BCL11A

CRISPR/Cas9 is a gene-editing technology that allows scientists to precisely cut and modify DNA in living organisms. Credit: Prostock-studio via Shutterstock. Amidst the long-running Cas9 patent battle, Editas Medicine has struck a deal to sell a portion of its future revenue stream from Vertex Pharmaceuticals to a subsidiary of DRI Healthcare in return for an immediate $57m cash boost. This move follows a December 2023 licence agreement in which Vertex paid Editas $50m upfront for nonexclusive rights to its ex vivo Cas9 gene editing technology. The technology targets the BCL11A gene in the blood disorders of sickle cell disease and beta-thalassemia. The deal encompasses Casgevy (exagamglogene autotemcel), the world’s first approved gene therapy that treats sickle cell disease and beta-thalassemia. Casgevy was co-developed by Vertex and CRISPR Therapeutics. The deal gave Vertex access to Editas’ tool for future advancements of the gene therapy. In return for $57m upfront, Editas will surrender up to 100% of certain future annual payments from Vertex, which could range between $5m and $40m a year, according to the 3 October announcement. Editas will also give up part of a potential $50m payment it could receive from Vertex if certain conditions are met. However, Editas keeps the rights to fixed payments for 2024 and could also get an additional payment if Vertex hits certain sales targets. Editas said that by monetising these future payments now, it strengthens its cash position while DRI Healthcare gains access to potential recurring revenue from Vertex’s licencing fees. CEO of Editas, Gilmore O’Neill said: “We are pleased to partner with DRI to monetise a portion of the licensing payments from the Vertex Cas9 license deal we announced last December, providing us with considerable non-dilutive capital that we can put to work immediately as we develop our pipeline of future medicines.” See Also: The benefits of using a nimble platform from phase to phase Switching from tablets: The benefits of using softgels The deal comes after a long legal battle over who owns key patents for CRISPR technology, which was discovered by several researchers and the basis of the 2020 Nobel Prize in Chemistry that was awarded to Emmanuelle Charpentier and Jennifer Doudna. Charpentier is the founder of CRISPR Therapeutics. The University of Vienna and the University of California challenged the Broad Institute at MIT and Harvard, over who had rights to the technology. In February 2022, the Broad Institute won a US Patent and Trademark Office Ruling. Editas licenced the technology from the Broad Institute, giving it the exclusive rights to use certain patents of human applications of CRISPR, including for RNA-guided nuclease Cas9 owned and co-owned by The Broad Institute of MIT and Harvard and the Rockefeller University, according to a 13 December 2023 press release by Editas. CRISPR-Cas9 is a gene-editing technology that allows scientists to precisely cut and modify DNA in living organisms. This legal fight is still ongoing, where Charpentier and Doudna’s patents are being challenged in the US and European patent courts. Their European patents were weakened in August 2024 after a ruling from a European technical appeals board said their original patent filing didn’t clearly explain how to use CRISPR and it doesn’t count as a proper invention. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Strengthens balance sheet with non-dilutive capital to enable further pipeline development and related strategic prioritiesCAMBRIDGE, Mass., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today announced the sale of certain future license fees and other payments owed to Editas Medicine 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. “We are pleased to partner with DRI to monetize a portion of the licensing payments from the Vertex Cas9 license deal we announced last December, providing us with considerable non-dilutive capital that we can put to work immediately as we develop our pipeline of future medicines,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. “We look forward to an ongoing relationship with DRI as we continue to execute our strategy.” Under the terms of the agreement, Editas Medicine will receive an upfront cash payment of $57 million in exchange for up to 100% of certain future annual license fees payable to Editas Medicine, ranging from $5 million to $40 million per year (inclusive of certain sales-based annual license fees that may become due) and a mid-double-digit percentage of Editas Medicine’s portion of a $50 million contingent upfront payment for which Editas Medicine is eligible under the Vertex license agreement. In addition to a portion of any such contingent upfront payment, Editas Medicine retains rights to fixed annual license fees for 2024 and a mid-single-digit million-dollar payment due to Editas Medicine in the event of Vertex’s achievement of certain annual sales milestones. In December 2023, Editas Medicine announced that the Company and Vertex entered into a license agreement. Under the terms of the agreement, Vertex obtained a non-exclusive license for Editas Medicine’s Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of sickle cell disease and beta thalassemia, including CASGEVY® (exagamglogene autotemcel). Editas Medicine is the exclusive licensee of certain CRISPR patent estates for making human medicines. These include a Cas9 patent estate owned and co-owned by Harvard University, Broad Institute, the Massachusetts Institute of Technology, and The Rockefeller University. TD Cowen served as exclusive financial advisor and WilmerHale served as legal advisor to Editas Medicine. Cravath, Swaine & Moore served as legal advisor to DRI Healthcare Trust. 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 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 initiation, timing, progress and results of the Company’s preclinical studies and its research and development programs and the Company’s expected use of the funds received from the financing. 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 pre-clinical studies and clinical trials, and clinical development of the Company’s product candidates 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.

The CRISPR/Cas9 gene-editing therapy Casgevy offers a one-time curative option for beta-thalassemia patients. Image: Shutterstock/Perception7 Following positive guidance issued by the National Institute for Health and Care Excellence (NICE), the CRISPR-based gene therapy Casgevy has been made available to patients with transfusion-dependent beta thalassemia (TDT) in England. Today (8 August), NICE released their final draft guidance recommending Casgevy coverage via the NHS. Casgevy was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The recommendation concerns patients aged 12 and older with severe beta-thalassemia needing blood transfusion, for whom blood and bone marrow transplant is suitable, but no matching donor is available. Consequently, Vertex announced a reimbursement agreement with NHS England making the treatment available beginning today. Casgevy will be accessible through the Innovative Medicines Fund for up to 460 eligible patients, as per NICE. The NICE decision came after an independent committee heard statements on how TDT impacts the lives of patients. TDT is a life-threatening inherited disease in which genetic mutation reduces or prevents production of healthy red blood cells and haemoglobin. The condition often manifests as intense fatigue; infants may suffer delayed development, misshapen bones, and enlarged organs. Treatment involves lifelong blood transfusions and iron chelation therapy. See Also: Takeda’s ADZYNMA gains EU approval for cTTP treatment Amneal’s CREXONT gains FDA approval for Parkinson’s treatment Stem cell transplant is a potentially curative option, but is limited by matched donor scarcity. This is especially true for people of South Asian ethnicity who are disproportionally affected and for whom matching donors are particularly rare, according to Dr. Diana Hernandez, a group leader at the medical nonprofit Anthony Nolan. Hernandez added that Casgevy also has potential against sickle cell disease – “Sickle cell largely affects patients from African and African-Caribbean backgrounds, who are less likely to have a matching stem cell donor, and this community has been waiting years for a new, effective treatment.” The Medicines and Healthcare products Regulatory Agency (MHRA) granted Casgevy the world-wide first authorisation for a CRISPR-based therapy in November 2023. The approval was for both TDT and sickle cell disease. At a list price of £1.65mn (~$2mn) per course, this reimbursement agreement has significantly increased availability for patients in England. Casgevy is a one-time therapy in which a patient’s haematopoietic stem cells are removed and edited through clustered regularly interspaced short palindromic repeats (CRISPR) technology that uses the Cas9 enzyme at the BCL11A gene. Modified cells are transfused into the patient, increasing foetal haemoglobin production in red blood cells without the risk of rejection. Vertex is also working with reimbursement authorities in the EU to make their therapy available to TDT and SCD patients, as has been done in the US , Saudi Arabia, and Bahrain. The company is poised to compete with bluebird Bio’s CRISPR-based SCD therapy Lyfgenia (lovotibeglogene autotemcel). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy