Around the Helix: Cell and Gene Therapy Company Updates – April 23, 2025

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. Exploring the ARCUS Approach to Next-Gen Gene Editing for Muscular Dystrophy

Precision Biosciences presented preclinical data related to its gene editing approach, ARCUS, in the context of Duchenne muscular dystrophy (DMD) at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas. During the conference, CGTLive®'s sister site NeurologyLive^® sat down with Cassandra Gorsuch, PhD, chief scientific officer at Precision, to discuss the mechanism behind this novel approach, and the promising early findings.

2. Cartesian Therapeutics’ mRNA CAR-T Descartes-08 Shows Efficacy and Safety in Myasthenia Gravis in Phase 2 Study

Cartesian Therapeutics’ Descartes-08, an investigational autologous mRNA-engineered chimeric antigen receptor T-cell therapy (CAR-T) therapy that targets B-cell maturation antigen (BCMA), has demonstrated therapeutic effects in a phase 2b double-blind, placebo-controlled, crossover trial (NCT04146051) in myasthenia gravis (MG). Sustained responses over a 12-month period were seen in treated patients, along with a safety profile that was aligned with previously reported data.

3. uniQure's Huntington Disease Gene Therapy AMT-130 Snags FDA Breakthrough Therapy Designation

uniQure's AMT-130, an adeno-associated virus (AAV) vector-based gene therapy currently being evaluated for the treatment of Huntington disease in 2 phase 1/2 clinical trials (NCT04120493 and NCT05243017), has been granted breakthrough therapy designation by the FDA. Notably, AMT-130 has previously been granted regenerative medicine advanced therapy, orphan drug, and fast track designations by the agency.

4. Questions of Access: Gene Therapy’s Next Chapter in β-Thalassemia

In the time since the FDA's approvals of bluebird bio’s betibeglogene autotemcel (beti-cel; marketed as Zynteglo) and Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel; marketed as Casgevy) for transfusion-dependent β-thalassemia (TDT), the 2 gene therapy products have begun to be rolled out into commercial use. This process has been slow-going, however, and to date the gene therapy products still have availability limited to certain centers of excellence. CGTLive® reached out to several experts to get their insight on the clinical experience of patients and treating physicians with the gene therapy products thus far.

5. BrainChild Bio's Pediatric Brain Tumor CAR-T BCB-276 Reels in Breakthrough Therapy Designation

BrainChild Bio's BCB-276, an investigational CAR-T therapy that targets B7-H3 and is being developed for the treatment of a type of pediatric brain tumor referred to as diffuse intrinsic ponJne glioma (DIPG), has received breakthrough therapy designation from the FDA. The therapy is currently being evaluated in the phase 1 BrainChild-03 clinical trial (NCT04185038), data from which supported the FDA's decision.

6. Jorna Therapeutics and Ono Pharmaceutical Teaming Up to Tackle RNA Editing Therapy Development

The 2 companies have launched an agreement, originally signed in December 2024, that will utilize a proprietary RNA editing platform developed by Jorna for therapeutic discovery activities. Under the agreement, Ono may choose to develop and commercialize candidates based on the findings of the platform, which incorporates AI technology.