Nuevocor Secures FDA IND Approval for Gene Therapy in LMNA DCM

Nuevocor, a biotechnology firm headquartered in Singapore, has received clearance from the U.S. Food and Drug Administration (FDA) for its investigational new drug (IND) application concerning NVC-001, a novel gene therapy targeting LMNA-related dilated cardiomyopathy (LMNA DCM). This announcement was made on June 10, 2025, with plans to initiate a first-in-human Phase 1/2 ascending-dose clinical trial involving adult patients early next year.

The LMNA DCM condition affects approximately 100,000 individuals across the United States and Europe, marked by mutations in the LMNA gene, which are crucial for maintaining nuclear envelope integrity. These mutations can lead to severe cardiac issues, including heart muscle weakening and arrhythmias, potentially culminating in end-stage heart failure. NVC-001 aims to restore the integrity of the nuclear envelope, thereby reducing mechanical stress on heart cells.

"This IND clearance marks a significant milestone in our mission to develop transformative therapies for patients with genetic cardiomyopathies by leveraging unique insights from our proprietary PrOSIA mechanobiology platform," stated Dr. Yann Chong Tan, co-founder and CEO of Nuevocor. According to Dr. Tan, NVC-001 represents the first disease-modifying therapy specifically designed to address the mechanobiological root causes of LMNA DCM, as opposed to traditional gene replacement therapies.

The planned clinical trial will administer a one-time intravenous dose of NVC-001, monitored over a 52-week period. Previous preclinical studies have indicated that NVC-001 could improve survival and cardiac function, adding to the optimism surrounding its clinical potential. According to a recent report from Nuevocor, the company is also advancing other gene therapy candidates, including NVC-001B and NVC-002, which are currently in preclinical development.

In May 2025, Nuevocor raised $45 million in Series B financing from notable investors, including Kurma Partners and Angelini Ventures, to support this promising clinical trial. Elia Stupka, PhD, managing director at Angelini Ventures, remarked, "Our investment in Nuevocor marks a strategic expansion into cardiovascular disease and gene therapy. We are excited about Nuevocor's innovative approach to treating genetic dilated cardiomyopathy."

Nuevocor's endeavor is part of a broader trend in the biotechnology sector, where companies like Rocket Pharmaceuticals and Capricor Therapeutics are also exploring gene therapy solutions for cardiomyopathies. Rocket Pharmaceuticals is developing RP-A601, aimed at plakophilin-2 related Arrhythmogenic Cardiomyopathy, and Capricor Therapeutics is advancing deramiocel, intended for treating Duchenne muscular dystrophy cardiomyopathy.

As the field of gene therapy continues to evolve, the implications of Nuevocor's clinical trial could pave the way for new therapeutic options for patients facing genetic cardiomyopathies. The outcomes of this trial will be closely monitored, as they could significantly influence treatment paradigms and healthcare strategies for managing these debilitating conditions.

In conclusion, the FDA's IND approval represents a critical step forward for Nuevocor and the potential for transformative therapies in genetic cardiomyopathies. The success of NVC-001 could herald a new era in the treatment of LMNA DCM, offering hope to patients and their families.