Betibeglogene autotemcel

If Editas Medicine’s CRISPR gene-editing therapy for rare blood diseases reaches the market, it will be in the hands of a different company. The biotech on Tuesday announced plans to partner or out-license that clinical-stage ex vivo therapy, choosing instead to focus its resources on in vivo R&D that now has preclinical proof-of-concept data. The in vivo therapy, renizgamglogene autogedtemcel (reni-cel), has reached late-stage clinical development for severe sickle cell disease. Reni-cel is also in early-stage development for transfusion-dependent beta thalassemia. Patients who have these rare blood disorders already have ex vivo gene-editing treatment options. Last winter Vertex Pharmaceuticals’ Casgevy landed FDA approvals for sickle cell disease and beta thalassemia. Bluebird Bio’s last winter received FDA approval for Lygenia for sickle cell disease. A different Bluebird gene-editing therapy named Zynteglo won FDA approval in 2022 for beta thalassemia. Editas’s reni-cel would be third to market, but the Cambridge, Massachusetts-based company has said it could be best in the class of ex vivo therapies, made by editing a patient’s hematopoietic stem cells in a lab and infusing those cells back into the patient. These modified cells are intended to correct the anemia caused by these blood disorders and increase levels of fetal hemoglobin. presented by Sponsored Post The Promise of Value-Based Care and MedTech Innovation Monica Vajani, Executive Director for mHUB’s MedTech Accelerator, discusses how mHUB is helping innovators transition healthcare towards value-based care. By Monica Vajani - mHUB Dosing is continuing in reni-cel’s pivotal sickle cell study with 28 adult patients dosed to date; dosing is being scheduled for adolescent patients. Editas said data will be presented during the upcoming American Society of Hematology meeting in December. The beta thalassemia study is on track to present clinical data by the end of this year. Speaking during a Tuesday webcast, Editas CEO Gilmore O’Neill said the company is mindful of the financial cost that comes with commercializing an ex vivo autologous therapy. Placing reni-cel in the hands of another company would allow Editas to substantially reduce its 2025 spending. “We have previously said that we would be open to partnering reni-cel, but now, with the progress we’re making on in vivo editing, we are actively undertaking a process to partner or out-license reni-cel to most effectively drive it to commercialization while allowing us to apply our full attention and capital to in vivo medicines,” O’Neill said. “The in vivo data we are sharing today makes us even more confident in this decision.” The data that has Editas excited show proof of concept for its in vivo editing approach in mouse models for both sickle cell disease and beta thalassemia. Specifically, the company reported its in vivo therapy enabled an editing level of 29% in hematopoietic stem cells after a single dose. This treatment led to fetal hemoglobin induction (HbF), indicated by the presence of HbF-expressing human red blood cells populating in the host by one month. O’Neill said this level of in vivo editing in mice after a single dose is “highly competitive” compared to data in the public domain for the development of an in vivo medicine for sickle cell disease and beta thalassemia. presented by Health Tech What’s Keeping Healthcare CIOs Up at Night: How to Improve Patient Satisfaction by Handling Calls More Efficiently Health systems have spent billions on portals while investments in modernizing the voice channel — the dominant preference of healthcare consumers — have taken a backseat. By Stephanie Baum In a note sent to investors, Leerink Partners analyst Mani Foroohar said the decision to seek an external partner for reni-cel makes sense given the cost of commercializing autologous ex vivo therapies. The near-term focus for Editas shares is the additional reni-cel data and the extent to which the company can show differentiation for the therapy to drive business development interest in this program. Editas said it has engaged investment bank Moelis & Company to lead the global process to partner or out-license reni-cel. Meanwhile, the company has secured non-dilutive financing to support its in vivo gene-editing focus. Earlier this month, Editas sold to DRI Healthcare Trust certain future license fees and payments owed under a Vertex Pharmaceuticals’ licensing agreement for Cas9 gene-editing technology. The DRI deal paid Editas $57 million up front. Editas reported its cash position as of the end of the third quarter of this year was about $265 million; or about $320 million including the DRI payment. O’Neill said the company has enough capital to fund operations into 2026. Public domain image by Flickr user NIH Image Gallery

Job reductions come shortly after bluebird bio, in its delayed annual securities filing, warned that it may not have enough financial resources to continue operating for a year. After bluebird bio acknowledged troubles keeping its business operational, the gene therapy biotech is launching a restructuring that will reduce the size of its workforce by about 25%.The overhaul, unveiled Tuesday, is designed to reduce bluebird’s cash operating expenses by about 20% and to help the company break even on quarterly cash flows in the second half of 2025. The layoff round means that nearly 100 bluebirds will have to leave the nest based on a head count of 375 full-time employees as of the end of June. Although cuts are being made across the board, R&D and general administration staffers will bear the brunt of the restructuring so that the company can focus its spending on commercial activities, bluebird Chief Financial Officer James Sterling said on an investor call Tuesday.  “The decision to reduce our workforce in support of a more focused set of priorities was made following a detailed review of the needs and capabilities of our organization,” bluebird CEO Andrew Obenshain said in a statement Tuesday.The move comes shortly after bluebird, in its delayed annual securities filing, again warned that there is “substantial doubt regarding the company’s ability to continue as a going concern,” an accounting term for when a company may not have enough financial resources to continue operating for a year. The company has flagged that risk for a couple years now.Based on a cash balance of about $193 million, including $49 million in restricted cash, as of the end of June, bluebird in August said it expects to fund its operations into the second quarter of 2025. Sterling on Tuesday confirmed that cash runway.Since its inception in 1992, bluebird has been focused on potentially curative gene therapies for rare diseases. While the company has launched three commercial gene therapies—Lyfgenia, Skysona and Zynteglo—it has never been profitable, excluding the sale of its FDA priority review vouchers. As of the end of 2023, bluebird’s accumulated deficit had reached $4.3 billion.Uptake of its gene therapies has so far been limited. During its second-quarter report in August, bluebird disappointed investors after narrowing its 2024 expectations to just about 85 patient starts, the lower end of its previous guidance. So far, bluebird has initiated treatment for 41 patients across its portfolio, up from 27 in mid-August. It expects about 40 new patient starts in the last three months of the year.Lyfgenia, the sickle cell gene therapy approved by the FDA in December 2023, is not showing any commercial edge over Vertex and CRISPR Therapeutics’ rival product Casgevy despite bluebird’s experience with gene therapies.For its cash flow break-even target, bluebird assumes it can achieve about 40 drug product deliveries per quarter. After that, the company has plans to double capacity for Lyfgenia in 2026 in anticipation of a similar launch ramp-up as the beta thalassemia gene therapy Zynteglo, bluebird’s chief commercial and operating officer Tom Klima said on Tuesday’s call.By bluebird’s estimate, about 20,000 sickle cell patients in the U.S. could be eligible for gene therapy, versus about 1,000 patients for Zynteglo. The company only records revenue upon product delivery, not when patients give their cells to start the treatment journey. The gap between those two time points ranges from about four months to five months.“This timeline underscores that converting starts into deliveries in cash and ultimately revenue is really an element of time as we execute our plan in these early phases of launch,” Klima said.Cost reductions and potential sales increases may still not be enough for bluebird to break even, as the biotech is separately moving to raise additional cash. During Tuesday’s call, Sterling declined to disclose how much money the company is seeking.Sterling pointed to bluebird’s recently amended loan agreement with Hercules Capital. Under the updated deal, bluebird can draw $25 million if it secures more than $75 million in gross cash proceeds from financing transactions elsewhere by Dec. 20, 2024. Another tranche of $25 million is available if bluebird can collect $100 million from other financing deals by that time or more than $125 million by June 30, 2025. Certain Lyfgenia patient starts metrics are also conditions for a Hercules capital infusion.Nevertheless, the CFO said, “that’s not necessarily the cash we’re seeking, nor do we necessarily feel we need those tranches to get there.” Bluebird has repeatedly faced challenges as the entire gene therapy field has charted a rocky commercial path. Back in 2022, the company reduced its workforce by 30% and backed out of the European market.“While we face the trials and tribulations of being a pioneer, first in R&D and now commercially, we believe we are well on our way to showing that a standalone gene therapy company can achieve financial sustainability,” Obenshain said on Tuesday’s call.Editor's Note: The story was updated with additional context around bluebird bio's "going concern" warning.