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RBC Global Healthcare Conference: Ahead of an important trial readout next month, Jeff Stein explains Cidara's unique approach to influenza and how it differs from vaccines May 21, 2025

Show notes

He describes the science behind Cidara's Cloudbreak Drug-Fc conjugates, which might have uniform efficacy regardless of how the flu changes each season. Plus, a preview of next month's readout, and how FDA discussions around this have been.

Stevenage, UK based TessellateBio recently signed a collaboration and licensing deal with Boehringer Ingelheim targeting tumors dependent on alternative lengthening of telomeres (ALT) for their growth May 20, 2025

Show notes

CEO Andree Blaukat describes TessellateBio's focus on synthetic lethality and the science behind this lead area they have partnered with Boehring Ingelheim on, which seeks to interrupt some cancers' ability to harness telomeres to continuously replicate. The deal is potentially work in excess of €500 million. Plus addition programs, including one targeting SKP2.

Cambridge, UK based Maxion Therapeutics raised a $72M series A in March to advance its KnotBody programs - which combine the benefits of knottins and antibodies and can target ion channels and GPCRs May 16, 2025

Show notes

CEO Arndt Schottelius describes the rationale behind the KnotBody platform and the company's future plans to develop them against things like inflammatory diseases such as atopic dermatitis and inflammatory bowel disease.

ASGCT 2025: HuidaGene presented preclinical and early clinical data for its CRISPR Cas12 based program for DMD in a Presidential Symposium talk May 16, 2025

Show notes

CEO Alvin Luk and CTO TJ Cradick explain how the company's Cas12 and 13 programs are different than Cas9, and they discuss the DMD presentation and how this program differs from the current microdystrophin product that is on the market. Plus, RNA editing programs targeting neovascular age-related macular degeneration and MECP2 Duplication Syndrome (MDS), and a DNA program targeting ALS.

ASGCT 2025: Dyno Therapeutics CEO Eric Kelsic discusses the latest in capsids for CNS, ophthalmology, and neuromuscular conditions May 15, 2025

Show notes

He talks about new capsids Dyno announced at this years' conference and the science behind getting them to specific places in the body. Plus, the company's new 'Frontiers Program' and a Genetic Agency Technology Conference coming up in November.

ASGCT 2025: Physicians and scientists raced to make a bespoke gene editing therapy for an infant who urgently needed one in months - a first hand account, and what this means for drug development May 15, 2025

Show notes

As published in NEJM today, UPenn's Kiran Musunuru describes how the team used base editing in a matter of months to treat an infant with CPS1 deficiently, and his thoughts on what the learnings of this case are for drug development.

ASGCT 2025: Coave Therapeutics presented a late breaker showing a positive profile for its cerebrospinal fluid administered ligand-engineered AAV2-based capsid May 15, 2025

Show notes

CEO Rodolphe Clerval walks us through Coave's "ALIGATER' ligand technology and describes the importance of the cerebrospinal administered program. Plus, comments on the company's IV program for CNS conditions, and a recent €32 million series A.

Cambridge, UK based Alchemab Therapeutics last week signed a second collaboration with Eli Lilly worth up to $415M to license its IND ready program for ALS and other neurodegenerative diseases May 15, 2025

Show notes

CEO Jane Osbourn describes Alchemab's platform, which looks for people who are resilient to disease and then sequences them to understand why, and discusses this week's deal and the company's prior one with Lilly as well.

ASGCT 2025: The Co-Founder of ProQR discusses the company's Axiomer RNA base editing technology - which will see a CTA filed this quarter and data by the end of the year May 15, 2025

Show notes

ProQR Co-Founder & CSO Gerard Platenburg describes the company's RNA editing platform and the journey he has seen this field go through over time. Plus, updates on programs for cholestatic diseases, Rhett Syndrome, and MASH.

ASGCT 2025: Capsida Biotherapeutics is using its expertise in capsid design to develop gene therapies that are tissue specific, and in the case of the CNS, can be delivered via IV and crosses the BBB May 14, 2025

Show notes

CEO Peter Anastasiou describes the importance of capsid design and how Caspida is developing its own gene therapies for CNS conditions. A first program recently had its IND cleared.

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