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uniQure's “game changing” data announced in September, which showed significant slowing of Huntington’s disease (HD) progression in patients treated with its gene therapy candidate AMT-130, may not be enough to secure FDA approval. We also discuss Recursion's pivotal leadership transitions, as Najat Khan, PhD, chief R&D officer and chief commercial officer, is set to take over as the company's CEO effective January 1. The AI drug developer has made big bets filling the biology data gap and recently announced a "Google Map of the brain" to advance neurodegenerative disease targets. In open-source AI for drug discovery, the release of the latest Boltz model, BoltzGen, advances the platform from structural predictions to the design of "any" therapeutic modality, all available for commercial use.
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:
uniQure Staggers as FDA Questions Data for Huntington’s Gene Therapy Candidate
By Alex Philippidis, GEN Edge, November 3, 2025
StockWatch: uniQure Shares Reach Five-Year High on “Game Changing” Huntington’s Data
Alex Philippidis, GEN Edge, September 28, 2025
Gene Therapy Significantly Slows Huntington Disease Progression
GEN, September 24, 2025
Recursion, Roche Unveil Microglia Map of Neuro Disease Targets
By Alex Philippidis, GEN Edge, October 29, 2025
BoltzGen Democratizes AI Therapeutic Design, Expands Druggable Universe
By Fay Lin, PhD, GEN, October 27, 2025
The State of AI in Drug Discovery On Demand
Hosted by Corinna Singleman, PhD
Hosted by Jonathan D. Grinstein, PhD
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We are gearing up for our annual AI in Drug Discovery summit in a few days. So, for this episode, we focused on some recent AI news coverage. First, GEN senior editor Fay Lin, PhD, visited Seattle recently for some AI-focused meetings. She discusses her experiences in the Emerald City including an exclusive in-person interview with Nobel laureate, David Baker, PhD, on what’s real and what’s hype when it comes to using AI to design proteins de novo. Then we discuss new research from University of California, San Diego, that used machine learning to identify networks of genes that could be targeted to reprogram cancer stem cells. We also go over some early studies that point to a possible drug candidate for colon cancer.
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:
Uduak Thomas, GEN, October 6, 2025
AI in Protein Design: Hype vs. Reality Explained by David Baker
By Fay Lin, PhD, GEN, October 20, 2025
Nobel Anniversary: David Baker Keynotes STEM Fundraiser by ARCS Foundation Seattle
By Fay Lin, PhD, GEN, October 14, 2025
AI Treatment Reprograms and Triggers Cancer Stem Cells to Self-Destruct
GEN, October 20, 2025
The State of AI in Drug Discovery Registration
Hosted by Corinna Singleman, PhD
Hosted by Jonathan D. Grinstein, PhD
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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:
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
Hosted by Corinna Singleman, PhD
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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
Hosted by Corinna Singleman, PhD
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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
Hosted by Corinna Singleman, PhD
Hosted by Jonathan D. Grinstein, PhD
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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
Hosted by Corinna Singleman, PhD
Hosted by Jonathan D. Grinstein, PhD
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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
Hosted by Corinna Singleman, PhD
Hosted by Jonathan D. Grinstein, PhD
The State of Biotech Summit Registration
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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
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
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
By Kevin Davies, PhD, GEN, March 3, 2025
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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 Options
By 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
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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 Trials
By Alex Phillipidis, GEN, Jan 30, 2025.
Recursion Announces Promising Clinical Data on Lead AI-Based Drug Candidate for Brain Disease
By Fay Lin, PhD, GEN Edge, Feb 5, 2025.
AAV Delivered NanoCas CRISPR System Edits Muscle in Non-Human Primates
By GEN, Feb 3, 2025.
Next-Gen Cas12a System Enables Precise Single and Multiplexed Gene Editing in Cancer
By Corinna Singleman, PhD, GEN, Jan 30, 2025.
AgBio Companies Embrace Gene Editing for Stronger Food Future
By Uduak Thomas, GEN Feb issue, Feb 3, 2025.
Top 10 Takeover Targets of 2025
By Alex Phillippidis, GEN, Feb 3, 2025.
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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 Franklin
GEN Webinar presented by Matthew Cobb, PhD, Oct 16, 2020.
BBC film (1987).
Nobel Winner Svante Pӓӓbo Discovered the Neandertal in Our Genes
By 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 Milestones
GEN, Mar 7, 2024.
GEN 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 Approach
GEN, May 21, 2023.
AI Finds Autism-Causing Mutations in “Junk” DNA
GEN, May 28, 2024.
Pollution and Stress Alter Brain Development and Social Behavior in Male Mice
GEN, Aug 3, 2022.
“Mechanistic Basis of Resistance to PCBs in Atlantic Tomcod from the Hudson River”
Wirgin, et al., Science, Mar 11, 2011.
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