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Editas Medicine, Inc. (Nasdaq: EDIT) is a clinical-stage genome editing company committed to developing transformative genomic medicines to treat serious diseases. The company leverages its proprietary CRISPR/Cas9 and CRISPR/Cas12a technology to create precise and corrective molecular modifications at the genetic level, aiming to address the root cause of various diseases. Editas Medicine has made significant strides in the development and commercialization of its genome editing platform. Currently, the company is advancing its flagship program, EDIT-301, now known as renizgamglogene autogedtemcel (reni-cel), which shows promise as a one-time, durable treatment for severe sickle cell disease (SCD) and beta thalassemia (TDT). Recent data from the RUBY and EdiTHAL trials indicate that reni-cel is well-tolerated and has demonstrated significant clinical benefits, including early correction of anemia and sustained increases in fetal hemoglobin.
Financially, Editas Medicine maintains a strong position, with cash, cash equivalents, and marketable securities amounting to $446.4 million as of September 30, 2023. The company's financial strategy is bolstered by strategic partnerships such as the recent license agreement with Vertex Pharmaceuticals and an extended collaboration with Bristol Myers Squibb. These agreements extend Editas Medicine's cash runway into 2026, supporting ongoing and future projects.
Editas Medicine is also pioneering in vivo genome editing. Recent preclinical data presented at the American Society of Gene and Cell Therapy (ASGCT) meeting showcased the company's progress in lipid nanoparticle (LNP) formulations for efficient in vivo delivery of AsCas12a mRNA, demonstrating significant scientific advancements in the field.
The company's mission is reinforced by its strong intellectual property portfolio, including exclusive licenses for CRISPR/Cas12a and CRISPR/Cas9 genome editing systems from Broad Institute and Harvard University. The company remains dedicated to translating groundbreaking genomic research into clinical applications, aiming to provide life-changing therapies for patients worldwide.
For the latest updates, including financial results, clinical trial progress, and strategic initiatives, visit the Editas Medicine website.
Editas Medicine (EDIT) announced a strategic transition to focus exclusively on in vivo CRISPR-edited medicines, following recent breakthroughs in preclinical studies. The company achieved ~40% editing of the HBG1/2 promoter site using their proprietary targeted lipid nanoparticle delivery system, resulting in 20% HbF expressing human red blood cells. They also demonstrated high-efficiency liver editing in non-human primates.
As part of this transition, Editas is ending development of reni-cel and implementing significant cost-saving measures, including a 65% workforce reduction over the next six months. Several management changes were announced, including the departure of Chief Medical Officer Baisong Mei and two board members. The restructuring is expected to extend the company's cash runway into Q2 2027.
Editas Medicine (NASDAQ: EDIT) presented updated clinical data from the Phase 1/2/3 RUBY trial of reni-cel in 28 patients with severe sickle cell disease (SCD). The trial demonstrated positive results with 27 of 28 patients remaining free of vaso-occlusive events post-treatment. Key findings include early normalization of total hemoglobin from 9.8 g/dL at baseline to 13.8 g/dL at Month 6, and sustained fetal hemoglobin levels above 40%.
The treatment was well-tolerated with a safety profile consistent with myeloablative busulfan conditioning. Patients showed successful engraftment with median times of 23 days for neutrophil and 25 days for platelet engraftment. Only two serious adverse events possibly related to reni-cel were reported. The median follow-up was 9.5 months, with 11 patients having more than one year of follow-up.
Editas Medicine (Nasdaq: EDIT), a clinical-stage gene editing company, has announced its participation in three major healthcare investor conferences. The company will be featured in fireside chat sessions at Guggenheim's Inaugural Healthcare Innovation Conference on November 12 at 4:00 p.m. ET in Boston, the Stifel 2024 Healthcare Conference on November 19 at 1:50 p.m. ET in New York, and the 7th Annual Evercore ISI HealthCONx Conference on December 3 at 1:20 p.m. ET in Coral Gables. Live webcasts will be available on the company's website, with replays accessible for approximately 30 days after each event.
Editas Medicine (EDIT) reported Q3 2024 financial results, highlighting a net loss of $62.1 million ($0.75 per share). The company achieved preclinical proof of concept for in vivo HBG1/2 editing in HSPCs using proprietary targeted LNP technology for sickle cell disease and beta thalassemia treatment. Cash position stands at $265.1 million, expected to fund operations into Q2 2026. The company secured a $57 million upfront payment from DRI Healthcare Trust through the sale of certain future license fees. Research and development expenses increased to $47.6 million, while revenues decreased to $0.1 million.
Editas Medicine (Nasdaq: EDIT) announced significant progress towards its 2024 goals, including achieving in vivo preclinical proof of concept for hematopoietic stem and progenitor cell (HSPC) editing. The company utilized its proprietary targeted lipid nanoparticle (tLNP) to achieve a 29% editing level in HSPCs after a single dose, resulting in 20% HbF-expressing human red blood cells. This advancement is important for developing an in vivo treatment for sickle cell disease and beta thalassemia.
Editas also initiated a process to partner or out-license reni-cel, focusing resources on in vivo pipeline development. The company completed enrollment for the RUBY trial for severe sickle cell disease and the EdiTHAL trial for transfusion-dependent beta thalassemia. Additionally, Editas secured $57 million in non-dilutive capital through a sale agreement with DRI Healthcare Trust and ended Q3 2024 with approximately $265 million in cash and equivalents.
Editas Medicine (Nasdaq: EDIT) and Genevant Sciences have announced a collaboration and nonexclusive license agreement to develop in vivo gene editing medicines. The partnership will combine Editas' CRISPR Cas12a genome editing systems with Genevant's proprietary lipid nanoparticle (LNP) technology for two undisclosed targets in Editas' upregulation strategy.
Under the agreement, Genevant has granted Editas a nonexclusive worldwide license for certain LNP technology to develop mRNA-CRISPR Cas12a-LNP products. Genevant is eligible to receive up to $238 million in upfront and contingent milestone payments, as well as tiered royalties on future product sales.
This collaboration aims to advance Editas' vision of becoming a leader in in vivo programmable gene editing medicine, leveraging Genevant's expertise in LNP delivery systems, which have emerged as a preferred approach for delivering gene editing constructs.
Editas Medicine (Nasdaq: EDIT), a clinical-stage gene editing company, has announced a strategic update webinar scheduled for Tuesday, October 22, 2024, at 8:00 a.m. ET. The webinar will focus on the company's progress towards its 2024 goals, including the significant achievement of establishing in vivo preclinical proof of concept.
The presentation will also cover business development and financial updates. Interested parties can access the live and archived webcast through a provided link or via the Events & Presentations page on the company's website. A replay of the webinar will be available in the Investors section of the Editas Medicine website following the conclusion of the event.
This strategic update offers an opportunity for stakeholders to gain insights into Editas Medicine's recent advancements and future directions in the field of gene editing.
Editas Medicine (Nasdaq: EDIT) has announced a $57 million upfront cash payment from DRI Healthcare Trust in exchange for certain future license fees and payments under its Cas9 license agreement with Vertex Pharmaceuticals. This non-dilutive capital will support Editas Medicine's pipeline development and strategic priorities.
The deal includes up to 100% of future annual license fees ranging from $5 million to $40 million per year and a mid-double-digit percentage of Editas Medicine's portion of a potential $50 million contingent upfront payment from Vertex. Editas Medicine retains rights to fixed annual license fees for 2024 and a mid-single-digit million-dollar payment upon Vertex achieving certain annual sales milestones.
This agreement follows Editas Medicine's December 2023 non-exclusive license to Vertex for Cas9 gene editing technology in sickle cell disease and beta thalassemia treatments, including CASGEVY®.
Editas Medicine (Nasdaq: EDIT), a clinical-stage gene editing company, has announced its participation in four upcoming investor conferences in September and October 2024. The company's management will engage in fireside chat discussions about Editas and its programs, including reni-cel.
The conferences include:
- Morgan Stanley 22nd Annual Global Healthcare Conference on September 4 in New York
- 2024 Wells Fargo Healthcare Conference on September 5 in Boston
- 2024 Cantor Global Healthcare Conference on September 17 in New York
- Chardan 8th Annual Genetic Medicines Conference on October 1 in New York
Live webcasts of Editas Medicine's presentations will be available on the company's website, with archived replays accessible for approximately 30 days after each event.
Editas Medicine (Nasdaq: EDIT) reported its Q2 2024 results and provided business updates. The company is on track to present additional clinical data from the RUBY trial for sickle cell disease and the EdiTHAL trial for beta thalassemia by year-end. Editas also aims to establish in vivo preclinical proof-of-concept for an undisclosed indication by year-end.
Financial highlights include:
- Cash, cash equivalents, and marketable securities of $318.3 million as of June 30, 2024
- Net loss of $67.6 million, or $0.82 per share
- Research and development expenses increased to $54.2 million
The company expects its current financial position to fund operations into 2026.
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