Editas Medicine, Inc.

iStock, leolintang BioSpace has been compiling a list of the most innovative and exciting biotechs for a decade. Here we take a look back at noteworthy companies from each of those lists. 2024 marked the 10th year of BioSpace ’s NextGen list, a collection of biotechs that are on the cutting edge and poised to break barriers in the industry. As we prep next year’s list, to be released in January, we decided to take a look back at a notable honoree from each of our past lists. There were many fascinating companies that have gone on to great things; on the other hand, sometimes things don’t go as well as we predicted. We have a few not-so-great stories to tell, too. ___________________________________________ 2024 ReNAgade Therapeutics Status: Bought by Orna Therapeutics This company made our list for a pretty big reason: a massive $300 million series A in one of the toughest financing environments in recent memory. The company was launched in May 2023 to develop RNA medicines to treat diseases anywhere in the body. One of the lead investors in the round was MPM BioImpact, whose managing partner Ansbert Gadicke founded ReNAgade. Early in the biotech’s history as a stealth company, there was a bit of foreshadowing as to where the company would end up. ReNAgade struck up a partnership with Orna Therapeutics, which was also working on RNA therapeutics. The work attracted the attention of Merck in a deal worth up to $3.5 billion with development and commercialization milestones. Given the money on the line, Orna went all in on ReNAgade, buying out the biotech in May of this year—almost a year to the day after its launch. Financial details were not disclosed. See the full class of 2024 . ___________________________________________ 2023 Mariana Oncology Status: Bought by Novartis What a year 2023 turned out to be for Mariana Oncology. After raising a $175 million series B in September of that year, the company was bought out by Novartis for $1 billion , plus $750 million in milestones. Originally known as Curie Therapeutics, Mariana emerged from stealth with a $75 series A raise in December 2021 to develop new radiopharmaceuticals. The company caught our eye and made the 2023 list, but apparently Novartis was keeping an eye too. The radiopharmaceutical-hungry Big Pharma said at the time of the deal that Mariana’s technology would help it double down in the field of radioligand therapeutics. On a recent earnings call, Novartis executives expressed their excitement in the Mariana technology, saying that candidates from the buyout are speeding toward the clinic and could go after well-established antibody-drug conjugate (ADC) targets. See the full class of 2023 . ___________________________________________ 2022 Eikon Therapeutics Status: Operating Eikon has spent the two years since landing on our list racking up cash and poaching executives from CEO Rodger Perlmutter’s former employer Merck. The biotech’s raises have been huge in a really tough market: a $148 million series A in May 2021, $518 million for a January 2022 series B and most recently, $106 million in series C financing in June 2023 . At the most recent raise, Eikon also announced the acquisition of a clinical pipeline. The company now boasts oncology assets in Phase I and II testing for non-small cell lung cancer and melanoma, respectively. The rest of Eikon’s pipeline is in preclinical research or discovery. See the full class of 2022 . ___________________________________________ 2021 EQRx Status: Bought by Revolution Medicines, assets terminated EQRx emerged with a lofty goal we could all get behind: develop drugs at a lower cost. It raised a cool $200 million in a July 2020 series A, then caught the special purpose acquisition company (SPAC) wave to the Nasdaq just over a year later, adding $1.8 billion in an August 2021 IPO. The company was helmed at first by serial biotech entrepreneur Alexis Borisy. Trouble emerged shortly thereafter, with EQRx cutting lead asset sugemalimab for NSCLC after determining it had no path to market. The company shifted to two other assets, aumolertinib and lerociclib, but later dropped the former. Several rounds of layoffs made the company ever smaller before EQRx finally gave up entirely. The assets were terminated as Revolution Medicines bought out the company and its hefty $1 billion bank account. Borisy has since gone on to start a venture capital firm called Curie.Bio . See the full class of 2021 . ___________________________________________ 2020 Cerevel Therapeutics Status: Bought by AbbVie Cerevel as a company is no more, but its medicines live on at AbbVie, which bought the biotech for $8.7 billion in December 2023. The deal was part of a trend of neuroscience-focused biotechs being bought up by Big Pharma, with Cerevel moving in the same month as Bristol Myers Squibb bought Karuna Therapeutics for $14 billion. BMS was rewarded quickly with a recent FDA approval for KarXT (now Cobenfy) for schizophrenia, but AbbVie too has had success since sealing the deal. Cerevel’s investigational Parkinson’s disease therapy tavapadon improved control of motor symptoms when used with common treatment levodopa in a Phase III trial that read out in April. The data would support a regulatory filing for the therapy, executives said at the time. Two additional late-stage readouts for the therapy are due later this year. But the real star of the deal was emraclidine, which is in Phase III studies for schizophrenia. Cerevel also brought a major depression treatment to the table. “These assets clearly will be great additions to our neuroscience franchise,” said AbbVie’s CEO Rob Michael on the company’s second quarter earnings call. See the full class of 2020 . ___________________________________________ 2019 Genevant Status: Operating One of Roivant’s offshoots, Genevant is still operating today, working on developing nucleic acid delivery technology. Just last week, Genevant signed a deal with CRISPR gene editing company Editas Medicines to develop in vivo gene editing medicines for two undisclosed targets. Genevant has also recently signed deals with Novo Nordisk, Tome Biosciences, Gritstone bio and Repair Biotechnologies. But perhaps the most compelling story from Genevant since we named them to our NextGen list has been their litigation with famed biotech Moderna over the lipid nanoparticle delivery system used for its approved COVID-19 vaccine. Genevant and Arbutus Biopharma filed several patent infringement claims against Moderna arguing that the vaccine developer used their technology without license or proper compensation. A judge sided with Arbutus and Genevant in April, with a trial set for next year. See the full class of 2019 . ___________________________________________ 2018 BlueRock Therapeutics Status: Bought by Bayer Bayer bought cell therapy biotech BlueRock Therapeutics in August 2019 for $1 billion, but rather than absorbing the company into itself, the German pharma kept the name and unit operating. This unusual but refreshing strategy means that the company essentially lives on. In June 2023, BlueRock reported that Parkinson’s disease stem cell therapy bemdaneprocel was well tolerated with no significant side effects in a Phase I trial with 12 patients. It was the first cell therapy to show positive results in human testing, Bayer said at the time. A September update showed similar safety results. The therapy has since received a regenerative medicine advanced therapy tag from the FDA. Elsewhere in the pipeline, BlueRock’s investigational iPSC-derived cell therapy OpCT-001 for certain retinal disorders was cleared for human testing in September. The therapy was licensed from FUJIFILM Cellular Dynamics and Opsis Therapeutics. See the full class of 2018 . ___________________________________________ 2017 Decibel Therapeutics Status: Bought by Regeneron Regeneron made a splash at this year’s American Society of Gene and Cell Therapy meeting in Baltimore when it showcased results of a Phase I/II trial of hearing loss therapy DB-OTO. The therapy restored hearing in two children who had a genetic form of hearing loss. While Regeneron’s branding was behind the conference presentation, the gene therapy came from Decibel Therapeutics. Regeneron bought the biotech for $109 million in August 2023, at which point the two companies had already been working together for six years. The biotech was integrated into Regeneron’s genetic medicines portfolio. See the full class of 2017 . ___________________________________________ 2016 CRISPR Therapeutics Status: Operating CRISPR Therapeutics made history last year as the first company, along with partner Vertex Pharmaceuticals, to gain an FDA approval for a CRISPR-based gene edited therapy. The companies were granted an FDA greenlight for Casgevy in sickle cell disease. The same day, bluebird bio got a similar nod for a competing gene therapy, though not one that involves CRISPR technology. The FDA approval was a triumph of modern biotechnology. But the roll out has been slow given the nature of gene therapy treatment, and only recently have patients begun receiving infusions of the medicine. Casgevy is now also approved for beta thalassemia, another blood disorder. The company has turned to the rest of its pipeline, including a CD19-directed CAR T cell program called CTX112, which is being tested in both cancer and autoimmune disorders. CTX131 is in development for hematological malignancies as well. CEO Sam Kulkarni has expressed confidence in CRISPR’s leading position in gene editing and pledged to be on top of emerging technologies. “One of the most important considerations for us is that we don’t get disrupted,” he said at the Goldman Sachs healthcare conference in June. “We are the leaders in the gene editing space and we have been, but we want to make sure that we’re looking at every improvement that’s coming out there.” See the full class of 2016 . ___________________________________________ 2015 Spark Therapeutics Status: Bought by Roche Like BlueRock, Spark Therapeutics was folded into a Big Pharma but kept its identity. Spark earned a spot on our very first NextGen list in 2015 thanks to its active clinical hemophilia program and fundraises totaling $122.8 million. The company caught the eye of Roche, which swooped in with a $4.8 billion bid in February 2019. The deal eventually turned into a bit of a boondoggle, with regulatory delays slowing things down until it finally closed that December. At issue was Roche’s hemophilia Hemlibra. Regulators worried that Spark’s hemophilia program and Hemlibra together could stifle competition and create a monopoly. Spark brought with it the approved eye disease gene therapy Luxturna. With the deal closed, Spark has charged forward under the Roche banner. The unit is developing a 500,000-square foot Gene Therapy Innovation Center in Philadelphia as its flagship manufacturing space. See the full class of 2015 .

Editas Medicine is ousting its ex vivo sickle cell and beta thalassemia gene therapy to instead focus on an in vivo treatment for the diseases. The company announced Tuesday that it’s seeking to partner or out-license its lead clinical program, known as reni-cel. The press release also detailed the gene editing company’s new and ambitious focus to develop an in vivo treatment for sickle cell disease and beta thalassemia, and divulged some initial results for the therapy in mice. During an analyst call on Tuesday morning, Editas CEO Gilmore O’Neill said the company is not yet sharing a timeline for when it plans to begin human studies with the in vivo therapy. To date, no in vivo gene editing treatments for sickle cell disease or thalassemia have been studied in clinical trials. “We believe there are several beneficial financial differences versus making ex vivo autologous cell therapies,” Editas CFO Erick Lucera said during the call. “Among them is our belief that in vivo medicines will provide larger market opportunities and do so with lower cost of goods and lower clinical trial costs.” Editas’ pivot comes as the companies that brought to market two sickle cell gene therapies, which require a patient’s blood stem cells to be extracted and then returned to their body, have seen slow commercial launches. While Vertex and bluebird bio have said that the launches will take time, the broader cell and gene therapy field has also seen cooling interest from investors and pharma companies. And bluebird bio, which is solely focused on gene therapies, has been forced to renegotiate a loan, lay off staff and cut costs in recent months. Editas would have been a distant third-to-market with reni-cel, which uses gene editing to modify blood stem cells that are taken from the patient so they make fetal hemoglobin. The edited cells are then infused back into the patient. Beam Therapeutics is also developing a base-edited ex vivo sickle cell gene therapy. “Investors had grown increasingly skeptical and cautious on reni-cel’s commercial prospects,” Stifel analyst Dae Gon Ha told investors on Tuesday. Editas’ shares $EDIT fell about 8% Tuesday morning. The shift to in vivo gene editing marks yet another pivot for Editas, which was founded in 2013 to develop new therapies from CRISPR technology. The company had been working on a treatment for a rare inherited form of blindness, as well as cancer programs, before shifting its focus in 2023 to its ex vivo sickle cell disease and beta thalassemia therapy, which commanded a larger market than the rare form of blindness it had been working on. The in vivo approach would deliver a gene editor using a fatty acid envelope targeted at cells outside the liver to induce fetal hemoglobin expression in blood stem cells directly in the body. Typically, these fatty acid particles called LNPs like to accumulate in the liver, and delivery beyond the liver has been a white whale for genetic medicine developers. In mice engrafted with human blood stem cells, Editas said that researchers observed 29% editing efficiency with a single dose of therapy, and that approximately 20% of fetal hemoglobin-expressing red blood cells populated the host at one month. A handful of other companies, including Tessera Therapeutics , are also trying to develop in vivo gene editing treatments for sickle cell disease. The company also announced on Monday that it signed a non-exclusive license deal with Genevant Sciences for LNP technology for two undisclosed targets. The agreement would give Genevant up to $238 million in upfront and milestone payments plus royalties. Editas declined to disclose the upfront payment of the deal.

Achieved in vivo preclinical proof of concept of hematopoietic stem and progenitor cell editing by utilizing Editas Medicine’s proprietary targeted LNP as a key step forward toward developing a novel in vivo treatment for sickle cell disease and beta thalassemia Initiated process to partner or out-license reni-cel to focus resources on in vivo pipeline development Company to present data and discuss strategic update in a Company-sponsored Webinar today at 8:00 a.m. ET CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today announced its achievement of in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells. The Company observed high levels of editing of the HBG1/2 promoter, leveraging our clinically validated upregulation strategy, utilizing a novel and Editas-proprietary targeted lipid nanoparticle (tLNP) formulation for extrahepatic tissue delivery. The Company is also providing business development and financial updates, including that it initiated a process to partner or out-license reni-cel. “Achieving in vivo preclinical proof of concept in a desired, extrahepatic target cell type, delivered utilizing a proprietary Editas LNP that works outside the liver, puts us on a clear path to develop a potentially first- and best-in-class in vivo gene edited medicine for the treatment of sickle cell disease and beta thalassemia,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. “I am delighted with our concrete progress on in vivo functional upregulation as we drive towards our long-term vision to be a leader in in vivo programmable gene editing, specifically achieving in vivo preclinical proof of concept ahead of schedule.” Dr. O’Neill added, “Simultaneously, we are laser focused on the optimal use of capital as we continue development of reni-cel, invest in our in vivo pipeline and vision, and map to the future. As part of that focus, we frequently evaluate opportunities to most efficiently advance reni-cel, a potentially best-in-class cell therapy for the treatment of sickle cell disease and beta thalassemia, towards commercialization and ultimately deliver it to patients in need. We believe that the best option for both patients and our shareholders is for us to seek an alternative such as a global partner or out-licensing, which would allow for further development and ultimately commercialization of reni-cel with or by another party and would allow us to substantially reduce spend in 2025.” In Vivo Proof of Concept Data Highlights Achieved highly competitive level of in vivo hematopoietic stem and progenitor cell (HSPC) editing utilizing a novel, Editas-proprietary targeted lipid nanoparticle (t-LNP) for extrahepatic tissue delivery in a humanized mouse model, mice engrafted with human hematopoietic stem cells.Enabled an editing level of 29% in HSPCs after a single dose by utilizing a hematopoietic stem cell targeting strategy and tLNP formulation optimization.Further demonstrated that editing with the Company’s proprietary tLNP formulation resulted in the functional outcome of HbF induction, indicated by the presence of HbF expressing human red blood cells (on average 20%) that populate in the host by one month. This level of in vivo editing in a humanized mouse model after a single dose constitutes a highly competitive dataset relative to data in the public domain for the development of an in vivo medicine for sickle cell disease and beta thalassemia. Ex Vivo Hemoglobinopathies Reni-cel (renizgamglogene autogedtemcel, previously EDIT-301) for Severe Sickle Cell Disease (SCD) On-track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up in the RUBY trial at the American Society of Hematology (ASH) Annual Meeting and Exposition, December 7-10, 2024.As previously disclosed, the Company has completed enrollment of the adolescent and adult cohorts of the Phase 1/2/3 RUBY trial for SCD.Manufacturing drug product for the initial adolescent cohort patients.The Company continues to dose adult patients in the RUBY trial and has dosed 28 patients to date. Reni-cel for Transfusion-dependent Beta Thalassemia (TDT) On-track to present additional clinical data from the EdiTHAL trial by year-end 2024.The Company completed enrollment of the adult cohort of the EdiTHAL trial for TDT and continues to dose patients. Other Corporate Highlights On October 3, 2024, Editas Medicine announced the sale of certain future license fees and other payments owed to the Company under its Cas9 license agreement with Vertex Pharmaceuticals to a wholly owned subsidiary of DRI Healthcare Trust (DRI) for an upfront cash payment of $57 million. The upfront cash payment brings non-dilutive capital to Editas Medicine, helping enable further pipeline development and related strategic priorities.The Company ended the third quarter 2024 with approximately $265 million of cash, cash equivalents, and marketable securities, or approximately $320 million following receipt of the upfront cash payment from DRI.The Company has engaged Moelis & Company LLC, a leading global independent investment bank, to lead the global process to partner or out-license reni-cel. In light of this webinar, the Company does not plan to host a conference call when it announces third quarter 2024 financial results next month. Webinar Presentation Details: The live and archived webcast of the Company’s webinar presentation will be accessible through this webcast link, or through the Events & Presentations page of the “Investors” section of the Company’s website. A replay of the webinar will be available upon conclusion of the webinar in the Investors section of the Editas Medicine website at https://www.editasmedicine.com/. About renizgamglogene autogedtemcel (reni-cel)Reni-cel, formerly known as EDIT-301, is an experimental gene editing medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Reni-cel consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by AsCas12a, a novel, proprietary, highly efficient, and specific gene editing nuclease. Red blood cells derived from reni-cel CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT. About Editas MedicineAs a clinical-stage gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com. Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Company’s intent to partner or out-license reni-cel and any benefits resulting therefrom, the initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including presenting additional clinical data from the RUBY trial at the ASH Annual Meeting and Exposition and from the EdiTHAL trial by year-end 2024, the potential of, and expectations for, the Company’s product candidates, including any in vivo gene edited medicines the Company may develop, and the Company’s expectations regarding cash runway. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials, including the RUBY and EdiTHAL trials, and clinical development of the Company’s product candidates, including reni-cel; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from preclinical studies and clinical trials will be predictive of the final results of the study or trial or the results of any future clinical trials; expectations for regulatory approvals to conduct trials or to market products; and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference in this press release.