Touching Base

We are still talking about big pharma deals and biotech fundraising in this episode. The big news this week was Parabilis Medicines’s history-making IPO. We dive into the drug developer’s plans for the eye-popping $770.5 million that it raised. Next, we discuss the details of a collaboration between Merck and Protillion Biosciences to use artificial intelligence to discover multiple therapeutic candidates. Turning to some newly published research, we discuss the early results of a first-in-human clinical trial that is testing a dual vaccine against Lassa fever and rabies, a CRISPR system engineered to selectively trigger cancer cell death by chromatin shredding, and a novel mRNA delivery platform for delivering gene therapies starting with Duchenne muscular dystrophy. 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:  StockWatch: Parabilis Medicines Makes Wall Street History with $770.5M IPOBy Alex Philippidis, GEN Edge, June 14, 2026Merck, Protillion Launch AI Drug Discovery Collaboration with Up-to-$510M in Milestone PaymentsBy Alex Philippidis, GEN Edge, June 16, 2026First-in-Human Trial Reports Promising Dual Lassa–Rabies Vaccine DataGEN, June 9, 2026CRISPR Shreds Undruggable Cancer Cells with PrecisionBy Fay Lin, PhD, GEN Edge, June 8, 2026New mRNA Delivery Platform Restores Muscle Function in DMD ModelsGEN, June 11, 2026Touching 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.

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

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

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

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

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

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.