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.
相关临床试验
8
5 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2013
进行中（未招募）
4
50.0%
已完成
2
25.0%
Enrolling By Invitation
1
12.5%
撤回
1
12.5%
暂无批准数据
- 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.
