Editas Medicine Reports Q1 2024 Results and Updates

Editas Medicine, Inc., a clinical-stage gene editing company, has announced its financial results for the first quarter of 2024 and provided several business updates. The company is making significant strides in its primary strategic areas, including ex vivo and in vivo gene editing, as well as business development and intellectual property.

One of the critical highlights is the progress in the Phase 1/2/3 RUBY clinical trial for severe sickle cell disease (SCD). Editas has completed enrollment of the adult cohort and has started enrolling multiple patients in the adolescent cohort. They are on schedule to present a comprehensive clinical data set in mid-2024 and further updates by the end of the year. Additionally, the EdiTHAL trial for transfusion-dependent beta-thalassemia (TDT) continues to enroll and dose patients, with additional clinical data expected to be shared in mid-2024 and more updates by year-end.

Editas is also advancing its in vivo gene editing capabilities. The company will present pre-clinical data at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting. These presentations will include studies on the use of lipid nanoparticle (LNP)-mediated delivery systems and engineered AsCas12a messenger RNA (mRNA) for gene editing in multiple cell types, including liver cells. Another focus is on developing novel in vivo gene editing technologies using potent large serine recombinases (LSRs) to enable whole gene knock-in, potentially expanding the range of treatable conditions.

In terms of business development, Editas extended its collaboration with Bristol Myers Squibb for two additional years. This partnership aims to research, develop, and commercialize autologous and allogeneic alpha-beta T cell medicines for cancer and autoimmune diseases. There is also an option to extend this collaboration for an additional two years.

On the intellectual property front, oral arguments were held before the U.S. Court of Appeals for the Federal Circuit regarding the CRISPR patent interference case. A decision is anticipated in the second half of 2024. This case involves a dispute over specific patents for CRISPR/Cas9 editing in human cells between the Broad Institute and a consortium including the University of California, the University of Vienna, and Emmanuelle Charpentier.

The company also appointed Gregory Whitehead as Executive Vice President and Chief Technical and Quality Officer. With over 25 years of experience in the biotech industry, Whitehead will lead the company’s technical development, operations, and quality departments. His extensive background includes roles at Rubius Therapeutics and bluebird bio, where he was instrumental in scaling organizations to support gene therapy clinical trials and commercial launches.

Financially, Editas reported a net loss of $62.0 million for the first quarter of 2024, compared to $49.0 million for the same period in 2023. This increase in net loss is primarily due to higher research and development expenses, which rose by $11.0 million to $48.8 million. This increase is mainly attributed to higher sublicense and license payments and clinical and manufacturing costs associated with the reni-cel program. General and administrative expenses decreased by $3.7 million to $19.3 million, primarily due to lower professional service expenses and reduced legal and patent costs.

As of March 31, 2024, Editas had $376.8 million in cash, cash equivalents, and marketable securities, which the company expects to fund its operations into 2026.

Editas Medicine continues to participate actively in scientific and medical conferences, including upcoming investor events like the Bank of America Health Care Conference 2024, the 2024 RBC Capital Markets Global Healthcare Conference, and Stifel’s 2nd Annual Genetic Medicines Forum.

Overall, Editas Medicine is making significant progress toward becoming a commercial-stage company and advancing its gene editing medicines for serious, previously untreatable diseases.