viral-vectors
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Carbon Biosciences
Carbon Biosciences is a Waltham, Massachusetts preclinical genetic-medicine company building gene therapies on non-AAV parvovirus vectors. Founded by Longwood Fund with gene-therapy pioneers John Engelhardt and Robert Kotin, it uses a proprietary vector engine (PAVE) to design capsids from the broad parvovirus family - including human bocavirus - that carry larger genetic payloads, target specific tissues, and provoke little pre-existing immunity, opening the door to re-dosing. Its lead program targets cystic fibrosis by delivering the full-length CFTR gene to the lung, with a second vector aimed at cardiac disease.
KaliVir Immunotherapeutics
KaliVir Immunotherapeutics is a clinical-stage biotech developing next-generation oncolytic viral immunotherapies for cancer. Its proprietary Vaccinia Enhanced Template (VET) platform engineers vaccinia viruses that can be delivered intravenously, replicate selectively in tumors, and express therapeutic payloads that reshape the tumor microenvironment. Founded in 2019 and headquartered in Pittsburgh, the company advances an internal lead candidate, VET3-TGI, alongside partnered programs licensed to Astellas Pharma and Roche.
GEMMA Biotherapeutics
GEMMA Biotherapeutics (GEMMABio) is a Philadelphia-based gene therapy company spun out of the University of Pennsylvania and founded by AAV pioneer James M. Wilson. Launched in October 2024 with $34 million in seed funding, GEMMABio builds the research, manufacturing, and product-development infrastructure to move adeno-associated virus (AAV) gene therapies from the bench to the bedside faster and more affordably - with a focus on rare and ultra-orphan inherited diseases and a stated commitment to global patient access.
克睿基因 Cure Genetics
克睿基因 Cure Genetics is a clinical-stage biotech founded in Suzhou in 2016 that develops cell and gene therapies for solid tumors and genetic diseases. It runs two proprietary platforms: AIMS, an allogeneic CAR-NKT cell therapy approach aimed at hard-to-treat solid tumors, and VELP, a directed-evolution AAV vector platform that builds high-quality viral libraries for tissue-targeted gene delivery to the nervous system, heart, and kidney. Its lead programs include CGC729 (anti-CD70 CAR-NKT for renal cell carcinoma) and CGC-602 (a PD-1-IL-2v bispecific antibody).