Gene Therapy Startups

Kopra Bio develops cancer immunotherapy technology that converts cancer cells into biofactories to produce their own destruction. The company operates in the biotech and cancer treatment space.

10 investors

ParcelBio develops APEXm, a proprietary mRNA platform that enhances protein expression for longer-lasting therapeutic effect compared to conventional mRNA technologies. The platform targets autoimmune disease, oncology, and therapeutic protein replacement applications.

10 investors

Nephrogen develops AI-powered AAV delivery vehicles for genetic medicines targeting kidney and pancreatic diseases. The company uses high-throughput in vivo screening to design tissue-specific viral vectors that are significantly more efficient and less immunogenic than existing alternatives.

8 investors

FinalDose develops a programmable DNA-targeting molecule designed to recognize and destroy cells carrying specific genetic sequences, with initial applications in oncology across cancer types.

7 investors

Uncommon Therapeutics uses advanced technologies to develop drugs targeting rare genetic diseases, starting with Rett Syndrome, a neurodevelopmental disorder affecting approximately 1 in 10,000 girls. The company combines disease expertise with efficient drug development to advance therapeutics from discovery to clinical application.

7 investors

64x Bio develops cell-line engineering and production technologies to overcome manufacturing bottlenecks in advanced and next-generation therapies. It serves biopharma and therapeutics developers by combining large-scale parallelized screening with computational analysis to engineer high-yield cell lines.

6 investors

Rejuvenation Technologies develops mRNA therapeutics designed to extend healthspan by lengthening short telomeres, with preclinical results in fibrotic liver and lung disease. The company is moving toward clinical trials via IND-enabling studies and builds on a Stanford-patented platform.

6 investors

Indee Labs develops a non-viral intracellular delivery system based on microfluidic vortex shedding (µVS) to deliver nucleic acids, proteins, and gene editing complexes into patient and donor T cells. The technology targets T cell immunotherapy discovery, development, and manufacturing.

4 investors

Modulari-T develops proprietary cell therapy technologies, including the MARC receptor and MoVe platform, based on primary research into immune system architecture. The company serves the cell therapy and immunotherapy market.

4 investors

Andson Biotech develops analytical tools for the analysis of biotherapeutics, aimed at supporting the discovery and development of biologic drugs.

3 investors

Sixfold Bioscience develops patented drug delivery systems for RNA therapeutics, including siRNAs for gene silencing and mRNAs for gene expression. The company works on commercialising these delivery technologies in the gene therapy and nanomedicine space.

3 investors

WhiteLab Genomics uses AI and computational methods to analyze biological data and accelerate genomic drug discovery and development. The company serves pharmaceutical and biotech organizations seeking to reduce timelines and de-risk the development of genomic medicines.

3 investors

Nuntius develops programmable nanocarriers for drug delivery that can target tissues beyond the liver and utilize machine learning to optimize carrier performance. The company serves pharmaceutical and biotech organizations developing targeted therapeutics.

2 investors

Perception Biosystems develops automated, robotics-driven manufacturing systems for gene therapy, aiming to produce gene therapies for human clinical trials faster and more cheaply than conventional facilities. The company operates a robotic lab in the SF Bay Area and targets rare disease applications.

1 investor