Editas Medicine Linkedin .hamburger .st0{fill:none;stroke:#FFFFFF;stroke-width:0.5;} Overview In Vivo Medicines Ex Vivo Cell Medicines Posters & Presentations OUR RESEARCH AND PIPELINE In Vivo Medicines and Ex Vivo Cell Medicines Our mission is to translate the power and potential of gene editing into a broad class of differentiated and transformational medicines that can transform lives. We have focused our research and development efforts on diseases for which there are currently few or no treatments available. Editas Medicine works with two distinct CRISPR nucleases: Cas9 and Cas12a (also known as Cpf1). Both nucleases have distinct gene editing and targeting capabilities. Using these two nucleases, we can access a broad range of genetic mutations and develop targeted and durable gene edited medicines. We are researching and developing two types of gene editing medicines: in vivo gene edited medicines that edit genes inside the body, and ex vivo gene edited cell medicines where the gene editing occurs outside the body to create edited cell medicines that are administered to patients. Leveraging both of these approaches will help us target a broader range of diseases. Research and Pipeline In Vivo Gene Edited Medicines Ocular EDIT-101 Leber Congenital Amaurosis 10 (LCA10) Neurological Diseases EDIT-102: Usher Syndrome 2a (USH2A) Autosomal Dominant Retinitis Pigmentosa 4 (RP4) Other Organs Ex Vivo Gene Edited Cell Medicines Hemoglobinopathies EDIT-301: Sickle Cell Disease (SCD) EDIT-301: Transfusion-dependent beta thalassemia (TDT) Cellular Therapy Medicines Oncology αβ T Cells iPSC NK (iNK) Cells In Vivo Gene Edited Medicines We are pursuing a number of diseases using an in vivo editing approach, and are excited about the progress we’ve made in ocular diseases. Take a look at the work we’re doing to improve outcomes for people with Leber congenital amaurosis 10 (LCA10) and other genetic ocular diseases. Ocular Disease EDIT-101 for LCA10 Our approach to LCA10 is to target a disease-causing mutation in the CEP290 gene that causes degeneration in ocular photoreceptor cells—cells critical for experiencing normal vision. We’ve developed an experimental CRISPR medicine called EDIT-101 designed to remove the CEP290 mutation. This approach may restore normal protein expression, photoreceptor function, and ultimately, vision. Usher Syndrome 2A (USH2A) Mutations in exon 13 of the USH2A gene are most often responsible for the development of USH2A. This information supports the design of a CRISPR medicine that edits exon 13 to restore healthy USH2A protein expression, consequently restoring the function of photoreceptors. Autosomal Dominant Retinitis Pigmentosa 4 (RP4) Retinitis pigmentosa is a set of rare inherited retinal disorders that cause a progressive vision loss. We believe that gene editing is well suited to address autosomal dominant mutations that drive the disease subset known as RP4, enabling the persistence of vision. Other In Vivo Research We are pursuing in vivo gene edited medicines for neurological diseases. These projects are in the discovery phase. Ex Vivo Gene Edited Cell Medicines We are also developing several ex vivo gene edited cell medicines using ex vivo editing. We have two approaches to making ex vivo gene edited cell medicines. The first approach is to remove an individual’s cells, edit them, then reintroduce them to the patient. The second approach is to have a universal cell population that can be edited and then given to any patient who needs it, without needing the patient to donate cells first. The potential applications for this technology are numerous, but we’re currently focusing our research on cancer and certain hematologic (blood) diseases. Cancer We are working with several cell types to apply genome editing to develop cell medicines for a broad range of cancers. We’re exploring two approaches: autologous ex vivo gene edited cell medicines and allogeneic ex vivo gene edited cell medicines. Both approaches have the potential to revolutionize how we treat patients with cancer at the DNA level. Blood Diseases EDIT-301 for Sickle Cell Disease (SCD) and Beta-thalassemia SCD and beta-thalassemia are serious hematologic diseases that continue to have unmet medical needs. In the US alone, there are 90,000 to 100,000 people living with SCD. We have developed a potentially best-in-class treatment, known as EDIT-301, for these diseases that utilizes CRISPR/Cas12a to edit the beta-globin locus and directly increase fetal hemoglobin. Posters and Presentations We ground our work in a wide range of fundamental scientific knowledge. 2021 2020 2019 2018 2017 2016 2015 2014 2013 2012 November 12, 2021 Alexander G. Allen, et al. GAPDH knock-in of high-affinity CD16 and mbIL-15 in iPSC-derived NK cells drives high-level expression and increased anti-tumor function. Presented at The Society for Immunotherapy of Cancer (SITC) Annual Meeting 2021, November 10-14, 2021. Download September 23, 2021 John Zuris. An Engineered AsCas12a Nuclease Facilitates the Rapid Generation of Therapeutic Cell Medicines. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021. Download September 23, 2021 Keith Jarvis. IND-enabling Small-Scale Guide RNA Production Under GMP for CRISPR Based Cell Therapies. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021. Download September 23, 2021 Steve Wolk. Characterization of gRNAs and Ribonucleoproteins for CRISPR Applications. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021. Download September 21, 2021 Jean-Noel Lemercier, et al. Characterization of CRISPR RNPs by Ion-Exchange Chromatography. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021. Download September 21, 2021 Pranjali Ghude, et al. Understanding CRISPR RNA Secondary Structure Impact on Ribonucleoprotein (RNP) Behavior by SEC-PAGE. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021. Download August 20, 2021 J. A. Zuris. A Method for Highly Efficient Knock-in and Expression of Transgene Cargos for Next Generation Cell-based Medicine. Presented at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting, August 18-20, 2021. Download August 18, 2021 Eugenio Marco et al. CALITAS: a CRISPR-Cas-aware ALigner for In silico off-TArget Search. Presented at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting, August 18-20, 2021. Download June 11, 2021 Edouard De Dreuzy, et al. Preclinical Data Supporting the Initiation of the EDIT-301 Phase I/II RUBY Clinical Trial for the Potential Treatment of Sickle Cell Disease. Presented at the 26th Congress of the European Hematology Association (EHA), June 9 – 17, 2021. Download May 11, 2021 M. Allocca, D. Reyon, et al. Advances Toward a Dual AAV CRISPR-Cas9-based “Knockout and Replace” Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa. The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 11 – 14, 2021 Download May 10, 2021 H. MacLeod. The development of CRISPR-based medicines for the treatment of ocular diseases. The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 11 – 14, 2021 Download May 4, 2021 N. Dilmac, et al. CRISPR Gene Editing Rescues Deficits in Human USH2A Mutant Retinal Organoids. The Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, May 1-7, 2021 Download May 3, 2021 M. Allocca, et al. Advances Toward a Dual AAV CRISPR-Cas9-based “Knockout and Replace” Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa. The Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, May 1-7, 2021 Download April 10, 2021 K. Wong, et al. CRISPR-Cas12a Gene Editing Enhances Functional Metabolism of Natural Killer Cells and Enables Tumor Cell Cytolysis in Metabolically Stressful Conditions That Inhibit Effector Cell Function. Presented at the American Association for Cancer Research (AACR) Annual Meeting, April 10 – 15, 2021 Download April 10, 2021 Christopher M. Borges, et al. A Multiplexed CRISPR-Cas12a Gene-Edited Healthy Donor-Derived NK Cell Therapy with Increased Granzyme B and Degranulation Supports Improved Serial Killing Capacity. Presented at the American Association for Cancer Research (AACR) Annual Meeting, April 10 – 15, 2021 Download Research and Pipeline CRISPR Gene Editing For Patients Who We Are Join Our Team Investors Terms of Use Privacy Policy Get in Touch Newsroom Editas Medicine Linkedin ©Copyright 2021 Editas Medicine