Kinea Bio
Biotechnology company founded in 2020 focused on genetic-medicine approaches for neuromuscular diseases. Develops platform technologies to enable expression or delivery of large therapeutic proteins and muscle-targeted gene transfer, and advances programs through preclinical, translational, and IND-enabling development.
Industries
Nr. of Employees
small (1-50)
Products
Platform for expressing large therapeutic proteins via split-intein trans-splicing
A platform approach that joins separately delivered protein segments inside cells to reconstitute large functional proteins that exceed single-vector packaging limits.
Muscle-targeted regulatory cassette library
Library of muscle-specific regulatory elements for tuning transgene expression across skeletal and cardiac tissues over a wide dynamic range.
Engineered muscle-tropic viral vectors
Engineered AAV vector capsids optimized for enhanced delivery to muscle and reduced hepatic exposure.
Cell-derived nanovesicle delivery platform for non-viral repeat dosing
Nanovesicle-based delivery system derived from human cells intended to enable repeated administrations of genetic cargo with lower immunogenicity than viral vectors.
SIMPLI-GT™
Split Intein Mediated Protein Ligation Gene TherapyTM overcomes AAV packaging limitations by joining protein segments together to form large functional proteins, enabling production of larger or full-length proteins for diseases like Duchenne muscular dystrophy and dysferlinopathies.
Myotropic Capsids
Proprietary engineered adeno-associated viral vectors (AAVs) formulated for neuromuscular diseases, improving muscle-specific delivery to lower safety risks and reduce treatment costs.
Platform for expressing large therapeutic proteins via split-intein trans-splicing
A platform approach that joins separately delivered protein segments inside cells to reconstitute large functional proteins that exceed single-vector packaging limits.
Muscle-targeted regulatory cassette library
Library of muscle-specific regulatory elements for tuning transgene expression across skeletal and cardiac tissues over a wide dynamic range.
Engineered muscle-tropic viral vectors
Engineered AAV vector capsids optimized for enhanced delivery to muscle and reduced hepatic exposure.
Cell-derived nanovesicle delivery platform for non-viral repeat dosing
Nanovesicle-based delivery system derived from human cells intended to enable repeated administrations of genetic cargo with lower immunogenicity than viral vectors.
SIMPLI-GT™
Split Intein Mediated Protein Ligation Gene TherapyTM overcomes AAV packaging limitations by joining protein segments together to form large functional proteins, enabling production of larger or full-length proteins for diseases like Duchenne muscular dystrophy and dysferlinopathies.
Myotropic Capsids
Proprietary engineered adeno-associated viral vectors (AAVs) formulated for neuromuscular diseases, improving muscle-specific delivery to lower safety risks and reduce treatment costs.
Services
Collaborative preclinical-to-IND program planning, execution of IND-enabling studies, and translational strategy support for gene-therapy candidates.
Execution of in vivo efficacy studies in rodent and large-animal models and integration with in vitro iPSC-derived functional assays.
Collaborative preclinical-to-IND program planning, execution of IND-enabling studies, and translational strategy support for gene-therapy candidates.
Execution of in vivo efficacy studies in rodent and large-animal models and integration with in vitro iPSC-derived functional assays.
Expertise Areas
- Gene therapy development for neuromuscular diseases
- AAV vector design and immunogenicity evaluation
- Non-viral nanovesicle delivery systems
- Translational medicine and IND-enabling studies
Key Technologies
- Intein-mediated protein trans-splicing
- Muscle-specific expression cassettes (regulatory libraries)
- Engineered AAV capsids for tissue tropism
- Cell-derived nanovesicle delivery (non-viral)
News & Updates
Publication reporting outcomes of AAV-gene therapy studies in the mdx mouse model of DMD.
Publication describing the split-intein protein trans-splicing approach for expressing large proteins in gene therapy contexts.
Press release on financing and a capsid license to support a gene-therapy program for dysferlin-related diseases.
Announcements of Phase 1 and Phase 2 NIH Small Business Innovation Research grants awarded to support research in myotonic dystrophy.
Publication reporting outcomes of AAV-gene therapy studies in the mdx mouse model of DMD.
Publication describing the split-intein protein trans-splicing approach for expressing large proteins in gene therapy contexts.
Press release on financing and a capsid license to support a gene-therapy program for dysferlin-related diseases.
Announcements of Phase 1 and Phase 2 NIH Small Business Innovation Research grants awarded to support research in myotonic dystrophy.