We are rapidly translating our specific, efficient and versatile CRISPR/Cas9 gene-editing platform into therapies to treat hemoglobinopathies, cancer, diabetes and other diseases. Our multi-disciplinary team of world-class researchers and drug developers works every day to translate our CRISPR/Cas9 technology into breakthrough human therapeutics. Our lead program targeting the blood diseases β-thalassemia and sickle cell disease has entered clinical testing, as has our first allogeneic CAR-T program targeting B-cell malignancies. We are also advancing additional blood stem cell, immuno-oncology, regenerative medicine and in vivo programs towards the clinic. Source
No articles found.
Neos Therapeutics develops, manufactures, and commercializes innovative extended-r...
Neos Therapeutics develops, manufactures, and c...
We are a global frontrunner in making oral vaccines a reality. Weâve created a p...
We are a global frontrunner in making oral vacc...
Crinetics is a clinical stage pharmaceutical company focused on the discovery, dev...
Crinetics is a clinical stage pharmaceutical co...
For more than 40 years, IntriCon has been meeting the rising demand for smaller, p...
For more than 40 years, IntriCon has been meeti...
Fresenius is a global healthcare group offering high-quality products and services...
Fresenius is a global healthcare group offering...
REGENXBIO is a leading biotechnology company focused on the development, commercia...
REGENXBIO is a leading biotechnology company fo...
Join the National Investor Network and get the latest information with your interests in mind.