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  • Editors’ Choice: Top Stories of 2025
    2025/12/18
    In the final episode of 2025, the GEN team came together to reflect on some of the stories over the last year that they each found impactful. Some of the stories were covered in GEN’s December issue, but our editors took the time to discuss some topics in a bit more depth during the podcast. The discussion began with an update on the progress of AI use in drug discovery. Cell and gene therapy was a big topic with the positive outcome for Baby KJ, while Sarepta dealt with a rollercoaster of a year with their DMD treatment, Elevidys. Research and biotech in Africa are growing and the discussion ranged from challenges to promise in research and development on the continent. This year’s NIH budget cuts and impacted institutions rounded out the conversation.Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, Phd, Kevin Davies, Phd, John Sterling, 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: GEN Editors Reflect on Six of the Biggest Stories of the YearBy Alex Philippidis, Savannah Wiegel, Fay Lin, PhD, Kevin Davies, PhD, Uduak Thomas, and Julianna LeMieux, PhD, GEN, December 4, 2025StockWatch: Sarepta Says Deaths of Two Elevidys Patients Posted on FDA Database Unrelated to Treatment By Alex Philippidis, GEN Edge, August 10 StockWatch: As Prasad Exits FDA, Analysts See Benefit for Sarepta, CGT Stocks By Alex Philippidis, GEN Edge, August 3 FDA Probes Death of Brazilian Boy Linked to Sarepta’s Elevidys GEN Edge, July 27 Second DMD Patient Dies After Treatment with Sarepta Gene Therapy By Alex Philippidis, GEN Edge, June 15 DMD Patient Dies After Treatment with Sarepta Gene Therapy By Alex Philippidis, GEN Edge, March 18From The Editor in ChiefJohn Sterling, December Issue of GEN Gene Therapy in AfricaThe State of CRISPR & Genome Editing Summit, June 11, 2025TOP 50 NIH By Alex Philippidis, GEN, December 4, 2025 FDA Cell & Gene Therapy roundtable: Cell and Gene Therapy Leaders Tell FDA: “Believe in American Solutions” By Alex Philippidis, and Kevin Davies, PhD, GEN Edge, June 5, 2025Scientists in NYC Rally to Defend and Stand Up for ScienceBy Corinna Singleman, GEN, March 10, 2025Touching 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.
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    41 分
  • Lung Organoids, Autism and Sarcopenia Insights in Mice, and a Bidding War Ends
    2025/12/04

    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, 2025


    Autism-Linked Behaviors in Mice Reduced by Nutrient Trio

    GEN, December 3, 2025


    Muscle Stem Cells Bolstered by ECM Protein Tenascin-C

    GEN, December 5, 2025

    Protego Closes $130M Series B to Fund Pivotal Trial of AL Amyloidosis Candidate

    By Alex Philippidis, GEN Edge, December 3, 2025

    Lundbeck Ends Bidding War for Avadel, After Alkermes’ Up-to-$2.37B Offer

    By Alex Philippidis, GEN Edge, December 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.

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    36 分
  • New Nasal Vaccines, Korro Stock Dips, J&J and Merck Acquisitions, and Dyno GATC
    2025/11/20

    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, 2025


    Novel 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, 2025


    J&J Expands Cancer Pipeline with $3B Halda Acquisition

    By Alex Philippidis, GENEdge, November 18, 2025


    Merck to Acquire Cidara for $9.2B, Bolstering Antiviral Pipeline

    By Alex Philippidis, GEN, November 15, 2025

    AI Meets Genetic Agency at Dyno GATC 2025

    By Fay Lin, PhD, GENEdge, November 14, 2025


    Genetic Agency on Display at GATC 2025

    By Kevin Davies, PhD, and Fay Lin, PhD, GEN, November 13, 2025


    Dyno GATC Announces AI Agents, Muscle Capsid, and Manufacturing Partner

    By 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

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

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    42 分
  • uniQure Staggers at FDA, Recursion's Microglia Map, and Leadership Transitions
    2025/11/07

    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.

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    32 分
  • Artificial Intelligence in the Emerald City and Targeting Colon Cancer Stem Cells 
    2025/10/24

    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

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

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    24 分
  • Nobel Prizes Announced and Breakthroughs for Huntington’s and Rare Diseases
    2025/10/10

    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.

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    21 分
  • BPI 2025 Debrief and Takeaways
    2025/09/26

    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.

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    28 分
  • Developing New Therapies Through Partnership, Electricity, and Immunotherapy
    2025/09/05

    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.

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    28 分