Nuevocor Snags IND Clearance for Gene Therapy Trial in LMNA DCM
In light of the IND clearance, the company intends to go forward with plans for a first-in-human phase 1/2 ascending-dose clinical trial.
Singapore-based company Nuevocor has received clearance from the FDA for an investigational new drug (IND) application for NVC-001, an adeno-associated virus (AAV) vector-based gene therapy intended to treat LMNA-related dilated cardiomyopathy (LMNA DCM).1
In light of the IND clearance, the company intends to go forward with plans for a first-in-human phase 1/2 ascending-dose clinical trial. Nuevocor expects to launch the 52-week trial, which will be open to adult patients, early next year. Participants in the trial will receive a 1-time dose of the gene therapy product via intravenous infusion.
LMNA DCM, which is found in approximately 100,000 people across the United States and Europe, is characterized by nuclear envelope disruption caused by mutations in LMNA, a gene necessary for nuclear envelope integrity. The condition results in heart muscle weakening, arrhythmias, and eventually end-stage heart failure.
NVC-001 is intended to provide restoration of nuclear envelope integrity by decreasing aberrant mechanical stress on nuclei. It is based on Nuevocor’s PrOSIA mechanobiology platform, a proprietary platform that is intended to address issues in disease pathways shared across more than one cardiomyopathy rather than attempting to tackle individual gene mutations as in traditional gene replacement therapy approaches. Notably, the gene therapy showed the ability to effect benefits in areas including survival and cardiac function in preclinical research.
"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," Yann Chong Tan, PhD, the cofounder and chief executive officer of Nuevocor, said in a statement.1 "NVC-001 is the first disease-modifying therapy for LMNA DCM designed to address the underlying mechanobiological root cause of disease. We look forward to initiating our clinical trial to bring this potentially life-changing therapy to patients."
According to Nuevocor’s pipeline page on its website, NVC-001 is the company’s program that has reached the furthest stage in its development, but it has several other pathway-specific genetic medicines in preclinical development.2 These include NVC-001B and NVC-002, the indications for which remain undisclosed. The company also states that it has other programs in the discovery stage of development.
Notably, in May 2025, Nuevocor announced that it had received $45 million USD in Series B financing from a group of investors that includes Kurma Partners and Angelini Ventures, among others, to support the development of NVC-001.3 At the time it was noted that the funding would help enable a planned phase 1/2 clinical trial with clinical sites in the United States and Europe. The company also stated at the time that it plans to open a new office in Paris, France.
"Our investment in Nuevocor marks a strategic expansion for Angelini Ventures – into cardiovascular disease, gene therapy, and the Singapore biotech ecosystem,” Elia Stupka, PhD, the managing director at Angelini Ventures, said in a May 2025 statement.3 “We are excited by Nuevocor's novel approach to treating genetic-dilated cardiomyopathy by targeting mechanobiological pathways rather than simply replacing genes. This one-time therapy has the potential to transform patient outcomes and ease healthcare burdens. We are proud to support their mission and join the Board, alongside Kurma, ClavystBio, EDBI, Boehringer Ingelheim Venture Fund, and other investors, as the company grows globally, including into Europe.”
Nuevocor is one of a number of companies seeking to apply gene therapy approaches to cardiomyopathies. Several others include Rocket Pharmaceuticals, which is developing RP-A601, an investigational AAV vector-based gene therapy intended to treat plakophilin-2 related Arrhythmogenic Cardiomyopathy, and Capricor Therapeutics, which is developing deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended to treat Duchenne muscular dystrophy cardiomyopathy.4,5
