In Vivo Gene Therapies
Review how we are developing potentially life-changing therapies driven by innovative technology.
Pioneering Genetic Medicines for Challenging Diseases
In vivo gene therapies are living medicines in which genetic material is directly inserted into a patient’s body to treat disease. In vivo gene therapies can carry genetic material to specific cells to treat diseased organs or they can be designed to provide instructions to cells and tissues to make specific proteins that can treat disease.
ElevateBio® is advancing novel programs internally and enabling partners in their development of next-generation therapies for otherwise intractable diseases.
Where the Technology is the Therapy
ElevateBio is advancing an array of in vivo gene therapy technologies and therapeutic programs with curative potential. Our platform technologies and ecosystem of capabilities, when combined, enable us to discover, develop, manufacture, and commercialize a broad spectrum of therapies with life transforming potential for patients.
In Vivo Gene Therapies
Gene Edited Therapies
Gene 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 double-strand breaks, base editing, and reverse transcriptase editing.
RNA-based Therapeutics
Ribonucleic acid (RNA) therapeutics are driven by RNA’s unique role in the coding and expression of genes. In RNA-based therapeutics, RNA molecules are delivered to cells to provide genetic instructions to make specific proteins, which can treat or prevent disease, or to disrupt the production of disease-causing proteins.
ElevateBio is developing novel RNA technologies, which can be combined with a variety of delivery systems across the ElevateBio ecosystem, to develop novel ways to target disease.
Adeno-Associated Virus (AAV) Vector Gene Therapies
Adeno-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.
Partner with ElevateBio®
Wherever you are in your cell and gene therapy product lifecycle, we can strengthen and accelerate the development of your transformative therapies with our enabling technologies unmatched manufacturing capabilities.