Genetic Medicines
// Genetic Medicines
Accelerating Therapeutic Development for the Genetic Medicine Industry
ElevateBio® is powering the cell and gene therapy industry forward by helping partners realize the potential of their therapies from concept to cure. We have integrated a differentiated set of platform technologies, including full-spectrum gene editing, with industry-leading manufacturing expertise to develop genetic medicines faster and more efficiently.
Driving a Diverse Array of Modalities
We have expansive expertise to design, develop, and manufacture ex vivo and in vivo cell and gene therapies across a range of therapeutic designs.
We provide comprehensive GMP manufacturing solutions for mRNA vaccines and genetic medicines for all phases of development.
Ex Vivo Cell & Gene Therapies
Ex vivo cell and gene therapies are living medicines in which immune cells and genetic material are engineered outside of the body and then reintroduced to patients. This engineering is designed to improve immune cells’ ability to fight disease. ElevateBio modifies the genetic material through its proprietary gene editing technologies and engineering expertise to optimize therapies and directly target the source of disease.
Autologous Cell TherapyAutologous cell therapy is a form of personalized medicine that uses a patient’s own genetically modified immune cells to treat disease. In autologous therapies, cells are removed from a patient, engineered to elicit the desired therapeutic effect, and reintroduced to the patient. To create autologous therapies, like chimeric antigen receptor (CAR) T and T cell receptor (TCR) cell therapies, ElevateBio combines target selection, cell and vector engineering and gene editing to modify cells and optimize their performance. |  |
Allogeneic Cell TherapyAllogeneic cell therapy is an ‘off-the-shelf’ form of immunotherapy designed to be readily accessible for patients. Cells are derived from healthy donors or cell lines and engineered to be available for treatments on demand. Allogeneic therapies are highly scalable with increased manufacturing efficiency relative to autologous approaches. ElevateBio leverages its deep gene editing and cell and vector engineering expertise to optimize cells for allogeneic cell therapies. |  |
Lentiviral Vector Cell & Gene TherapiesLentiviral vectors are a method to deliver a gene as a therapy for patients. Engineered outside of the body, lentiviral vectors can be used to express the gene of interest in autologous, allogeneic, and iPSC-derived cell therapies. |  |
In Vivo Cell & Gene Therapies
In vivo cell and gene therapies are living medicines delivered directly into a patient’s body to treat disease. ElevateBio develops and delivers these therapies using its integrated platforms to precisely target disease at its source and improve patient outcomes.
Gene Edited TherapiesGene edited therapies are powered by edits made to DNA to treat the most challenging genetic disorders. Novel technologies allow genetic material to be removed, added, or altered at a specific location in the genome within the cells and tissues of interest. ElevateBio’s Life Edit gene editing platform provides flexible editing and unprecedented access to the genome to make any edit, anywhere possible. We are advancing a full spectrum of gene edit types, including nuclease editing, base editing, and reverse transcriptase editing. |  |
Cell TherapiesIn vivo cell therapies deliver genetic instructions directly into a patient’s body to reprogram immune cells to recognize and attack disease cells. Unlike traditional approaches that require extracting a patient’s cells for laboratory engineering, this approach engineers therapeutic cells in real-time within the body. Leading applications include CAR-T therapies, where T cells are reprogrammed to recognize and attack cancer cells directly in the patient. This approach aims to reduce treatment timelines while make cell therapies accessible to more patients. ElevateBio’s targeted delivery capabilities and Life Edit gene editing tools provide the precision necessary to engineer functional therapeutic cells in the body. |  |
Adeno-Associated Virus (AAV) Vector Gene TherapiesAdeno-associated virus (AAV) vectors are a method to deliver DNA to cells or tissues to target disease. The AAV vectors are engineered to transport genetic material into the body, which then instructs cells to provide the desired therapeutic effect. Our viral vector engineering technologies can support all stages of development, including end-to-end process development for AAV vectors. |  |
mRNA Vaccines & Therapeutics
We provide cGMP manufacturing capabilities to accelerate the development and production of mRNA vaccines and genetic medicines. We can support mRNA drug substance and drug product manufacturing, for any stage of development. We provide the infrastructure, expertise, and regulatory support needed to bring your mRNA vaccines and therapeutics to market efficiently.
Gene Editing Technologies and R&D
ElevateBio Life Edit is our gene editing technology and R&D business, featuring one of the world’s largest and most diverse libraries of RNA-guided editing libraries, opening a new frontier of medicine.
cGMP Production, Process Development and Analytical Development Services
ElevateBio BaseCamp is our cGMP manufacturing business, designed to deliver genetic medicines from concept through commercialization.
Partner with ElevateBio®
Wherever you are in your cell and gene therapy product lifecycle, we can strengthen and accelerate the development of transformative therapies with our enabling technologies unmatched manufacturing capabilities.
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