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.

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

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

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

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