Editas Medicine Inc
NASDAQ:EDIT
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Editas Medicine Inc
Editas Medicine, Inc. engages in the development and commercialization of genome editing technology. The company is headquartered in Cambridge, Massachusetts and currently employs 264 full-time employees. The company went IPO on 2016-02-03. The firm is focused on developing transformative gene editing medicines to treat a range of serious diseases. The company has developed a gene editing platform based on clustered, regularly interspaced short palindromic repeats (CRISPR) technology. CRISPR uses a protein-ribonucleic acid (RNA) complex composed of an enzyme, including either CRISPR associated protein 9 (Cas9) or CRISPR from Prevotella and Francisella 1 (Cpf1), bound to a guide RNA molecule designed to recognize a particular deoxyribonucleic acid (DNA) sequence. The firm is focused on both in vivo gene editing medicines, in which the medicine is injected or infused into the patient to edit the cells inside their body, and ex vivo gene-edited cell medicines, in which cells are edited with its technology. The firm's gene editing medicine programs include EDIT-101, EDIT-102, EDIT-103, EDIT-301 and EDIT-202.
Editas Medicine, Inc. engages in the development and commercialization of genome editing technology. The company is headquartered in Cambridge, Massachusetts and currently employs 264 full-time employees. The company went IPO on 2016-02-03. The firm is focused on developing transformative gene editing medicines to treat a range of serious diseases. The company has developed a gene editing platform based on clustered, regularly interspaced short palindromic repeats (CRISPR) technology. CRISPR uses a protein-ribonucleic acid (RNA) complex composed of an enzyme, including either CRISPR associated protein 9 (Cas9) or CRISPR from Prevotella and Francisella 1 (Cpf1), bound to a guide RNA molecule designed to recognize a particular deoxyribonucleic acid (DNA) sequence. The firm is focused on both in vivo gene editing medicines, in which the medicine is injected or infused into the patient to edit the cells inside their body, and ex vivo gene-edited cell medicines, in which cells are edited with its technology. The firm's gene editing medicine programs include EDIT-101, EDIT-102, EDIT-103, EDIT-301 and EDIT-202.
Clinical Progress: Editas completed enrollment of both adult and adolescent cohorts in its reni-cel sickle cell and beta-thalassemia trials and is on track to share additional data by year-end.
Reni-cel Data: All RUBY trial patients remain free from vaso-occlusive events post-infusion, with robust hemoglobin correction and high fetal hemoglobin levels.
In Vivo Pipeline: The company remains on schedule to deliver preclinical proof-of-concept for an in vivo program by the end of 2024, leveraging a differentiated gene upregulation strategy.
Financial Position: Cash, cash equivalents and marketable securities totaled $318 million at June 30, expected to fund operations into 2026.
Guidance: Management continues to benchmark its BLA strategy for reni-cel against CASGEVY, with ongoing FDA dialogue and no filing timeline yet provided.
Strategic Options: Editas is open to partnering reni-cel for commercialization outside the US and potentially within the US as well, focusing its resources on progressing in vivo programs.