Editas Medicine Announces Strategic Transition to in vivo Gene Editing Company with Intent to Achieve Human Proof of Concept in Approximately Two Years
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 (EDIT) ha annunciato una transizione strategica per concentrarsi esclusivamente su medicine editate con CRISPR in vivo, a seguito di recenti progressi negli studi preclinici. L'azienda ha raggiunto circa il 40% di editing del sito promotore HBG1/2 utilizzando il proprio sistema di consegna a nanoparticelle lipidiche mirate, con un risultato del 20% di globuli rossi umani esprimenti HbF. Hanno anche dimostrato un'editing del fegato ad alta efficienza in primati non umani.
Come parte di questa transizione, Editas sta interrompendo lo sviluppo di reni-cel e implementando misure significative per ridurre i costi, compresa una riduzione della forza lavoro del 65% nei prossimi sei mesi. Sono stati annunciati diversi cambiamenti nella gestione, tra cui la partenza del Direttore Medico Baisong Mei e di due membri del consiglio. Si prevede che la ristrutturazione estenderà la liquidità dell'azienda fino al secondo trimestre del 2027.
Editas Medicine (EDIT) anunció una transición estratégica para centrarse exclusivamente en medicamentos editados con CRISPR in vivo, tras los recientes avances en estudios preclínicos. La compañía logró aproximadamente un 40% de edición del sitio promotor HBG1/2 usando su sistema de entrega de nanopartículas lipídicas dirigidas, lo que resultó en un 20% de glóbulos rojos humanos expresando HbF. También demostraron una edición hepática de alta eficiencia en primates no humanos.
Como parte de esta transición, Editas está finalizando el desarrollo de reni-cel e implementando medidas significativas de ahorro de costos, incluyendo una reducción del 65% en la fuerza laboral en los próximos seis meses. Se anunciaron varios cambios en la dirección, incluyendo la salida del Director Médico Baisong Mei y de dos miembros del consejo. Se espera que la reestructuración prolongue el capital disponible de la empresa hasta el segundo trimestre de 2027.
Editas Medicine (EDIT)는 최근 전임상 연구의 돌파구에 따라 CRISPR 편집 약물에 대한 집중을 위해 전략적 전환을 발표했습니다. 이 회사는 독자적인 표적 지질 나노입자 전달 시스템을 사용하여 HBG1/2 프로모터 부위의 약 40% 편집을 달성했으며, 그 결과 20%의 HbF를 표현하는 인간 적혈구를 생성했습니다. 그들은 또한 비인간 영장류에서 높은 효율의 간 편집을 입증했습니다.
이 전환의 일환으로 Editas는 reni-cel의 개발을 종료하고, 향후 6개월 동안 65%의 인력 감축을 포함한 상당한 비용 절감 조치를 시행하고 있습니다. 경영진의 변화가 발표되었으며, 여기에는 최고 의료 책임자 Baisong Mei와 두 명의 이사회의 퇴임이 포함됩니다. 이 구조조정은 2027년 2분기까지 회사의 자금 수명을 연장할 것으로 예상됩니다.
Editas Medicine (EDIT) a annoncé une transition stratégique pour se concentrer exclusivement sur les médicaments modifiés par CRISPR in vivo, à la suite de récentes percées dans des études précliniques. L'entreprise a réussi à obtenir environ 40% de modification du site promoteur HBG1/2 en utilisant son propre système de livraison par nanoparticules lipidiques ciblées, ce qui a entraîné 20% de globules rouges humains exprimant HbF. Ils ont également démontré une édition hépatique à haute efficacité chez les primates non humains.
Dans le cadre de cette transition, Editas met fin au développement de reni-cel et met en œuvre des mesures significatives d'économies, notamment une réduction de 65% de son effectif dans les six mois à venir. Plusieurs changements de direction ont été annoncés, y compris le départ du directeur médical Baisong Mei et de deux membres du conseil d'administration. On s'attend à ce que la restructuration prolonge la période de liquidités de l'entreprise jusqu'au deuxième trimestre 2027.
Editas Medicine (EDIT) hat einen strategischen Übergang angekündigt, um sich ausschließlich auf in vivo CRISPR-bearbeitete Medikamente zu konzentrieren, nachdem es jüngste Durchbrüche in präklinischen Studien gegeben hat. Das Unternehmen erzielte eine Bearbeitungsrate von etwa 40% des HBG1/2-Promotorbereichs unter Verwendung seines proprietären zielgerichteten Lipidnanopartikel-Abgabesystems, was zu 20% HbF-exprimierenden menschlichen roten Blutkörperchen führte. Sie demonstrierten auch eine hoch effiziente Leberbearbeitung bei nicht-menschlichen Primaten.
Im Rahmen dieses Übergangs beendet Editas die Entwicklung von reni-cel und implementiert erhebliche Kostensenkungsmaßnahmen, darunter eine Reduzierung der Belegschaft um 65% in den nächsten sechs Monaten. Mehrere Managementänderungen wurden angekündigt, darunter der Abgang des Chief Medical Officer Baisong Mei und zweier Vorstandsmitglieder. Es wird erwartet, dass die Umstrukturierung die Liquidität des Unternehmens bis zum zweiten Quartal 2027 verlängert.
- Achieved ~40% editing efficiency in HBG1/2 promoter site using proprietary delivery system
- Successful demonstration of high-efficiency liver editing in non-human primates
- Cost restructuring extends cash runway into Q2 2027
- Strategic focus on in vivo editing could expand therapeutic possibilities
- Discontinuation of reni-cel development program
- 65% workforce reduction over next 6 months
- Loss of key executives including Chief Medical Officer
- Two board members resigning effective December 31, 2024
Insights
The strategic pivot by Editas Medicine marks a significant shift in the company's trajectory. The decision to focus on in vivo CRISPR gene editing is backed by promising preclinical data, particularly the ~40% editing efficiency in hematopoietic stem cells and successful liver editing in non-human primates. However, the discontinuation of reni-cel development and the 65% workforce reduction represents a major restructuring that will fundamentally change the company's operational scope.
The extension of cash runway into Q2 2027 through cost-cutting measures provides significant operational flexibility, but comes at the expense of current clinical programs. The transition to in vivo editing could potentially offer a more scalable and commercially viable approach, though it effectively resets the company's clinical timeline. The focus on extrahepatic delivery systems and upregulation capability could differentiate Editas in the competitive gene editing landscape.
This strategic overhaul presents a mixed financial outlook. The 65% workforce reduction will significantly lower operational costs, with the cash runway extension to Q2 2027 providing substantial financial breathing room. However, abandoning reni-cel after failing to secure a commercial partner raises concerns about the company's ability to monetize its pipeline.
The shift to in vivo editing could potentially reduce manufacturing complexity and treatment costs compared to ex vivo approaches, improving long-term commercial viability. Yet, this pivot essentially moves Editas back to preclinical stage, which could impact investor confidence in the near term. With a market cap of just
- Focus on in vivo CRISPR-edited medicines based on Editas researchers’ recent scientific progress in multiple tissues:
- Achieved pre-clinical in vivo proof of concept of high level HBG1/2 promoter editing and HbF induction in a humanized mouse model for treatment of sickle cell disease and beta thalassemia with a single dose of an HSC-targeted lipid nanoparticle (tLNP) formulation
- Achieved in vivo proof of concept of high efficiency editing in the liver in non-human primates
- Ending development of reni-cel after extensive search did not yield a commercial partner
- The Company will work closely with the clinical trial sites, regulators, and other parties to determine the path forward for patients enrolled in the RUBY and EdiTHAL trials
- Initiating cost savings measures and reduction in headcount to align workforce and resources to in vivo pipeline, extending cash runway into Q2 2027
- Conference call and webcast today at 5:00 p.m. ET
CAMBRIDGE, Mass., Dec. 12, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading gene editing company, today announced a critical pivot to optimize its cost structure, extend its cash runway into Q2 2027, and position the Company to accelerate its intent to achieve in vivo human proof of concept in approximately two years.
“Recent scientific breakthroughs by the Editas team have convinced us that the timelines around the near-term viability of in vivo CRISPR-edited medicines have accelerated meaningfully. Two years ago, we laid out our strategy and objective to become a leader in in vivo programmable gene editing. Based on these advances, we are transitioning to a fully in vivo company. We believe the ability to provide in vivo gene editing that functions via gene upregulation across tissues holds the potential to significantly expand the addressable therapeutic possibilities for CRISPR-based gene editing and uniquely position Editas to be a leader in the field moving forward,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine.
The Company transition follows the recent in vivo pre-clinical proof of concept in multiple tissues:
- Hematopoietic Stem Cells (HSCs):
- Editas achieved ~
40% editing of the HBG1/2 promoter site after using a novel, Editas-proprietary targeted lipid nanoparticle (tLNP) for extrahepatic tissue delivery to deliver a single dose of its clinically validated Cas12a editing machinery directly to human hematopoietic stem cells (HSCs) in mice engrafted with human HSCs.1 - HBG1/2 biology has been validated and derisked in patients with reni-cel in the RUBY trial.
- The editing in HSCs with the Company’s proprietary tLNP formulation resulted in the meaningful 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.
- Editas achieved ~
- Liver:
- The Company achieved in vivo proof of concept of high efficiency editing in the liver in non-human primates under its collaboration with Genevant.
The Company intends to share pre-clinical data and further development timelines from these programs in the first quarter of 2025.
In vivo HSC editing success is expected to enable extrahepatic tissues/cell types targeting beyond HSCs and demonstrates the potential of “plug ‘n play” in an in vivo extrahepatic LNP platform. The Company’s upregulation capability additionally enables a differentiated strategy for liver targets for diseases with high unmet need and first-in-class opportunities.
In connection with Editas Medicine’s transition to an in vivo company, the Company initiated a reduction in headcount that will eliminate approximately
Additionally, Emma Reeve and Meeta Chatterjee, Ph.D. are resigning from the Board of Directors, effective December 31, 2024. Jessica Hopfield, Ph.D., has been named Chair of the Board, effective December 31, 2024.
Dr. O’Neill added, “We want to extend our deepest appreciation to patients, investigators, clinical sites staff, and our employees who have shown tremendous dedication and commitment to developing a potentially transformational medicine like reni-cel. We also want to express specific gratitude to the patients in our clinical trials and their caregivers whose dedication to disease research for their community makes us even more committed to accelerating our efforts towards an in vivo program for sickle cell disease and beta thalassemia.”
Conference Call
The Editas Medicine management team will host a conference call and webcast today at 5:00 p.m. ET. To access the call, please dial 1-877-407-0989 (domestic) or +1 201-389-0921 (international) and ask for the Editas Medicine conference call. A live webcast of the call will also be available on the Investors section of the Editas Medicine website at www.editasmedicine.com, and a replay will be available approximately two hours after its completion.
About Editas Medicine
As a leading 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 www.editasmedicine.com.
Forward-Looking Statements
This 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 transition to a fully in vivo company, the intention to achieve human proof of concept in approximately two years, and the potential success of its in vivo gene editing programs, the timing for releasing additional pre-clinical data, the anticipated effects, including potential cost savings, of the Company’s decision to discontinue development of reni-cel and initiate the related reduction in headcount, the scope and timing of the reduction in headcount, and the expected extension of the Company’s 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; availability and timing of results from preclinical studies; expectations for regulatory approvals to conduct trials; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; and that the decision to discontinue clinical development of reni-cel and the related reduction in headcount may have unexpected consequences or not result in the expected cost savings. 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.
1 Previously disclosed editing of
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