Latest news with #EditasMedicine
Business Upturn
4 hours ago
- Business
- Business Upturn
Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June
Achieved 58% mean editing at five months after a single dose using high efficiency HSC delivery, demonstrating therapeutically relevant editing levels using a clinically validated strategy. Achievement supports development of a novel, in vivo approach to treating sickle cell disease and beta thalassemia. CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today shared new in vivo data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) with a single dose of proprietary targeted lipid nanoparticle (tLNP) in non-human primates (NHPs). This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for the treatment of sickle cell disease and beta thalassemia. The Company reported these data in a presentation available today and will detail the data in a poster session on Saturday, June 14th 6:30 – 7:30 p.m. CEST (12:30 – 1:30 p.m. EDT) at the European Hematology Association (EHA) 2025 Congress in Milan, Italy. In this study, the Company's proprietary tLNP formulation delivered HBG1/2 promoter editing cargo to HSCs in NHPs. Latest data from this ongoing NHP study showed that at five months a single intravenous administration of Editas' tLNP resulted in mean on-target editing levels in the HBG1/2 promoter region of 58% in HSCs: well exceeding the predicted editing threshold of ≥25% required for therapeutic benefit. In addition to achieving therapeutically relevant editing levels, the biodistribution data in NHPs with Editas' tLNP continue to show significant de-targeting of the liver in contrast to standard LNPs. 'These data from our in vivo HSC program confirm our ability to achieve high efficiency delivery, therapeutically relevant editing levels and favorable biodistribution in NHPs. These data validate the further development of Editas' proprietary HSC-tLNP for editing of the HBG1/2 promoters for the treatment of sickle cell disease and beta thalassemia,' said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. Editas Medicine's in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to edit with high efficiency and minimize off-target editing. Editing the HBG1/2 promoters with AsCas12a with the investigational medicine reni-cel led to robust increases in HbF and total hemoglobin (Hb) in clinical trials. The presentation details are listed below. Abstracts can be accessed on the EHA website , and the presentation will be posted on the Editas Medicine website during the conference. Poster Presentation Details: Title: Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for β-hemoglobinopathies Date/Time: Saturday, June 14, 2025, 6:30 – 7:30 p.m. CEST/ 12:30 – 1:30 p.m. EDT Location: Allianz MiCo, Milano Convention Centre Session: Poster Session 2 About Editas Medicine As a pioneering 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 in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision in vivo gene editing 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 . Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same.
Yahoo
5 hours ago
- Business
- Yahoo
Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June
Achieved 58% mean editing at five months after a single dose using high efficiency HSC delivery, demonstrating therapeutically relevant editing levels using a clinically validated strategy. Achievement supports development of a novel, in vivo approach to treating sickle cell disease and beta thalassemia. CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today shared new in vivo data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) with a single dose of proprietary targeted lipid nanoparticle (tLNP) in non-human primates (NHPs). This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for the treatment of sickle cell disease and beta thalassemia. The Company reported these data in a presentation available today and will detail the data in a poster session on Saturday, June 14th 6:30 - 7:30 p.m. CEST (12:30 – 1:30 p.m. EDT) at the European Hematology Association (EHA) 2025 Congress in Milan, Italy. In this study, the Company's proprietary tLNP formulation delivered HBG1/2 promoter editing cargo to HSCs in NHPs. Latest data from this ongoing NHP study showed that at five months a single intravenous administration of Editas' tLNP resulted in mean on-target editing levels in the HBG1/2 promoter region of 58% in HSCs: well exceeding the predicted editing threshold of ≥25% required for therapeutic benefit. In addition to achieving therapeutically relevant editing levels, the biodistribution data in NHPs with Editas' tLNP continue to show significant de-targeting of the liver in contrast to standard LNPs. 'These data from our in vivo HSC program confirm our ability to achieve high efficiency delivery, therapeutically relevant editing levels and favorable biodistribution in NHPs. These data validate the further development of Editas' proprietary HSC-tLNP for editing of the HBG1/2 promoters for the treatment of sickle cell disease and beta thalassemia,' said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. Editas Medicine's in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to edit with high efficiency and minimize off-target editing. Editing the HBG1/2 promoters with AsCas12a with the investigational medicine reni-cel led to robust increases in HbF and total hemoglobin (Hb) in clinical trials. The presentation details are listed below. Abstracts can be accessed on the EHA website, and the presentation will be posted on the Editas Medicine website during the conference. Poster Presentation Details: Title: Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for β-hemoglobinopathies Date/Time: Saturday, June 14, 2025, 6:30 - 7:30 p.m. CEST/ 12:30 – 1:30 p.m. EDT Location: Allianz MiCo, Milano Convention Centre Session: Poster Session 2 About Editas Medicine As a pioneering 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 in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision in vivo gene editing 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 CONTACT: Media and Investor Contacts: media@ ir@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
25-05-2025
- Business
- Yahoo
1 Stock Down 97% That Could Double, According to Wall Street
Editas Medicine focuses on developing gene-editing treatments. The struggling biotech recently gave up on its leading pipeline candidate. Even if it matches Wall Street's estimates in the next year, the stock is too risky. 10 stocks we like better than Editas Medicine › Over the past few years, investors have moved away from somewhat speculative and unprofitable companies to put their money into safer, steadier investments. Editas Medicine (NASDAQ: EDIT), a gene-editing-focused clinical-stage biotech, is firmly in the speculative camp, which is why its shares are down by 97% since early 2021. The stock is trading for about $1.50 right now. However, its average price target of $3.38 (according to Yahoo! Finance) implies a potential upside of about 125%. Should investors scoop up the company's shares expecting them to soar? Developing and marketing gene-editing therapies is challenging, as Editas Medicine knows well. The company's previous leading program was called reni-cel. It was being tested as a potential treatment for two rare blood disorders: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Here is the problem: Ex vivo gene-editing therapies are complex to administer. The process requires collecting patients' cells, editing them, and reinserting them into the patients. Even going through the clinical trial phase for a treatment of this kind is more expensive than if it were a simple oral pill. Though reni-cel was making progress and showing strong signs of efficacy with the patients who had received treatment, Editas Medicine decided to discontinue its development because it couldn't find a commercial partner with big pockets. Even before that, though, Editas Medicine was fighting an uphill battle. Between 2022 and 2023, three gene-editing therapies were approved in the U.S.: Zynteglo, Lyfgenia, and Casgevy. Zynteglo and Lyfgenia treated TDT and SCD, respectively, while Casgevy targeted both. Editas Medicine's relatively slow progress with reni-cel didn't bode well with investors, considering there were already competing therapies on the market. The company's decision to discontinue this program was the right one -- the move hardly makes the stock particularly attractive, but trying to push reni-cel toward commercialization despite its slow progress and the competitive landscape would have been prohibitively expensive. Editas Medicine has decided to pivot toward developing in vivo gene-editing therapies. Unlike the ex vivo variety, these kinds are administered via injection into the body of therapeutic genes -- so there is no need to collect the patients' cells. Editas Medicine has partners for some of its in vivo programs. It is working on some medicines with Bristol Myers Squibb, a leading pharmaceutical company. Further, Editas Medicine has decreased expenses and costs thanks to discontinuing the development of reni-cel and laying off a sizable portion of its workforce. It expects its cash and equivalents balance of $221 million as of the end of the first quarter to keep it afloat until the second quarter of 2027. With a stock price of $1.50, it wouldn't be too surprising to see this company more than double in value in the next year, perhaps because of progress with an early-stage clinical program, or licensing deals for one of its medicines, or buyout interest from a larger drugmaker. However, Editas Medicine is far too risky a stock for long-term investors. Its current candidates haven't even started human clinical trials yet. It will be years before any get close to approval. It's also worth pointing out that Editas Medicine gave up the development of EDIT-101 and EDIT-103, two potential gene-editing therapies for various eye diseases, also because it failed to find a commercial partner. Given the biotech's poor track record and the current state of its operations, investing in the stock right now and expecting it to generate strong returns over the long run wouldn't be that different from gambling. Before you buy stock in Editas Medicine, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Editas Medicine wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $639,271!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $804,688!* Now, it's worth noting Stock Advisor's total average return is 957% — a market-crushing outperformance compared to 167% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 19, 2025 Prosper Junior Bakiny has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Bristol Myers Squibb. The Motley Fool recommends Editas Medicine. The Motley Fool has a disclosure policy. 1 Stock Down 97% That Could Double, According to Wall Street was originally published by The Motley Fool Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Globe and Mail
25-05-2025
- Business
- Globe and Mail
1 Stock Down 97% That Could Double, According to Wall Street
Over the past few years, investors have moved away from somewhat speculative and unprofitable companies to put their money into safer, steadier investments. Editas Medicine (NASDAQ: EDIT), a gene-editing-focused clinical-stage biotech, is firmly in the speculative camp, which is why its shares are down by 97% since early 2021. The stock is trading for about $1.50 right now. However, its average price target of $3.38 (according to Yahoo! Finance) implies a potential upside of about 125%. Should investors scoop up the company's shares expecting them to soar? Where to invest $1,000 right now? Our analyst team just revealed what they believe are the 10 best stocks to buy right now. Continue » Editas Medicine's challenges Developing and marketing gene-editing therapies is challenging, as Editas Medicine knows well. The company's previous leading program was called reni-cel. It was being tested as a potential treatment for two rare blood disorders: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Here is the problem: Ex vivo gene-editing therapies are complex to administer. The process requires collecting patients' cells, editing them, and reinserting them into the patients. Even going through the clinical trial phase for a treatment of this kind is more expensive than if it were a simple oral pill. Though reni-cel was making progress and showing strong signs of efficacy with the patients who had received treatment, Editas Medicine decided to discontinue its development because it couldn't find a commercial partner with big pockets. Even before that, though, Editas Medicine was fighting an uphill battle. Between 2022 and 2023, three gene-editing therapies were approved in the U.S.: Zynteglo, Lyfgenia, and Casgevy. Zynteglo and Lyfgenia treated TDT and SCD, respectively, while Casgevy targeted both. Editas Medicine's relatively slow progress with reni-cel didn't bode well with investors, considering there were already competing therapies on the market. The company's decision to discontinue this program was the right one -- the move hardly makes the stock particularly attractive, but trying to push reni-cel toward commercialization despite its slow progress and the competitive landscape would have been prohibitively expensive. Is there any hope for the stock? Editas Medicine has decided to pivot toward developing in vivo gene-editing therapies. Unlike the ex vivo variety, these kinds are administered via injection into the body of therapeutic genes -- so there is no need to collect the patients' cells. Editas Medicine has partners for some of its in vivo programs. It is working on some medicines with Bristol Myers Squibb, a leading pharmaceutical company. Further, Editas Medicine has decreased expenses and costs thanks to discontinuing the development of reni-cel and laying off a sizable portion of its workforce. It expects its cash and equivalents balance of $221 million as of the end of the first quarter to keep it afloat until the second quarter of 2027. With a stock price of $1.50, it wouldn't be too surprising to see this company more than double in value in the next year, perhaps because of progress with an early-stage clinical program, or licensing deals for one of its medicines, or buyout interest from a larger drugmaker. However, Editas Medicine is far too risky a stock for long-term investors. Its current candidates haven't even started human clinical trials yet. It will be years before any get close to approval. It's also worth pointing out that Editas Medicine gave up the development of EDIT-101 and EDIT-103, two potential gene-editing therapies for various eye diseases, also because it failed to find a commercial partner. Given the biotech's poor track record and the current state of its operations, investing in the stock right now and expecting it to generate strong returns over the long run wouldn't be that different from gambling. Should you invest $1,000 in Editas Medicine right now? Before you buy stock in Editas Medicine, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Editas Medicine wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $639,271!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $804,688!* Now, it's worth noting Stock Advisor 's total average return is957% — a market-crushing outperformance compared to167%for the S&P 500. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of May 19, 2025
Yahoo
16-05-2025
- Business
- Yahoo
A new CFO tasked with taking ‘scrappy' Editas from last year's layoffs into new gene editing territory
This story was originally published on PharmaVoice. To receive daily news and insights, subscribe to our free daily PharmaVoice newsletter. Welcome to First 90 Days, a series dedicated to examining how pharma executives and other leaders are planning for success in their new roles. Today, we're speaking to Amy Parison, CFO at Editas Medicine, which is undergoing a shift in focus to in vivo therapies and navigating the many challenges of the gene editing space. Gene editing biotech Editas Medicine is no stranger to the drug development pivot. And along with a major reorganization and scientific focal shift, the company has put its financial decisionmaking in the hands of a first-time CFO while it gears up for the next phase. After a cost-cutting shuffle announced at the end of last year in which the company slashed roughly 65% of its workforce, Editas switched gears from an ex vivo gene editing platform to in vivo technology. The change moves the therapeutic process from an external procedure in a lab to one that's delivered directly into a patient. Editas' previous lead ex vivo candidate in sickle cell anemia wasn't a clinical failure. Rather, the drug, called reni-cel, suffered from a lack of a commercial partner, leading to a cash crunch at Editas. Now back to the drawing board, the company is focusing on preclinical candidates using hematopoietic stem cells in diseases like sickle cell and beta thalassemia as well as liver-directed gene editing, all with the more convenient and practical application of in vivo delivery. Navigating the transition is newly minted CFO Amy Parison who, after working as head of finance alongside the company's former CFO, took the reins at the end of March. "In biotech, you grow numb to operating at a loss. You're thinking more about cash runway and how to extend that." Amy Parison CFO, Editas Medicine Tasked with launching into the company's new focus, Parison looks at Editas as a 'scrappy' biotech willing to change its priorities to match the moment. Gene editing has long been a tricky area both in terms of the complex science and the financial calculus it takes to pull it off. Here, we spoke to Parison about Editas' unique position in the gene editing space, what lies ahead for a CFO managing a platform shift while maintaining a cash runway, and some of the potential political and financial snags the company could face as it heads in this new direction. This interview has been edited for brevity and style. AMY PARISON: What jumps out to me is our science and what we're showing in our data, and even with former programs, what we've done is special. There's a lot of potential, and I'm excited [about] how we've pivoted to in vivo [gene editing]. That's the future of gene editing and where it's going to have the biggest impact. A few months before the transition, I read an article about how women are well-positioned to be great CFOs even though it's been such a male-dominated field — it's about forming connections. I've seen that finance touches everything, that we're the lifeblood of the company. Beyond what people traditionally think of as the number crunching and investor outreach, what I'm excited about is being a part of the whole company and thinking strategically about not only their finances but everything they're doing. In biotech, you grow numb to operating at a loss. You're thinking more about cash runway and how to extend that. So while I was trained as an accountant, my financial acumen and strategy overpowers traditional accounting. My predecessor [former Editas CFO Erick Lucera], who set me to take the reins, instilled in me that the best cash is the cash you have. That's how I think about expenses and operating where we are, especially in these markets — being scrappy and capital efficient and making sure we're producing the best science with the best capital we have. Scrappy is being resourceful and rolling up your sleeves to do things that aren't necessarily in your job description or even where you've had formal training, but something you're excited about and committed to seeing through to the end. That's super important for biotech success. We don't have the luxury of a large headcount or huge infrastructure or resources, but that's motivating. The people who are scrappy are generally the ones who are motivated to move forward. There are lots of different flavors of gene editing. We can look at ex vivo gene editing, and Editas did have a phase 3 program. But there are lots of ways to deliver gene editing therapy — and we are moving from ex vivo to in vivo, and that's where the industry is at with gene editing. It's a fairly new technology, and so I think people need to understand how it works and how to think about it. There's been a lot of political uncertainty over the past few months, and we're all digesting what's being said and how to take that forward. It's been challenging to interpret that type of narrative and figure out what to do with it. So far, we haven't seen any ramifications of that rhetoric, and while the administration is fairly new in their tenure, once we understand what is being said or not being said, I think things will start to level out. We're always looking to the future. And while these are early-stage discussions for us, we're fortunate that we've seen companies like Vertex [Pharmaceuticals] and Bluebird [Bio] price their therapies on the market. There's a balance to figuring out the pricing for these therapies that can be a one-and-done treatment. I'm looking forward to thinking more about that, and we're using that as a guidepost. As the industry matures, we'll be part of it and mature with it. Recommended Reading Why layoffs are not the answer to biopharma's troubled market Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
