aav-gene-therapy
(4)
Nuevocor
Nuevocor is a Singapore-founded, clinical-stage biotechnology company developing genetic medicines for cardiomyopathies driven by aberrant mechanobiology - the mechanical stresses that damage heart-muscle cells. Spun out of Singapore's A*STAR research agency in 2021, the company built a proprietary discovery engine called PrOSIA (Protein Organ Specific Interactome Analysis) to map the functional root causes of genetic heart disease. Its lead program, NVC-001, is an AAV-based gene therapy for LMNA-related dilated cardiomyopathy, an aggressive inherited heart condition with no targeted treatment. Nuevocor received FDA IND clearance for NVC-001 in June 2025 and has raised roughly US$80.6 million to date, including a US$45 million Series B in May 2025.
Form Bio
Form Bio is an Austin-based computational life sciences company that builds AI-driven software to speed up the design, characterization, and manufacturing of genetic medicines, especially adeno-associated virus (AAV) cell and gene therapies. Spun out of Colossal Biosciences in 2022 with a $30M Series A, its platform lets scientists model billions of therapeutic vector designs in silico to improve efficacy, safety, and manufacturability before ever entering the lab, targeting the manufacturing bottleneck that drives the majority of FDA rejections in the field.
NeuShen Therapeutics
NeuShen Therapeutics is a clinical-stage biotech developing novel small-molecule and gene-therapy treatments for central nervous system disorders such as schizophrenia, depression and epilepsy. Founded in 2021 by Joan Huaqiong Shen, MD, PhD, and headquartered in Shanghai with an R&D center in the Boston area, the company runs a pipeline led by NS-136, a selective M4 receptor positive allosteric modulator for schizophrenia, and NS-041, a highly selective KCNQ2/3 activator for epilepsy and depression.
Sangamo Therapeutics, Inc.
Sangamo Therapeutics is a clinical-stage genomic medicine company in Brisbane, California that engineers zinc finger proteins to switch genes on, off, or rewrite them. After three decades pioneering zinc finger technology, it is racing to bring its lead gene therapy ST-920 for Fabry disease to market while building a neurology pipeline aimed at crossing the blood-brain barrier - all under the pressure of a 2026 search for strategic alternatives.