GeneToBe
GeneToBe, Inc. is a company focused on next-generation precision medicine and gene therapy. Based in Ann Arbor, MI, it specializes in preclinical and clinical stage assets, utilizing advanced technologies such as CRISPR/Cas9 platform (miCas9) to develop genetic therapies for various diseases, including rare genetic disorders like Usher Syndrome and Cystic Fibrosis. The company is led by a team with extensive experience in drug discovery, development, and commercialization, and is actively involved in research, publications, and collaborations to advance gene editing and therapeutic solutions.
Industries
Nr. of Employees
small (1-50)
Products
Engineered Cas9-based HDR-enhancing nuclease
A Cas9-based gene-editing nuclease modified with a small HDR-promoting peptide module to increase precise large-fragment knock-in efficiency and reduce undesired indels.
Gene-edited rabbit disease models (Cystic Fibrosis, Usher Syndrome)
Preclinical rabbit models carrying disease-relevant mutations (including CFTR ΔF508 and CLRN1 variants) that recapitulate hearing and vision deficits for evaluation of therapeutics.
Preclinical therapeutic programs for Cystic Fibrosis and Usher Syndrome
In-house translational programs combining gene-editing approaches and small-molecule interventions to develop potential lifelong therapies for genetic disorders.
Engineered Cas9-based HDR-enhancing nuclease
A Cas9-based gene-editing nuclease modified with a small HDR-promoting peptide module to increase precise large-fragment knock-in efficiency and reduce undesired indels.
Gene-edited rabbit disease models (Cystic Fibrosis, Usher Syndrome)
Preclinical rabbit models carrying disease-relevant mutations (including CFTR ΔF508 and CLRN1 variants) that recapitulate hearing and vision deficits for evaluation of therapeutics.
Preclinical therapeutic programs for Cystic Fibrosis and Usher Syndrome
In-house translational programs combining gene-editing approaches and small-molecule interventions to develop potential lifelong therapies for genetic disorders.
Services
Design and production of knock-out, knock-in, and transgenic rabbit and other large-animal models for translational research and therapeutic evaluation.
In vivo efficacy, safety, and natural history characterization of therapeutics in gene-edited animal models to support candidate selection for clinical development.
Design support and planning for randomized, double-blind, placebo-controlled clinical studies derived from preclinical candidate selection.
Management and disclosure of patent applications and peer-reviewed publications documenting platform and program data.
Design and production of knock-out, knock-in, and transgenic rabbit and other large-animal models for translational research and therapeutic evaluation.
In vivo efficacy, safety, and natural history characterization of therapeutics in gene-edited animal models to support candidate selection for clinical development.
Design support and planning for randomized, double-blind, placebo-controlled clinical studies derived from preclinical candidate selection.
Management and disclosure of patent applications and peer-reviewed publications documenting platform and program data.
Expertise Areas
- Genome editing and nuclease engineering
- Homology-directed repair optimization
- Large-animal (rabbit/pig) preclinical model development
- Translational development for rare genetic diseases
Key Technologies
- Engineered CRISPR/Cas9 with HDR-promoting peptide fusion
- Homology-directed repair (HDR) enhancement
- RAD51 recruitment motifs
- ZFN and TALEN nucleases
News & Updates
GeneToBe's colleagues, Dr. Ricardo Garcia and Professor Wanqing Liu, will attend the AASLD Liver Meeting to explore new discoveries and advances in hepatology. They invite contact for discussions on activities targeting MASH and other disease indications.
A scientific article published in Translational Vision Science & Technology on February 16, 2023, discussing gene mutations in rabbits related to Usher Syndrome.
Hirai et al. The sodium/glucose cotransporters as potential therapeutic targets for CF lung diseases
A study published in Molecular Therapy: Methods & Clinical Development in March 2022, exploring therapeutic targets for cystic fibrosis lung diseases.
Research article in Frontiers in Genetics on January 22, 2021, about generating CFTR mutant rabbits.
GeneToBe developed a novel CRISPR/Cas9 platform (miCas9) that enhances gene editing efficacy, reduces off-target effects, and minimizes on-target indels, advancing the safety and efficiency of gene therapy.
GeneToBe's colleagues, Dr. Ricardo Garcia and Professor Wanqing Liu, will attend the AASLD Liver Meeting to explore new discoveries and advances in hepatology. They invite contact for discussions on activities targeting MASH and other disease indications.
A scientific article published in Translational Vision Science & Technology on February 16, 2023, discussing gene mutations in rabbits related to Usher Syndrome.
Hirai et al. The sodium/glucose cotransporters as potential therapeutic targets for CF lung diseases
A study published in Molecular Therapy: Methods & Clinical Development in March 2022, exploring therapeutic targets for cystic fibrosis lung diseases.
Research article in Frontiers in Genetics on January 22, 2021, about generating CFTR mutant rabbits.
GeneToBe developed a novel CRISPR/Cas9 platform (miCas9) that enhances gene editing efficacy, reduces off-target effects, and minimizes on-target indels, advancing the safety and efficiency of gene therapy.