philippidis, GEN Edge, July 20, 2025

Sarepta Axes 500, 36% of Workforce, in Restructuring after DMD Patient Deaths

By Alex Philippidis, GEN Edge, July 16, 2025

AstraZeneca Commits $50B More to U.S. Manufacturing, R&D Projects

By Alex Philippidis, GEN Edge, July 22, 2025

AstraZeneca’s New $300M Plant Provides Control of Cell Therapy Production

By Gareth John Macdonald, GEN, May 14, 2025

Beyond Baby KJ: Next Steps in Manufacturing Genome Editing Cures

GEN Live, July 30, 2025

Mitochondrial Disease Milestone: Eight Babies Born Free of Disease via Pronuclear Transfer

By Julianna LeMieux, PhD GEN, July 16, 2025

Undruggable No More: AI Hits Disordered Proteins, Unlocks Therapy Targets

By Fay Lin, PhD GEN, July 18, 2025

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Merck & Co. has acquired Verona Pharma for $10 billion in a deal designed to expand the pharma giant’s portfolio of cardio-pulmonary disease treatments. In a step toward virtual cell benchmarking, the Arc Institute has launched the Virtual Cell Challenge to evaluate the ability of transcriptome AI models to generalize to new cell contexts for therapeutic applications. Fujifilm rebrands life science companies to position itself as a drug development leader. Additionally, AAV-OTOF gene therapy shows promise in restoring hearing loss while a new proteomics tool gleams insight into telomere repair. We wrap up our episode with highlights from BIO 2025, as FDA Commissioner Martin Makary, MD, shared his vision for a “New FDA” focused on accelerating drug applications, industry partnerships, and applying AI.

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:

Merck to Acquire Verona Pharma for $10B, Adding Fast-Growing COPD Drug

By Alex Philippidis, GEN Edge, July 9, 2025

Arc Institute Launces Virtual Cell Challenge to Accelerate AI Model Development

By Fay Lin, PhD, GEN Edge, June 26, 2025

End-to-End: Fujifilm Rebrands Life Sciences Companies, Positioning Itself as Drug Development Cycle Leader

By Alex Philippidis, GEN Edge, July 8, 2025

AAV-OTOF Gene Therapy Trial Restores Hearing in Both Children and Adults 

By GEN, July 7, 2025

Proteomics Tool Sheds Light on Cancer Cells’ Telomere Repair Tactics

By GEN, July 7, 2025

Makary Calls for “New FDA” at BIO; Defends Agency’s Spring Job Cuts

By Alex Philippidis, GEN Edge, June 18, 2025

Cell and Gene Therapy Leaders Tell FDA: “Believe in American Solutions”

By Kevin Davies, PhD, and Alex Philippidis, GEN Edge, June 5, 2025

Behind the Breakthroughs Podcast

Hosted by Jonathan D. Grinstein, PhD

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Acast

BIO 2025, Huge Virtual Cell Dataset, AI-Designed De Novo Proteins

BIO 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 Base

Lilly to Acquire Verve Therapeutics for Up to $1.3B

By Alex Philippidis, GEN Edge, June 17, 2025

Second DMD Patient Dies After Treatment with Sarepta Gene Therapy

By Alex Philippidis, GEN Edge, June 15, 2025

Danon Disease Patient Dies in Rocket Gene Therapy Trial

By Alex Philippidis, GEN Edge, May 27, 2025

Xaira Therapeutics Releases Largest Perturb-Seq Dataset to Power the Virtual Cell

By Fay Lin, GEN Edge, June17, 2025

AI-Designed Proteins Created De Novo with New AlphaDesign

GEN, June 17, 2025

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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 

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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 Base

ASGCT 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, 2025

10x 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 

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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 Chicago

By Julianna LeMieux, PhD and Damian Doherty, GEN, April 29, 2025

Hope and Headwinds at AACR in Chicago

By Damian Doherty, GEN, April 29, 2025

Senator Tammy Baldwin Supports Science in AACR Speech

By Julianna LeMieux, PhD, GEN, April 29, 2025

CRISPR-Edited TILs Fight Advanced Colorectal Cancer in Patients

By GEN, May 4, 2025

Engineered Extracellular Vesicles Could Deliver Gene Editors, Therapeutic Proteins to Cells

By GEN, April 30, 2025

SARS-CoV-2 Likely Spread Through Wildlife Trade, Not Bat Migration

By GEN, May 7, 2025

Chatting with Author David Quammen about SARS-CoV-2 Will Leave You “Breathless”

Originally aired: November 16, 2022

Recursion Halts Four Pipeline Programs, Sharpening Cancer, Rare Disease Focus

By Alex Philippidis, GEN Edge, May 5, 2025

StockWatch: Second Time’s the Charm for Abeona’s Gene Therapy

By Alex Philippidis, GEN Edge, May 4, 2025

Predict First: BMS Executives Discuss Company’s AI Approach

By Alex Philippidis, GEN Edge, March 26, 2025

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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 Editing

By Fay Lin, PhD, GEN, April 22, 2025

Multi-Platform Sequencing Study of Four Generations Sheds Light on Mutation Rates

GEN, April 23, 2025 

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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 Discovery

By Uduak Thomas, GEN, April 8, 2025 

Danaher-IGI Beacon for CRISPR Cures Celebrates One-Year Anniversary

By Fay Lin, PhD, GEN, March 28, 2025

Human Cell Maps Uncover Insights in Pediatric Bone Cancer

By Fay Lin, PhD, GEN, April 10, 2025

StockWatch: Biopharma Funds Tumble with Wall Street as Industry Spared from Worst of Tariffs—for Now

By Alex Philippidis, GEN Edge, April 6, 2025 

FDA, NIH, CDC Stagger as HHS Axe Falls, Eliminating 10,000 Jobs

By Alex Philippidis, GEN Edge, April 1, 2025 

Industry Voices Alarm as Peter Marks Departs FDA

By Kevin Davies, PhD, and Alex Philippidis, GEN Edge, March 30, 2025

The State of Multiomics & NGS 2025

Broadcast Date: Wednesday, April 23, 2025

Resurrection Genomics: A Conversation with Colossal’s Chief Biology Officer Eriona Hysolli

The State of Omics 2024, April 17, 2024

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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 Base

Gene 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 Therapy

By Alex Philippidis, GEN Edge, March 18, 2025 

PiggyBac Transposon System Creates Transgenic Cynomolgus Monkeys

By Julianna LeMieux, PhD, GEN, March 26, 2025 

From Tools to Trials: Editing Therapy Companies Pivot to Development

By 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 

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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 

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