Editas Medicine, Inc. engages in the development and commercialization of genome editing technology. Its technology includes clustered, regularly interspaced short palindromic repeats (CRISPR), and CRISPR associated protein 9 (Cas9). The company was founded by Feng Zhang, Jennifer A. Doudna, George McDonald Church, J. Keith Joung and David R. Liu in September 2013 and is headquartered in Cambridge, MA.
Clinical Trials
8
5 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
2013
Active, not recruiting
4
50.0%
Completed
2
25.0%
Enrolling By Invitation
1
12.5%
Withdrawn
1
12.5%
No approval data available
- Editas Medicine says it remains on track to dose the first patients this year with EDIT-401, an in vivo CRISPR therapy designed to lower LDL cholesterol by upregulating the LDL receptor. - In non-human primate studies, EDIT-401 produced a mean 90% reduction in LDL cholesterol plus reductions in lipoprotein(a) and ApoB, with at least a sixfold rise in liver LDL receptor expression. - The planned Phase I/II trial uses a 3+3 dose-escalation design targeting a human-equivalent dose of about 0.6 mg/kg, with first-cohort safety data expected in the first quarter of next year. - Editas reported $212 million in cash as of the second quarter, a runway expected to fund operations into the second half of 2028.
- Lyora Therapeutics launched with $2.5 million in pre-seed funding to develop one-and-done genetic medicines targeting inherited retinal diseases that currently lack treatments. - The lead program, LYA-101, targets retinitis pigmentosa caused by PRPF31 mutations, with an IND filing planned within 18 months. - A second program, LYA-102, uses optimized CRISPR technology to address USH2A mutations in Usher Syndrome Type 2, with an exclusive option licensed from Editas Medicine. - The company is led by CEO Pam Stetkiewicz, PhD, and co-founded by ocular gene therapy pioneers Luk Vandenberghe, PhD, and Eric Pierce, MD, PhD, of Harvard Medical School.
- The global gene editing therapeutics market is projected to grow at a compound annual growth rate of 12.5%, fueled by expanding clinical applications and rising disease burden. - CRISPR-based technologies dominate the market due to their efficiency and cost-effectiveness, while oncology represents the largest therapeutic application segment. - High development costs, regulatory complexity, and off-target safety concerns remain significant barriers to broader adoption and commercialization. - Key players including CRISPR Therapeutics, Intellia Therapeutics, Editas Medicine, and Beam Therapeutics are driving innovation through partnerships and investments in next-generation editing platforms.
- BreezeBio, formerly GenEdit, secured $60 million in Series B funding to advance its proprietary NanoGalaxy delivery platform and internal therapeutic pipeline focused on autoimmune diseases and oncology. - The company's lead candidate BRZ-101 is designed to restore immune tolerance in type 1 diabetes by delivering autoantigens and tolerogenic co-factors to antigen-presenting cells, advancing into IND-enabling studies. - BreezeBio's NanoGalaxy platform demonstrates targeted delivery to immune, cardiac, pulmonary, and central nervous systems using specialized polymer nanoparticles that can be dosed multiple times. - The funding will support the company's transition from a delivery platform to a therapeutics company while continuing its collaboration with Genentech and expanding capabilities for in vivo CAR-T development.
- Editas Medicine's EDIT-401 gene therapy demonstrated a 90% mean reduction in LDL cholesterol levels in non-human primate studies, significantly outperforming existing lipid-lowering therapies. - The company plans to submit an Investigational New Drug application by mid-2026 and achieve human proof-of-concept by year-end 2026. - EDIT-401 targets the LDLR gene to upregulate LDL receptor expression, positioning it as a potential first-in-class therapy for refractory hypercholesterolemia. - Editas reported $178.5 million in cash reserves as of June 2025, providing operational funding through Q2 2027 without near-term dilution concerns.
- The U.S. Court of Appeals for the Federal Circuit has partially vacated the Patent Trial and Appeal Board's previous decision in the CRISPR/Cas9 patent interference case, remanding it for further review. - Editas Medicine maintains confidence in its intellectual property portfolio, emphasizing that the decision does not affect existing licenses or its ability to license foundational CRISPR patents. - The company holds extensive patents covering CRISPR/Cas9 and CRISPR/Cas12a gene editing in human cells, with only a fraction involved in the ongoing interference proceedings.
- Korean biotech company ToolGen has filed patent infringement lawsuits against Vertex Pharmaceuticals in the UK and Netherlands over the world's first approved CRISPR gene therapy, Casgevy. - ToolGen claims Vertex's $2.2 million therapy was developed using its proprietary CRISPR RNP technology without proper licensing recognition. - The lawsuit targets Casgevy, which treats sickle cell disease and beta-thalassemia and has received marketing authorization in the UK, EU, and US. - ToolGen seeks a global licensing agreement rather than restricting patient access to the breakthrough therapy.
- CRISPR Medicine News tracks approximately 250 clinical trials involving gene-editing therapeutic candidates as of February 2025, with more than 150 trials currently active across diverse therapeutic areas. - CASGEVY became the first approved CRISPR-based therapy in 2023, receiving regulatory clearance in multiple regions for treating sickle cell disease and beta thalassemia through foetal haemoglobin induction. - Gene editing for blood disorders continues to lead the clinical landscape, with the majority of Phase 3 trials targeting sickle cell disease and beta thalassemia, while Phase 3 trials are also underway in hereditary amyloidosis and immunodeficiencies. - Clinical applications now span 15+ therapeutic areas including blood cancers, viral diseases, metabolic disorders, autoimmune diseases, inherited eye diseases, cardiovascular disease, and neurological conditions.
- The global CRISPR-Cas9 therapy market is rapidly expanding with over 25 therapies currently in clinical trials, many of which are expected to receive regulatory approval and enter the market within the next 5-7 years. - Casgevy, the first approved CRISPR-Cas9 therapy for sickle cell anemia and beta thalassemia, is currently available in the US, EU, UK, Saudi Arabia, and Bahrain at approximately $2 million per treatment course. - Applications of CRISPR-Cas9 technology are expected to expand beyond genetic disorders and cancers to include cardiovascular, neurodegenerative, and CNS diseases, potentially transforming into a multi-billion-dollar industry.
- Bayer is investing $300 million over five years in a new joint venture with CRISPR Therapeutics, plus $35 million for a minority stake in the gene-editing pioneer. - The collaboration will focus on developing potential curative treatments for hemophilia, congenital heart disease, and Stargardt disease using CRISPR-Cas9 gene-editing technology. - This partnership represents Bayer's first investment through its newly established Bayer LifeScience Centre and joins other major pharmaceutical companies including Vertex, Novartis, and Celgene in backing CRISPR-Cas9 technology.
