Touching Base

Latest Episodes:

Nobel Prizes Announced and Breakthroughs for Huntington’s and Rare Diseases Oct 10, 2025

Show notes

Metal-organic frameworks and peripheral immune tolerance were the big winners of the Nobel prizes in chemistry, and in physiology or medicine, respectively. In this episode of the podcast, we discuss the winners and the impacts of their discoveries. Then we move over to some business news where we discuss a novel gene therapy for Huntington’s disease from uniQure that made waves recently. Early clinical trial data for AMT-130 showed that it could meaningfully slow the progression of the disease by as much as 75%. Also in business news, a new partnership involving Arbor Biotechnologies and Chiesi Group aims to develop gene editing therapies to target rare liver diseases.

Join GEN editors Corinna Singleman, PhD, Alex Philippidis, 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:

Brunkow, Ramsdell, Sakaguchi Win Nobel Prize in Physiology or Medicine for Peripheral Immune Tolerance

Uduak Thomas, GEN, October 6, 2025

Metal-Organic Frameworks Win the 2025 Nobel Prize in Chemistry

Julianna LeMieux, PhD, GEN, October 8, 2025

Gene Therapy Significantly Slows Huntington Disease Progression

GEN, September 24, 2025

StockWatch: uniQure Shares Reach Five-Year High on “Game Changing” Huntington’s Data

Alex Philippidis, GEN Edge, September 28, 2025

Chiesi, Arbor Target Rare Liver Diseases in Up-to-$2.1B Gene Editing Collaboration

Alex Philippidis, GEN Edge, October 8, 2025

Touching Base Podcast

Hosted by Corinna Singleman, PhD

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

BPI 2025 Debrief and Takeaways Sep 26, 2025

Show notes

GEN has been at the forefront of biotech and bioprocessing news for over 40 years. Last week, editor in chief, John Sterling and deputy editor in chief, Julianna LeMieux, PhD, attended BioProcess International (BPI). This annual meeting is a hallmark event for GEN, where we attend talks, speak with a multitude of bioprocess companies, and keep up to date on the latest products and news for the year. John and Julianna discuss their experience at this year’s event. Additionally, GEN held its first ever client appreciation reception this year at BPI and Julianna describes the atmosphere.

Join GEN editors John Sterling, Julianna LeMieux, PhD, and Corinna Singleman, 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:

Boston’s Bioprocessing Buzz: GEN Reports Live from BPI 2025

Julianna LeMieux, PhD and John Sterling, GEN, September 18, 2025

Top 10 U.S. Biopharma Clusters 2025

Alex Philippidis, GEN, August 1, 2025

Top 10 Contract Development and Manufacturing Organizations 2025

Alex Philippidis, GEN, September 15, 2025

Touching Base Podcast

Hosted by Corinna Singleman, PhD

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

Developing New Therapies Through Partnership, Electricity, and Immunotherapy Sep 05, 2025

Show notes

Novartis has agreed to license and develop Arrowhead Pharmaceuticals’ preclinical stage small interfering RNA (siRNA) therapy ARO-SNCA, a potential treatment for Parkinson’s disease and other synucleinopathies, plus additional targets. Two stories this week covered very different types of cell reprogramming techniques for therapy development. The first approach may overcome the time delays and safety risks of traditional immunotherapies, especially for patients with aggressive, late-stage disease. Meanwhile, electrical stimulation of macrophages could represent a new therapy to boost the body’s own repair processes in a range of injury and disease situations. Finally, Eli Lilly saw its shares climb 5% this past week after announcing that its history-making oral obesity candidate, orforglipron, aced the Phase III ATTAIN-2 trial.

Join GEN editors Corinna Singleman, PhD, Alex Philippidis, 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:

The State of Biotech Summit Registration

Novartis Commits Up to $2.2B toward Developing Arrowhead siRNA Therapy

Alex Philippidis, GEN Edge, September 2, 2025

Off-the-Shelf Immunotherapy Demonstrates Multipronged Attack Against Cancer

GEN, August 29, 2025

Human Macrophages “Reprogrammed” by Electrical Stimulation to Encourage Faster Healing

GEN, September 2, 2025

StockWatch: Analysts See $10B+ in Sales for Lilly Oral GLP-1

Alex Philippidis, GEN Edge, September 1, 2025

Touching Base Podcast

Hosted by Corinna Singleman, PhD

Behind the Breakthroughs

Hosted by Jonathan D. Grinstein, PhD

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

Stem Cells in Space and AI for Nanoparticles and Peptide Design Aug 22, 2025

Show notes

This week’s episode explores two frontiers: space travel and artificial intelligence. First, we discuss a study testing whether mouse sperm cryopreserved and stored in the International Space Station could still produce healthy offspring back on Earth. It’s a question with real implications for the future of deep space missions and even space tourism. Then, we turn to recent advances in AI. We talk about a machine-learning model trained to predict more effective nanoparticles for delivering RNA vaccines and therapies. Also, we learn about an AI model that designs peptides for challenging targets in cancer and neurodegenerative disease without needing detailed protein structures.

Join GEN editors Corinna Singleman, PhD, 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:

The State of Biotech Summit Registration

Space Station Stem Cells Successfully Produce Healthy Mice

GEN, August 18, 2025

COMET's Rocket Speed: AI-Designed Nanoparticles Accelerate mRNA Therapies

GEN, August 18, 2025

Protein Language Model Hits Undruggable Targets, No Structure Required

By Fay Lin, PhD GEN, August 14, 2025

Touching Base Podcast

Hosted by Corinna Singleman, PhD

Behind the Breakthroughs

Hosted by Jonathan D. Grinstein, PhD

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

Reversing Parkinson’s in Mice, Protecting Primates from HIV, Top 10 Biotech Clusters, and Sarepta Aug 08, 2025

Show notes

This episode of GEN's Touching Base spotlights the unexpected relationship between breathing low oxygen and restoration of neuron function in a mouse model of Parkinson’s disease. In addition, a single gene therapy injected at birth now offers lasting protection from HIV in infant rhesus macaques. Rounding out our episode, we discuss key factors shaping this year’s A-List of Top 10 U.S. Biopharma Clusters and recap the regulatory conflict surrounding Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy, Elevidys®—including the departure of Vinay Prasad, MD, the FDA official behind the agency’s brief get-tough approach.

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:

GLP-1 Drug Semaglutide Linked to Reduced Muscle Strength in Mice

GEN, August 6, 2025

Out of Thin Air: Hypoxia Reverses Parkinson’s Symptoms in Mice

By Fay Lin, PhD GEN, August 6, 2025

Early Gene Therapy Provides Lasting HIV Protection in Newborn Primates

GEN, July 30, 2025

StockWatch: As Prasad Exits FDA, Analysts See Benefit for Sarepta, CGT Stocks

By Alex Philippidis, GEN Edge, August 3, 2025

Sarepta to Resume Shipping DMD Gene Therapy to Ambulant Patients

By Alex Philippidis, GEN Edge, July 28, 2025

Sarepta Under Scrutiny, AstraZeneca’s Big Bet, and AI vs. IDRs

By Alex Philippidis, Uduak Thomas, Fay Lin, PhD, Corinna Singleman, PhD, GEN, July 25, 2025

About Face: Sarepta to Pause Elevidys Shipments Temporarily

By Alex Philippidis, GEN Edge, July 21, 2025

Touching Base Podcast

Hosted by Corinna Singleman, PhD

Behind the Breakthroughs

Hosted by Jonathan D. Grinstein, PhD

The State of Biotech Summit Registration

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Sarepta Under Scrutiny, AstraZeneca’s Big Bet, and AI vs. IDRs Jul 25, 2025

Show notes

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 Acquires Verona, Virtual Cell Challenge, Gene Therapy Restores Hearing Jul 11, 2025

Show notes

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

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

BIO 2025, Huge Virtual Cell Dataset, AI-Designed De Novo Proteins Jun 18, 2025

Show notes

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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Rocket’s Gene Therapy Trial Paused, Boltz-2 Open Source, Plasma Swap Rewinds Aging Jun 06, 2025

Show notes

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.

Customized CRISPR for KJ, 10x and Illumina Lawsuits, Regeneron Bags 23andMe May 22, 2025

Show notes

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