CGTLive's 2024 Pillars of Progress: Top News in Cardiology

For all of 2024, our team was following the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.

From major data publications and presentations to FDA decisions and medical meetings, the team spent all year bringing the latest information to the website's front page.

Among our areas of focus in 2024 has been cardiology. The major news items appeared among the top pieces our team produced—but sometimes smaller stories reach those heights as well because of their clinical impact, their inventive mechanisms, or otherwise. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in cardiology over the course of 2024.

Here, we'll highlight some of the most-read content on CGTLive's cardiology page this year. Click the buttons to read further into these stories.

First Patient in US Trial Receives Nexcella’s Light Chain Amyloidosis CAR-T NXC-201

“I am excited to initiate the only CAR-T clinical trial currently recruiting for AL Amyloidosis patients that have progressed on front-line daratumumab (Darzalex)-combination therapy. A 1-time therapy such as NXC-201 would provide an attractive option for AL Amyloidosis patients and clinicians. There are no approved drugs for relapsed/refractory AL Amyloidosis today.”
—Heather Landau, MD

In July, Immix Biopharma dosed the first patient in the phase 1b/2 NEXICART-2 clinical trial (NCT06097832), the first trial in the United States evaluating subsidiary Nexcella's NXC-201 (formerly referred to as HBI0101), an investigational autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets B-cell maturation antigen (BCMA), for the treatment of relapsed/refractory (r/r) light chain (AL) amyloidosis. The open-label, single-arm, multisite NEXICART-2 study will aim to enroll around 40 participants in total.

Intellia Successfully Redoses CRISPR Gene Editing Therapy

“Today’s data showcase an exciting new platform advancement for Intellia and the field of gene editing. For the first time ever, we demonstrated that redosing with CRISPR, utilizing our proprietary, nonviral LNP-based delivery platform, enabled an additive pharmacodynamic effect on the target protein."
—John Leonard, MD

Intellia Therapeutics’ CRISPR editing therapy NTLA-2001 demonstrated the ability to safely be redosed, according to data from a phase 1 trial (NCT04601051) evaluating the therapy in patients with transthyretin (ATTR) amyloidosis. Follow-on data from patients that received a supplemental dose of NTLA-2001 were presented at the 2024 Peripheral Nerve Society (PNS) Annual Meeting, held June 22-25, in Montreal, Canada, by Jorg Taubel, MD, FFPM, FESC, chief executive officer and founder, Richmond Pharmacology.

Sardocor’s Heart Failure Gene Therapy SRD-001 Garners Fast Track Designation as First Patients Are Dosed

“The enrollment of the first patients in this gene therapy trial will serve to validate the large body of work that has implicated deficiency of calcium cycling in HFpEF.”
—Roger Hajjar, MD

In February, Medera subsidiary Sardocor’s SRD-001, an investigational adeno-associated virus (AAV) vector-based gene therapy being evaluated for the treatment of Heart failure with preserved ejection fraction (HFpEF) in the phase 1/2a MUSIC-HFpEF clinical trial (NCT06061549), was granted fast track designation by the FDA. In addition, it was announced that the first 3 participants in the trial had been dosed with the gene therapy.

Intellia’s Phase 3 Clinical Trial for CRISPR-Based Gene Therapy Enrolls First Patient

“It is with great excitement that our center has enrolled the first US patient in the MAGNITUDE trial, a pivotal study assessing a new avenue of treatment for human diseases, specifically ATTR-CM. The MedStar Health Infiltrative Cardiomyopathy/Advanced Heart Failure Program is passionate about improving the lives of our patients in the region and beyond."
—Farooq Sheikh, MD

In May, Intellia Therapeutics and Regeneron enrolled the first patient in their phase 3 MAGNITUDE clinical trial (NCT06128629) for NTLA-2001, an investigational CRISPR/Cas9-based gene therapy intended to treat ATTR with cardiomyopathy (CM). In the multinational, double-blind, trial, participants will be randomly assigned to receive treatment with either NTLA-2001 or a placebo intravenous infusion consisting of normal saline.

Cardiac Function Preserved in Long-Term Follow-Up of Patients Treated With Duchenne Muscular Dystrophy Gene Therapy SGT-001

“This is the first study that describes longitudinal cMRI findings in DMD subjects that have received microdystrophin gene therapy. We observed that cardiac function was preserved as the individuals age with expected worsening of DMD-related cardiomyopathy. Further studies are needed to better understand the effects of the DMD gene therapy in the heart and carefully quantify the extent of cardiac gene transfer.”
—Stephanie Salabarria, BHSc, and colleagues

Patients treated with SGT-001, Solid Biosciences’ investigational microdystrophin gene therapy for Duchenne muscular dystrophy (DMD), in the phase 1/2 IGNITE-DMD study (NCT03368742) showed preservation of cardiac function as they aged. The long-term follow-up data was presented in a poster at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida.