Data Roundup: April 2025 Features Updates for Cardiomyopathy Gene Therapy, Myasthenia Gravis CAR-T, and More

Last month, April 2025, the CGTLive® team was diligently tracking the latest data readouts and published literature on cell and gene therapies within oncology, neurology, rare diseases, and more.

As more and more innovative therapies enter the clinical trial field, more data is accrued every month, buoying excitement in the field and sometimes making or breaking the fates of small biotech companies. Last month delivered data updates from studies for Nanoscope Therapeutics’ retinitis pigmentosa (RP) gene therapy MCO-010, Lexeo Therapeutics’ Friedreich ataxia (FA) cardiomyopathy gene therapy LX2006, and more. Our team has highlighted these below.

Click the read more buttons for more details and information about each update.

Nanoscope Therapeutics’ Gene Therapy MCO-010 Restores Vision in Patients With Retinitis Pigmentosa

April 4, 2025 - Nanoscope Therapeutics’ MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy, showed the ability to restore vision in patients with RP in clinical data published Molecular Therapy.

In the paper, which is entitled “A synthetic opsin restores vision in patients with severe retinal degeneration," MCO-010 was described. A synthetic opsin with broad spectral sensitivity administered via intravitreal injection, Synthopsin-MCO-010 includes a promoter to specifically target ON-bipolar cells. In preclinical research, Synthopsin-MCO-010 demonstrated the ability to restore the visual behavior in RP mouse models, according to the authors. In the in-human study, the synthopsin was packaged into an optimised AAV2 gene therapy vector targeted at human retinal bipolar cells.

Four patients with RP with ABCA4 variants were enrolled in the study. The group included 3 male patients and 1 female patient, and the average age of the participants was 45.5 years. According to the authors, the participants had severe vision loss prior to treatment and were “unable to move around without the help of another person." Furthermore, all of the participants were considered legally blind and had hand-motion to light-perception vision.

Lexeo’s Gene Therapy LX2006 Reduces Abnormal LVMI in Patients With Friedreich Ataxia Cardiomyopathy

April 10, 2025 - Lexeo Therapeutics’ LX2006, an adeno-associated virus (AAV) vector-based gene therapy, has demonstrated the ability to reduce left ventricular mass index (LVMI) in patients with FA cardiomyopathy who had a normal LVMI at baseline.

The data come from patients treated across 2 studies: the Lexeo phase 1/2 SUNRISE-FA clinical trial (NCT05445323) and the phase 1a Weill Cornell Medicine investigator-initiated trial (NCT05302271). For the 6 patients across these studies who had an abnormal LVMI as baseline, 5 of the patients showed an improvement of more than 10% by 12 months posttreatment. In addition, at their most recent follow-up visit, 5 of the 6 patients have shown an LVMI within the normal range, as of a March 25, 2025 data cutoff. Lexeo reported that the mean improvement in LVMI for the 6 patients was 27% as of the latest follow-up visit and that a mean improvement of 25% in LVMI was seen by the 12-month posttreatment visit or sooner. Furthermore, it was noted that for the patients treated in the middle and high dose level cohorts, greater improvement was seen at earlier time points in comparison to patients who received the low dose, indicating a dose-dependent response. For the 6 patients in the studies who had a normal LVMI at baseline, most showed stabilization or improvement of LVMI over time.

Lexeo also reported that among 12 patients treated across both studies, 10 showed a decrease in lateral wall thickness at their latest visit and 11 showed more a greater than 25% decrease in high-sensitivity troponin I levels at their lates visit. In addition, improvements in functional measures, such as the modified Friedreich Ataxia Rating Scale and the Kansas City Cardiomyopathy Questionnaire, were recorded in the majority of patients.

Verve Therapeutics’ Base Editing Therapy VERVE-102 Reduces LDL-C in Patients With HeFH and CAD

April 15, 2025 - Verve Therapeutics’ VERVE-102, an investigational in vivo base editing therapy that uses a lipid nanoparticle (LNP) delivery system, has demonstrated the ability to reduce low-density lipoprotein cholesterol (LDL-C) and PCSK9 protein levels in patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) being treated in the phase 1b HEART-2 clinical trial (NCT06164730).

As of the March 13, 2025, data cut-off, 14 patients had been treated across 3 cohorts in HEART-2 and reached at least 28 days of follow-up. For the 4 patients treated in the 0.3 mg/kg cohort, LDL-C levels were decreased by 21% and PCSK9 levels were decreased by 46%. For the 6 patients treated at a dose of 0.45 mg/kg, LDL-C levels were decreased by 41% and PCSK9 levels were decreased by 53%. For the 4 patients treated in the 0.6 mg/kg cohort, LDL-C levels decreased by 53% and PCSK9 levels decreased by 60%. Verve noted that a patient treated at the 0.6 mg/kg dose showed the maximum decrease in LDL-C levels: 69%.

Verve also reported the results in terms of total RNA dose. From day 28 through the last available follow-up, a time-averaged percent change for mean LDL-C decreases from baseline was reported. For the 4 patients who received less than 25 mg total RAN dose, a –21% mean LDL-C reduction from baseline was observed; for the 7 patients who received a 25 mg to less than 50 mg total RNA dose, a –41% mean LDL-C reduction from baseline was observed; and for the 3 patients who received 50 mg to less than 60 mg total RNA dose, a –59% mean LDL-C reduction from baseline was observed.

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

April 23, 2025 - Cartesian Therapeutics’ Descartes-08, an investigational autologous mRNA-engineered 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.

In the trial, heavily pretreated, highly symptomatic patients with MG (n = 36) were randomly assigned in a 1:1 fashion to undergo a 12-month treatment phase with either Descartes-08 or a placebo. Treatment with the CAR-T therapy, in the 12 patients who had available data, led to reductions of 5.5 (±1.1) and 4.8 (±1.4) in Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores at 4 months and 12 months posttreatment, respectively. Furthermore, an average Quantitative Myasthenia Gravis Score (QMG) reduction of 4.8 (±1.7) points at 4 months posttreatment, which deepened through 12 months posttreatment (6.0 [±2.1]), was seen in these patients.

"This impressive data highlights the potential of Descartes-08 to serve as an important therapeutic option to deliver deep and sustained reductions in MG-ADL for patients with myasthenia gravis," trial investigator Tuan Vu, MD, a professor of neurology at the University of South Florida Morsani College of Medicine, and the director for Neuromuscular Medicine and EMG, said in a statement.¹ "The data in participants who had not received prior biologic therapy is particularly striking as this population is most comparable to the patient populations in trials of standard-of-care biologics."