Touching Base

Stories on lung organoids and autism were some of the most read this week. We kicked things off by digging into recent breakthroughs in both areas including an automated method for manufacturing iPSC-derived lung organoids and how a combination of supplements reduced autism-linked behaviors in mice. Still on the topic of new research, we discussed how a protein in the extracellular matrix helps muscle stem cells repair aging muscles. In business news, the bidding war between Lundbeck and Alkermes over Avadel Pharmaceuticals has ended. We talk about the possible implications of Lundbeck’s decision to back out and next steps for Alkermes. Then we dive into Protego Biopharma’s recent fundraising round, and efforts to develop small-molecule pharmacological chaperones that modulate protein stability in disease.   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:  Lung Organoids Generated in Bulk Using Stirred Bioreactor Technology GEN, December 1, 2025Autism-Linked Behaviors in Mice Reduced by Nutrient TrioGEN, December 3, 2025Muscle Stem Cells Bolstered by ECM Protein Tenascin-CGEN, December 5, 2025 Protego Closes $130M Series B to Fund Pivotal Trial of AL Amyloidosis CandidateBy Alex Philippidis, GEN Edge, December 3, 2025Lundbeck Ends Bidding War for Avadel, After Alkermes’ Up-to-$2.37B OfferBy Alex Philippidis, GEN Edge, December 1, 2025Touching Base Podcast Hosted by Corinna Singleman, PhD  Behind the Breakthroughs Hosted by Jonathan D. Grinstein, PhD 

Nasal vaccines show new promise for human papillomavirus (HPV) to address cervical cancer and whooping cough. In business news, Korro's stock craters 81% as AATD interim Phase I/II results miss expectations. Additionally, Johnson & Johnson acquires Halda to expand their cancer pipeline while Merck acquires Cidara to boost antivirals. Lastly, the inaugural Genetic Agency Technology Conference (GATC) hosted by Dyno Therapeutics brought together a diverse group of researchers, entrepreneurs, and patient advocates to discuss the mission of genetic agency, or an individual’s ability to take action at the genetic level to live a healthier life.  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:  Cervical Cancer May Be Treatable with Intranasal Nanogel Vaccine GEN, November 15, 2025Novel Nasal Spray Vaccine for Pertussis Shows Promise By Corinna Singleman, PhD, GEN, November 12, 2025 StockWatch: Korro Craters 81% on Data; Patient Death Sours Analysts on Intellia By Alex Philippidis, GENEdge, November 16, 2025J&J Expands Cancer Pipeline with $3B Halda Acquisition By Alex Philippidis, GENEdge, November 18, 2025Merck to Acquire Cidara for $9.2B, Bolstering Antiviral Pipeline By Alex Philippidis, GEN, November 15, 2025 AI Meets Genetic Agency at Dyno GATC 2025By Fay Lin, PhD, GENEdge, November 14, 2025Genetic Agency on Display at GATC 2025By Kevin Davies, PhD, and Fay Lin, PhD, GEN, November 13, 2025Dyno GATC Announces AI Agents, Muscle Capsid, and Manufacturing PartnerBy Fay Lin, PhD, GEN Edge, November 11, 2025 Touching Base Podcast Hosted by Corinna Singleman, PhD  Behind the Breakthroughs Hosted by Jonathan D. Grinstein, PhD

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 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