Adeno-associated virus based cardiomyopathy gene therapies (Nuevocor)

A Singapore-based startup raised a $45 million Series B to start testing a gene therapy in humans next year for a genetic heart muscle disorder, signaling that the AAV-based gene therapy field still has some bright spots. Nuevocor disclosed the funding on Tuesday morning. Investors include Kurma Partners, Angelini Ventures, EDBI, ClavystBio and Boehringer Ingelheim Venture Fund, among others. The money arrives as companies ranging from biotech startups to large drugmakers have deserted the AAV landscape. Most recently, Vertex ended all work on AAVs. But others are still attempting to advance the viral vectors for one-and-done treatments, including a brain cancer startup that turned to crowdfunding to secure money. Nuevocor got started with a $24 million Series A in 2021, based on work out of Singapore’s Agency for Science, Technology and Research. The company aims to reduce the force on the nuclear envelope since that damage can lead to cell death and other impairments in genetic cardiomyopathies, CEO Yann Chong Tan told Endpoints News . Other gene therapy companies like Lexeo Therapeutics are also doing work in cardiomyopathy, but they use gene replacement approaches. That’s not a “viable” approach for many genetic cardiomyopathies, Tan said, noting Nuevocor is working on “targeting the root cause of disease.” The lead program is an AAV-based gene therapy dubbed NVC-001 for LMNA-related dilated cardiomyopathy. About 100,000 people have the rare disease in the US and Europe, and they can progress from mild heart failure to end-stage heart failure in about five years, Tan said. A Phase 1/2 study in the US and Europe is set to begin early next year. Tan said accelerated approval is a possibility after the Phase 1/2, but the company needs to speak with regulators to see if that is a path it can take. He said “hundreds of families” have already shown interest in the trial, given the severity of the genetic disease and the lack of treatment options. With less than 30 employees today, Nuevocor plans to boost its team and expand into Europe, with a Paris office. It also has a presence in the Philadelphia area. It’s one of several Singapore biotechs raising money and trying to forge a global network, Tan said. Earlier this year, Seattle and Singapore biotech Callio Therapeutics nabbed a $187 million Series A and Leyden Labs bought a small Singapore startup called CoV Biotechnology. Meanwhile, venture firm and biotech creator Flagship Pioneering also established a partnership in the region last fall.