CRISPRMED25 Virtual Event Roundup

The CRISPR Medicine Conference 2025 virtual oral session titled "Transformative Gene Editing: From Rare Disease Models to Clinical Applications" delivered a diverse showcase of gene-editing technologies advancing treatment for devastating diseases. From pre-clinical studies in foetal genome editing to clinical-stage programmes, the session highlighted progress across multiple therapeutic areas.

In utero base editing for infantile Krabbe disease

Asma Naseem, who is a Senior Research Fellow at UCL Great Ormond Street Institute of Child Health, shared promising research on a foetal gene-editing approach for infantile Krabbe disease, a rare and fatal neurological disorder. She presented a prenatal in vivo gene-editing platform that aims to correct GALC mutations within the central nervous system, peripheral nervous system and hematopoietic system.

Using virus-like particles to deliver adenine base editors, she and colleagues have achieved base editing rates of 60-90% in pre-clinical models, with evidence that the base-editing reagents cross the foetal blood-brain barrier. Naseem emphasised that in utero intervention via intravenous injection offers several critical advantages, including early therapeutic administration before disease onset, improved accessibility to brain and haematopoietic progenitors, increased immune tolerance to the editing reagents, and reduced dosing compared to postnatal treatment approaches.

Gene-editing therapies as a new standard of care for dyslipidemia

Maria Mirotsou, Vice President of Discovery Biology at Scribe Therapeutics, highlighted the company’s proprietary CRISPR-based technology for targeting key drivers of dyslipidemia. She explained how Scribe’s CRISPR by Design™ approach allows them to iteratively engineer bacterial immune systems, including their proprietary compact CRISPR-CasX, to develop powerful genome and epigenome-editing tools with high activity, specificity, and deliverability.

Mirotsou presented exciting pre-clinical data on two of the company’s lead programmes—STX-1200 and STX-1400—which are designed to safely and effectively lower Lp(a) and triglyceride levels, respectively. Their pipeline comprehensively targets the key drivers of dyslipidemia: LDL-C, Lp(a), and triglycerides, offering a holistic approach to treating cardiometabolic diseases with the aim of establishing a new standard of care for patients suffering from these conditions.

In vivo CRISPR-based therapy for primary hyperoxaluria type 1

YolTech Therapeutics' Chief Technology Officer Emma Wang discussed the company's clinical-stage in vivo gene-editing programme targeting primary hyperoxaluria type 1 (PH1). She presented interim clinical data from the ongoing YOLT-203 dose escalation trial, emphasising the encouraging safety profile, pharmacodynamic responses, and efficacy results observed so far.

YOLT-203 is the first in vivo gene-editing therapy to show positive clinical data for PH1. YOLT-203 received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA in September 2024. You can read more about the proof-of-concept study for YOLT-203 in our recent interview with Emma Wang and YolTech Co-founder and CEO Yuxuan Wu.

Wang also highlighted YolTech's advances in enzyme discovery and engineering, along with its proprietary lipid nanoparticle (LNP) delivery systems that effectively target both hepatic and extrahepatic tissues.

Non-viral gene insertion for precise DSB-free genome engineering

We also heard from Blair Madison, Vice President of Research at Poseida Therapeutics, who presented the company’s advanced non-viral gene insertion platform for treating genetic disorders. Madison detailed how their proprietary Super piggyBac (SPB) technology enables stable gene integration at TTAA sites without inducing double-strand (DSB) breaks, offering advantages over AAV-based therapies including larger cargo capacity and reduced immunogenicity.

Pre-clinical data for haemophilia A revealed that the company’s LNP-SPB system achieved stable factor VIII expression across different age groups, with therapeutic effects sustained for more than one year. The platform incorporates a safety switch enabling on-demand regulation of transgene expression, and Madison highlighted recent engineering advances that improved transgene expression more than 10-fold in mice. He also introduced their site-specific SPB transposon technology, which achieves >99% on-target fidelity for more precise therapeutic applications.

Off-the-shelf CAR-T cell therapy using CRISPR hybrid RNA-DNA technology

The session concluded with a presentation from Tina Albertson, Chief Medical Officer for Caribou Biosciences, who shared data from the Phase 1 ANTLER trial for Caribou's off-the-shelf CAR-T cell therapy CB-010. Using proprietary CRISPR hybrid RNA-DNA (chRDNA) technology, CB-010 incorporates three strategic genome edits: knockout of the TRAC gene to reduce the risk of graft-versus-host disease (GvHD), insertion of a CD19-specific CAR, and knockout of PD-1 to prevent CAR-T cell exhaustion. In patients with relapsed or refractory B-cell non-Hodgkin lymphoma, CB-010 demonstrated a 76.1% overall response rate with no GvHD observed.

Albertson highlighted that those patients receiving CB-010 from donors with higher HLA matching showed improved outcomes. A key advantage of this allogeneic approach is rapid treatment initiation, with therapy beginning just days after a patient is deemed eligible.

Interactive poster session

Following the oral presentations, virtual CRISPRMED25 attendees from all over the world engaged in an interactive virtual poster session featuring additional innovations within gene-editing. Eleven posters highlighted advances in off-target assessment methods, novel delivery approaches, and next-generation editing templates. The poster topics ranged from innovative DNA modification technologies to simplified methods for creating transgenic models and fast quantification techniques to assess gene-editing outcomes.

In summary, this virtual session gave us a taste of the remarkable pace of innovation within CRISPR Medicine. For those who missed today's live presentations or would like to watch them again, the recording will soon be available on demand on our website.