Touching Base

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    Touching Base Podcast  Hosted by Corinna Singleman, PhD   Behind the Breakthroughs  Hosted by Jonathan D. Grinstein, PhD    

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:    Brunkow, Ramsdell, Sakaguchi Win Nobel Prize in Physiology or Medicine for Peripheral Immune Tolerance  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    Touching Base Podcast  Hosted by Corinna Singleman, PhD   Behind the Breakthroughs  Hosted by Jonathan D. Grinstein, PhD 

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 ToleranceUduak Thomas, GEN, October 6, 2025Metal-Organic Frameworks Win the 2025 Nobel Prize in ChemistryJulianna LeMieux, PhD, GEN, October 8, 2025Gene Therapy Significantly Slows Huntington Disease ProgressionGEN, September 24, 2025 StockWatch: uniQure Shares Reach Five-Year High on “Game Changing” Huntington’s DataAlex Philippidis, GEN Edge, September 28, 2025 Chiesi, Arbor Target Rare Liver Diseases in Up-to-$2.1B Gene Editing CollaborationAlex Philippidis, GEN Edge, October 8, 2025Touching Base PodcastHosted by Corinna Singleman, PhD

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 2025Julianna LeMieux, PhD and John Sterling, GEN, September 18, 2025Top 10 U.S. Biopharma Clusters 2025Alex Philippidis, GEN, August 1, 2025Top 10 Contract Development and Manufacturing Organizations 2025Alex Philippidis, GEN, September 15, 2025Touching Base PodcastHosted by Corinna Singleman, PhD

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 RegistrationNovartis Commits Up to $2.2B toward Developing Arrowhead siRNA TherapyAlex Philippidis, GEN Edge, September 2, 2025Off-the-Shelf Immunotherapy Demonstrates Multipronged Attack Against CancerGEN, August 29, 2025Human Macrophages “Reprogrammed” by Electrical Stimulation to Encourage Faster HealingGEN, September 2, 2025StockWatch: Analysts See $10B+ in Sales for Lilly Oral GLP-1Alex Philippidis, GEN Edge, September 1, 2025Touching Base PodcastHosted by Corinna Singleman, PhDBehind the BreakthroughsHosted by Jonathan D. Grinstein, PhD

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 

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 MiceGEN, August 6, 2025Out of Thin Air: Hypoxia Reverses Parkinson’s Symptoms in MiceBy Fay Lin, PhD GEN, August 6, 2025Early Gene Therapy Provides Lasting HIV Protection in Newborn PrimatesGEN, July 30, 2025StockWatch: As Prasad Exits FDA, Analysts See Benefit for Sarepta, CGT StocksBy Alex Philippidis, GEN Edge, August 3, 2025Sarepta to Resume Shipping DMD Gene Therapy to Ambulant PatientsBy Alex Philippidis, GEN Edge, July 28, 2025Sarepta Under Scrutiny, AstraZeneca’s Big Bet, and AI vs. IDRsBy Alex Philippidis, Uduak Thomas, Fay Lin, PhD, Corinna Singleman, PhD, GEN, July 25, 2025About Face: Sarepta to Pause Elevidys Shipments TemporarilyBy Alex Philippidis, GEN Edge, July 21, 2025Touching Base PodcastHosted by Corinna Singleman, PhDBehind the BreakthroughsHosted by Jonathan D. Grinstein, PhDThe State of Biotech Summit Registration

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 TemporarilyBy Alex Philippidis, GEN Edge, July 21, 2025StockWatch: Sarepta Shares Nosedive after LGMD Gene Therapy Patient DiesBy Alex Philippidis, GEN Edge, July 20, 2025Sarepta Axes 500, 36% of Workforce, in Restructuring after DMD Patient DeathsBy Alex Philippidis, GEN Edge, July 16, 2025AstraZeneca Commits $50B More to U.S. Manufacturing, R&D ProjectsBy Alex Philippidis, GEN Edge, July 22, 2025AstraZeneca’s New $300M Plant Provides Control of Cell Therapy ProductionBy Gareth John Macdonald, GEN, May 14, 2025 Beyond Baby KJ: Next Steps in Manufacturing Genome Editing CuresGEN Live, July 30, 2025Mitochondrial Disease Milestone: Eight Babies Born Free of Disease via Pronuclear TransferBy Julianna LeMieux, PhD GEN, July 16, 2025Undruggable No More: AI Hits Disordered Proteins, Unlocks Therapy TargetsBy Fay Lin, PhD GEN, July 18, 2025

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 DrugBy Alex Philippidis, GEN Edge, July 9, 2025 Arc Institute Launces Virtual Cell Challenge to Accelerate AI Model DevelopmentBy Fay Lin, PhD, GEN Edge, June 26, 2025End-to-End: Fujifilm Rebrands Life Sciences Companies, Positioning Itself as Drug Development Cycle LeaderBy Alex Philippidis, GEN Edge, July 8, 2025AAV-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 CutsBy Alex Philippidis, GEN Edge, June 18, 2025Cell 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 PodcastHosted by Jonathan D. Grinstein, PhD