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Operator
Operator
Good morning and welcome to the Editas Medicine first quarter 2024 conference call. (Operator Instructions) Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Christy Barnhart, Corporate Communications and Investor Relations at Editas Medicine.
早安,歡迎參加 Editas Medicine 2024 年第一季電話會議。(操作員說明)請注意,本次通話是應公司要求進行錄音的。我現在想將電話轉給 Editas Medicine 企業傳播和投資者關係部門的 Christy Barnhart。
Christy Barnhart - IR
Christy Barnhart - IR
And thank you. Good morning, everyone, and welcome to our first quarter 2024 conference call. Earlier this morning, we issued a press release providing our financial results and recent corporate updates. A replay of today's call will be available in the Investors section of our website approximately two hours after completion. After our prepared remarks, we will open the call for Q&A.
謝謝你。大家早安,歡迎參加我們的 2024 年第一季電話會議。今天早些時候,我們發布了一份新聞稿,提供了我們的財務表現和最近的公司更新。今天電話會議的重播將在會議結束後大約兩小時在我們網站的投資者部分提供。在我們準備好發言後,我們將開始問答環節。
As a reminder, various remarks that we make during this call about the Company's future expectations, plans and prospects constitute forward-looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of our most recent annual report on Form 10-K, which is on file with the SEC as updated by our subsequent filings.
需要提醒的是,我們在本次電話會議中就公司未來預期、計畫和前景發表的各種言論均構成前瞻性陳述,符合 1995 年《私人證券訴訟改革法案》中的安全港條款。由於各種重要因素的影響,實際結果可能與這些前瞻性陳述所表明的結果存在重大差異,包括我們最新的 10-K 表格年度報告的風險因素部分中討論的因素,該報告已向 SEC 備案由我們後續的文件更新。
In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date, except as required by law. We specifically disclaim any obligation to update or revise any forward-looking statements even if our views change.
此外,任何前瞻性陳述僅代表我們截至今天的觀點,不應被視為代表我們在任何後續日期的觀點,除非法律要求。即使我們的觀點發生變化,我們特別不承擔更新或修改任何前瞻性陳述的義務。
Now I will turn the call over to our CEO, Gilmore OâNeill.
現在我將把電話轉給我們的執行長吉爾摩·奧尼爾。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks, Christy, and good morning, everyone. Thank you for joining us today. On Editas is first quarter 2024 earnings call. With me today are four members of the Editas executive team our Chief Medical Officer, Baisong Mei; our Chief Scientific Officer, Linda Burkly; our Chief Financial Officer, Erick Lucera; and our Chief Commercial and Strategy Officer, Karen Deardof.
謝謝克里斯蒂,大家早安。感謝您今天加入我們。Editas 正在召開 2024 年第一季財報電話會議。今天與我在一起的有 Editas 執行團隊的四名成員,我們的首席醫療官 Baisong Mei;我們的首席科學官 Linda Burkly;我們的財務長 Erick Lucera;以及我們的首席商務和策略長 Karen Deardof。
We are pleased with Editas has momentum and progress in the first quarter of 2024. It has the stories to deliver life-changing medicines to patients with previously untreatable or undertreated genetically determined diseases and our vision and focus strategy is to position a test as a leader in in vivo program with gene edits.
我們對 Editas 在 2024 年第一季的勢頭和進展感到高興。它有故事為患有以前無法治療或治療不足的基因疾病的患者提供改變生活的藥物,我們的願景和重點策略是將測試定位為體內基因編輯計畫的領導者。
Three Pillars underpin our strategy. The first of those pillars is to drive ready cell and edited cell therapy for hemoglobinopathies and formerly known as editorial one toward BLA and commercialization. Second is to build an in vivo editing pipeline. And the third is to increase business development activities with a particular focus on monetizing our very strong intellectual property.
三大支柱支撐著我們的策略。第一個支柱是推動針對血紅蛋白病的現成細胞和編輯細胞療法,以前稱為 BLA 和商業化的編輯療法。二是建構體內編輯管道。第三是增加業務開發活動,特別著重於將我們強大的智慧財產權貨幣化。
At the start of 2024, we announced the following 2024 objectives for Randy. So we will provide a clinical update from the Ruby trial for severe sickle cell disease and the endometrial trial for transfusion dependent beta-thalassemia in mid-2024. And by year end 2024, we will complete adult cohort enrollment and initiate the adolescent cohort in Ruby and continue enrollment in editor for our in vivo pipeline, we will establish in-vivo preclinical proof of concept for an undisclosed indication. And for BD, we will leverage our robust IP portfolio and business developed to drive value and complement core gene editing technology capabilities. So how we executed against this strategy and these objectives in the first quarter.
2024 年初,我們為 Randy 宣布了以下 2024 年目標。因此,我們將在 2024 年中期提供針對嚴重鐮狀細胞疾病的 Ruby 試驗和針對輸血依賴性 β 地中海貧血的子宮內膜試驗的臨床更新。到 2024 年底,我們將完成成人隊列註冊,並在 Ruby 中啟動青少年隊列,並繼續為我們的體內管道進行編輯註冊,我們將為未公開的適應症建立體內臨床前概念證明。對於 BD,我們將利用我們強大的智慧財產權組合和開發的業務來推動價值並補充核心基因編輯技術能力。那麼我們在第一季如何執行這項策略和這些目標。
Let us start with Renaissance first on enrollment. We've been very pleased with the growing patient and healthcare provider interest in Renaissance. Indeed, we are delighted to share that we have completed enrollment in the adult cohort of the Ruby clinical trial. Additionally, we have enrolled multiple patients and have more in screening in the adolescent cohort of a Ruby study, which was launched at the beginning of this year. And we continue to enroll beta-thalassemia patients in our adult study. Dosing continues in both the Ruby and NFL studies.
讓我們先從Renaissance的招生開始。我們對患者和醫療保健提供者對 Renaissance 日益增長的興趣感到非常高興。事實上,我們很高興地告訴大家,我們已經完成了 Ruby 臨床試驗成人隊列的招募。此外,我們還招募了多名患者,並在今年年初啟動的 Ruby 研究的青少年隊列中進行了更多篩檢。我們繼續在成人研究中招募β-地中海貧血患者。Ruby 和 NFL 研究中的劑量仍在繼續。
Second, on clinical data, we remain on track to present a substantial clinical dataset of at least 18 sickle cell patients with 2 to 21 months of clinical follow-up in the Ruby study in the middle of 2024, and we will share a further update by year end. We are also on track to present clinical data from the at-home study of red cell transfusion dependent beta-thalassemia in the middle of 2024 and again by year end, based on my will share more ready foundations later on in this call.
其次,在臨床數據方面,我們仍有望在 2024 年中期的 Ruby 研究中提供至少 18 名鐮狀細胞病患者的大量臨床數據集,並進行 2 至 21 個月的臨床隨訪,我們將分享進一步的更新到年底。我們還將在 2024 年年中和年底之前提供紅血球輸注依賴性 β 地中海貧血家庭研究的臨床數據,基於我稍後將在本次電話會議中分享更多現成的基礎。
On the manufacturing front, I am pleased to share that we have promoted Greg Whitehead to the role of Chief Technology and Quality Officer, leading our technical development, technical operations and quality departments. Greg has more than 25 years of experience in the biotech industry has extensive cell and gene therapy, clinical and commercial development expertise.
在製造方面,我很高興地告訴大家,我們已晉升 Greg Whitehead 為首席技術和品質官,領導我們的技術開發、技術營運和品質部門。Greg 在生物技術產業擁有超過 25 年的經驗,擁有豐富的細胞和基因治療、臨床和商業開發專業知識。
Now let's turn to in-vivo and our pipeline development, where we continue to strengthen our in vivo discovery capabilities and continued lead discovery work on in vivo therapeutic targets in hematopoietic stem cells and other tissues. Importantly, we remain on track to establish in-vivo preclinical proof-of-concept for an undisclosed indication by the end of the year. Our internal development efforts are differentiated by leveraging the Indo crisper technology.
現在讓我們轉向體內和我們的管道開發,我們將繼續加強我們的體內發現能力,並繼續在造血幹細胞和其他組織的體內治療靶點上進行領先的發現工作。重要的是,我們仍有望在今年年底前針對未公開的適應症建立體內臨床前概念驗證。我們的內部開發工作透過利用 Indo 保鮮盒技術而與眾不同。
We already used to up-regulate gamma globin expression through direct debiting of the HBG. one two promoter size in our ex-vivo ready cell program. Our in vivo approach is aimed at functional up-regulation of gene expression in genetically defined diseases with a preliminary focus on rare and orphan patient populations in the medium to long term, we intend to expand to more common genetically determined diseases. Linda Berkeley, our CSO, will share more details on our in vivo strategy and progress towards building an in vivo pipeline later on in the call.
我們已經習慣透過直接扣款 HBG 來上調 γ 珠蛋白表現。我們的離體準備細胞程序中的一兩個啟動子大小。我們的體內方法旨在基因確定的疾病中基因表現的功能性上調,初步重點關注罕見和孤兒患者群體,從中長期來看,我們打算擴展到更常見的基因確定的疾病。我們的首席策略長琳達·伯克利 (Linda Berkeley) 將在稍後的電話會議中分享有關我們的體內策略和建立體內管道的進展的更多詳細資訊。
Finally, what is happening in business development. In March, we signed a two year extension to the collaboration with Bristol-Myers Squibb to research, develop and commercialize autologous and allogeneic alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. We also have options to extend that collaboration for an additional two years to date, Bristol-Myers Squibb has opted into 13 different programs across 11 gene targets to date, two programs are currently in IND-enabling studies and four programs are in late-stage discovery and the intellectual property.
最後,業務發展中發生了什麼事。今年 3 月,我們與百時美施貴寶簽署了為期兩年的合作延期協議,以研究、開發和商業化用於治療癌症和自體免疫疾病的自體和同種異體 α-β T 細胞藥物。我們也可以選擇將這種合作再延長兩年。階段發現和知識產權。
Yesterday, oral arguments were held before the US Court of Appeals for the Federal Circuit regarding an appeal of the Patent Trial and Appeal Board or PTAB, previous decision favoring Broad Institute in the US patent interference involving specific patents for crisper Cas nine editing in human cells between the University of California, University of Vienna and Emmanuelle sharp on TA for CDC. and the broad, we expect a decision on the case in the second half of 2024.
昨天,美國聯邦巡迴上訴法院就專利審判和上訴委員會(PTAB) 的上訴進行了口頭辯論,先前的裁決有利於布羅德研究所(Broad Institute) 涉及人類細胞中更清晰的Cas 9 編輯的特定專利的美國專利乾涉案加州大學、維也納大學和艾曼紐夏普 (Emmanuelle Sharp) 擔任 CDC 助理教練。總體而言,我們預計該案將在 2024 年下半年做出裁決。
Eric will share more BD. and IP. details later on in the call. We are energized by our progress and execution this quarter with our sharpened strategic focus, our world-class scientists and employees, our team drive and execution and strong balance sheet. We continue to build momentum to progress our strategy to deliver differentiated medicines to patients with serious diseases.
Eric將分享更多BD。和知識產權。稍後在通話中詳細說明。憑藉我們明確的策略重點、我們世界一流的科學家和員工、我們的團隊動力和執行力以及強大的資產負債表,本季的進展和執行力使我們充滿活力。我們繼續積蓄動力,推進我們為嚴重疾病患者提供差異化藥物的策略。
Now I will turn the call over to Baisong, our Chief Medical Officer.
現在我將把電話轉給我們的首席醫療官白松。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Thank you, Guillermo, and good morning, everyone. Let's talk about Renaissance, which is under clinical development for severe sickle cell disease and transfusion dependent beta-thalassemia. As Guillermo shared, we are pleased that we have completed enrollment in the adult cohort of the Phase one two three will be trial and the dosing continues in the adolescent cohort of there will be a study where we enrolled multiple patients and several more patients in screening, the interest and demand are high and very pleased about how quickly we have moved in screening and enrollment of the adolescent cohort.
謝謝你,吉列爾莫,大家早安。讓我們來談談Renaissance,它正在針對嚴重鐮狀細胞疾病和輸血依賴性β地中海貧血進行臨床開發。正如吉列爾莫所分享的,我們很高興我們已經完成了第一、二、三期試驗的成人隊列的入組,並且在青少年隊列中繼續給藥,我們將在一項研究中招募多名患者和更多患者進行篩選,人們的興趣和需求很高,並且對我們在青少年群體篩檢和登記方面的進展速度感到非常高興。
I'd like to thank colleagues and editors and our clinical trial partners for the collaboration and hard work. And more importantly, I would like to thank the patients, their families, investigators and study site staff for their trust and support in the ADHERE trial for transfusion dependent beta-thalassemia. We continue to move forward with enrollment and dosing. We look forward to sharing clinical data in the mid of this year and also at the year end, as I have shared.
我要感謝同事、編輯以及我們的臨床試驗合作夥伴的合作和辛勤工作。更重要的是,我要感謝患者、他們的家人、研究人員和研究中心工作人員對輸血依賴性β地中海貧血 ADHERE 試驗的信任和支持。我們繼續推進招募和給藥工作。正如我所分享的,我們期待在今年年中和年底分享臨床數據。
I visited and continue to build our Ruby in additional clinical trial sites and speak with the investigators. I appreciate the enthusiasm and support from investigators and study size. I'm pleased with the momentum of Renevia in patient recruitment freezes and dosing in both studies. I'm excited to hear from the investigators that patient goes to the rental sale have already seen positive changes in their lives.
我參觀了並繼續在其他臨床試驗地點建造我們的 Ruby,並與研究人員進行了交談。我感謝研究人員和研究規模的熱情和支持。我對 Renevia 在兩項研究中凍結患者招募和劑量的勢頭感到滿意。我很高興從調查人員那裡得知,參加租賃銷售的患者已經看到了他們的生活發生了積極的變化。
As we shared in our February earnings call, we aligned with FDA that will be clinical trial is now considered a Phase one two three trial for BLA filing. We also have alignment with the FDA on the study design endpoints and sample size. We look forward to future discussions with FDA and continue the calibration.
正如我們在 2 月的財報電話會議中分享的那樣,我們與 FDA 達成一致,該臨床試驗現在被視為 BLA 備案的一、二、三期試驗。我們也在研究設計終點和樣本量方面與 FDA 保持一致。我們期待未來與 FDA 的討論並繼續校準。
Turning to clinical data, as Gil mentioned, we are on track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up will be study in the middle of 2024 and a further update by year end 2024. What do we want to show there will be data set, including clinical data from at least 18 sickle cell patients with a 2 to 21 months of follow-up. And the editorial dataset waiting for the clinical data from seven patients with a four to months follow-up, we will present efficacy data, including total hemoglobin, fetal hemoglobin and the vessel completed events or VOE.
至於臨床數據,正如 Gil 所提到的,我們預計將提供鐮狀細胞疾病患者的實質臨床數據集,並將在 2024 年中期進行大量臨床追蹤研究,並在 2024 年底之前進一步更新。我們想要展示的是數據集,包括至少 18 名鐮狀細胞疾病患者的臨床數據,並進行了 2 至 21 個月的追蹤。編輯資料集正在等待 7 名患者的臨床數據,並進行 4 個月的隨訪,我們將提供療效數據,包括總血紅蛋白、胎兒血紅蛋白和血管完成事件或 VOE。
For sickle cell patient in will be studied and red blood cell transfusion for transfusion dependent thalassemia patients with PTCL study and safety data, including mutual appeal and probably engraftment for both steady. As a reminder, in December 2023, we shared safety and efficacy data from 11, we'll be patient and six additional patients. Once again, the data confirm the observation from our prior clinical readouts, including sales drilled only reflect the correction of anemia to a normal physiological range of total hemoglobin in sickle cell patients went out to a robust and sustained increase in fetal hemoglobin level in excess of 40%.
將研究針對鐮狀細胞疾病患者的PTCL研究和針對輸血依賴性地中海貧血患者的紅血球輸注的安全性數據,包括相互吸引力和可能的兩者穩定植入。提醒一下,2023 年 12 月,我們分享了 11 位病患和另外 6 位病患的安全性和有效性資料。數據再次證實了我們先前臨床讀數的觀察結果,包括鑽探的銷售僅反映鐮狀細胞患者的貧血校正至總血紅蛋白的正常生理範圍,胎兒血紅蛋白水平強勁且持續增加超過40%。
All Ruby sickle cell patients remained free of battle group events following Renaissance treatments. When you say are treated as because the location and transfusion dependent beta-thalassemia patients have shown successful engraftment have stopped the red blood cell transfusion and the safety profile of Renault sales to date is consistent with both of them, ablative conditioning and the autologous hematopoietic stem cell transplant.
所有紅寶石鐮狀細胞疾病患者在文藝復興治療後均未出現戰鬥群事件。當您說被視為因為位置和輸血依賴性β地中海貧血患者已顯示成功植入已停止紅血球輸注並且雷諾銷售迄今為止的安全性與他們兩者一致時,消融調理和自體造血幹細胞細胞移植。
These data reinforce our belief that we have a competitive product and a product potentially differentiated from other treatments with a rapid correction of needs, thanks to the deliberate choice of our discoveries have made early in the program, the choice of critical enzyme and the targeted two edits for increased fetal hemoglobin expression matters. Retail uses our proprietary, a scaffold A. enzyme to upregulate the HB. two and two promoter. Based on the clinical data thus far, we believe that sustained normal levels of total hemoglobin could be a potential point of differentiation for Renevia.
這些數據強化了我們的信念,即我們擁有一種有競爭力的產品,並且是一種可能與其他治療方法不同的產品,可以快速修正需求,這要歸功於我們在項目早期的發現、關鍵酶的選擇和目標兩種酶的深思熟慮的選擇。零售業使用我們專有的支架 A 酶來上調 HB。兩個和兩個啟動子。根據迄今為止的臨床數據,我們認為總血紅素持續正常水平可能是 Renevia 的潛在分化點。
Now I turn the call over to Linda, our Chief Scientific Officer space on Emblem.
現在我將電話轉給 Linda,她是我們 Emblem 空間的首席科學官。
Linda Burkly - EVP, Chief Scientific Officer
Linda Burkly - EVP, Chief Scientific Officer
Good morning, everyone. I'm happy to be talking to you this morning to share more details about our in-vivo strategy and our progress towards building an in vivo pipeline. I would like to take a moment to remind you why we believe in vivo medicines will be a disruptive transformative development in medical history with the potential to address genetically determined diseases with durable and curative outcomes for patients. First, in vivo medicines may reduce the administration burden of delivering editing medicines to patients in need, which will provide a broader access to patients all around the world.
大家,早安。我很高興今天早上與您交談,分享有關我們體內策略以及我們在建立體內管道方面取得的進展的更多詳細資訊。我想花點時間提醒您為什麼我們相信體內藥物將成為醫學史上的顛覆性變革發展,有潛力解決遺傳性疾病,為患者帶來持久和治癒的結果。首先,體內藥物可以減輕向有需要的患者提供編輯藥物的管理負擔,這將為世界各地的患者提供更廣泛的機會。
Second, off-the-shelf administration may allow for scalable manufacturing and lower cost to produce based on these two principles. We believe that in vivo gene editing will provide accessible cures for genetic diseases and therefore, it may be the most disruptive development in medical history.
其次,基於這兩個原則,現成的管理可以實現可擴展的製造並降低生產成本。我們相信體內基因編輯將為遺傳疾病提供可行的治療方法,因此,它可能是醫學史上最具顛覆性的發展。
So how will Editas position itself. There are many monogenic diseases that can potentially be cured with the gene editing approach. We have said that we will first target the development of treatments that are clearly differentiated from current standard of care, and that will leverage the aspects of crisper editing that give it a unique advantage over other therapeutic modalities.
那麼Editas將如何定位自己呢?有許多單基因疾病可以透過基因編輯方法治癒。我們說過,我們將首先致力於開發與當前護理標準明顯不同的治療方法,並將利用更清晰的編輯功能,使其比其他治療方式具有獨特的優勢。
Our internal development efforts are differentiated by leveraging Intel crisper technology reasons to up-regulate gammaCore globin expression through direct editing of the HBG. one two promoter site in our XC low renaissance program. Our in vivo approach is aimed at functional upregulation of gene expression in genetic diseases in rare and orphan patient populations from which we intend to expand to more common diseases. I'm also pleased to share several progress updates as we advance our in-vivo capabilities towards our long-term vision of being a leader in in vivo program, both gene editing.
我們的內部開發工作與眾不同,利用英特爾更清晰的技術理由,透過直接編輯 HBG 來上調 gammaCore 珠蛋白表現。我們的 XC 低復興計劃中的一兩個啟動子位點。我們的體內方法旨在對罕見和孤兒患者群體的遺傳疾病中的基因表現進行功能性上調,我們打算將其擴展到更常見的疾病。我還很高興分享一些進展更新,因為我們正在推進我們的體內能力,以實現我們成為體內專案(基因編輯)領導者的長期願景。
First and most importantly, as Gil mentioned, we remain on track to establish in-vivo preclinical proof of concept for an undisclosed indication by the end of the year. Editas is well positioned with established capabilities in the four main components of in vivo gene editing medicine, one guide RNA, two editing enzymes, three messenger RNA and four delivery technologies. And we are currently evaluating lipid nanoparticles for delivery of gene editing cargo into multiple tissue types with multiple companies. Additionally, we are evaluating next-generation delivery technology.
首先也是最重要的是,正如吉爾所提到的,我們仍有望在今年年底前針對未公開的適應症建立體內臨床前概念驗證。Editas 在體內基因編輯醫學的四個主要組成部分(一種引導 RNA、兩種編輯酶、三種信使 RNA 和四種遞送技術)方面擁有成熟的能力,處於有利位置。我們目前正在與多家公司合作評估脂質奈米顆粒將基因編輯貨物輸送到多種組織類型中的作用。此外,我們正在評估下一代交付技術。
Second, our in-vivo capabilities with the potential to be used in developing transformative in vivo gene editing medicines are demonstrated by the preclinical data we are presenting at the American Society of Gene and Cell Therapy or ASGCT Annual Meeting in three presentations taking place on Thursday and Friday of this week on Friday, in an oral presentation, we will share in vivo preclinical data from mouse studies using lipid nanoparticle mediated delivery of an optimized guide RNA and engineered AS. casualties messenger RNA in poster presentations on Thursday and Friday, we will share preclinical data demonstrating a F. capital, a guide RNA modifications.
其次,我們在周四舉行的美國基因與細胞治療學會或 ASGCT 年會上的三場演講中展示的臨床前數據證明了我們的體內能力有可能用於開發變革性體內基因編輯藥物在周五和在週五的口頭報告中,我們將分享來自小鼠研究的體內臨床前數據,這些數據使用脂質奈米顆粒介導的優化引導RNA和工程化AS的傳遞。在周四和周五的海報展示中,我們將分享臨床前數據,展示 F. 資本,指導 RNA 修飾。
It enables high potency gene editing in multiple cell types, including in the liver and improve gene editing outcomes in vivo, enabling the development of in vivo gene editing medicine and research on identifying potent large hearing companies is LSR as a foundation to develop novel in vivo gene editing technologies for holding knock-in, expanding potential in vivo gene editing targets for developed medicines. Third, edit test crisper based in vivo gene editing capability has been clinically validated. Notably in 2020, I had us as the first company ever to treat human with an in vivo delivered crisper based gene editing medicine at it one on one.
它能夠在包括肝臟在內的多種細胞類型中進行高效基因編輯,並改善體內基因編輯結果,從而促進體內基因編輯醫學的發展,以及識別有效的大型聽力公司的研究是LSR作為開發新型體內基因編輯的基礎基因編輯技術用於進行敲入,擴大已開發藥物的潛在體內基因編輯目標。第三,基於編輯測試保鮮盒的體內基因編輯能力已被臨床驗證。值得注意的是,在 2020 年,我們成為第一家使用體內交付的基於基因編輯藥物的一對一治療人類的公司。
In fact, earlier this week, the New England Journal of Medicine published a manuscript entitled gene editing, Percepta 90 associated retinal degeneration, detailing our formally development candidate that it went to one for the treatment of Leber's Congenital Amaurosis type 10 or LCA10. It is established clear in vivo human proof of concept in 2022.
事實上,本週早些時候,《新英格蘭醫學雜誌》發表了一篇題為「基因編輯,Percepta 90 相關視網膜變性」的手稿,詳細介紹了我們正式開發的候選藥物,該候選藥物用於治療萊伯先天性黑蒙10 型或LCA10。將於 2022 年建立明確的人體體內概念驗證。
And we are pleased that the results from this groundbreaking clinical trial were published by the New England Journal of Medicine. These progress updates demonstrate Editas execution on our in vivo strategy and our proven in vivo gene editing capabilities. And I look forward to sharing more details about our in-vivo development strategy and our progress towards building an in vivo pipeline later this year.
我們很高興這項突破性的臨床試驗的結果由《新英格蘭醫學雜誌》發表。這些進展更新證明了 Editas 對我們體內策略的執行以及我們經過驗證的體內基因編輯能力。我期待在今年稍後分享有關我們體內開發策略以及我們在建立體內管道方面取得的進展的更多細節。
Now I will turn the call over to Eric, our Chief Financial Officer.
現在我將把電話轉給我們的財務長埃里克。
Erick Lucera - EVP, CFO
Erick Lucera - EVP, CFO
Thank you, Linda, and good morning, everyone. I'm happy to be speaking to you, and I'm excited to provide updates on our business development achievements, intellectual property and financial results for the first quarter of 2024. First, in regard to business development, as Gil mentioned, in March, we announced a two year extension to the collaboration with Bristol-Myers Squibb to research, develop and commercialize autologous and allogeneic alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. We also have options to extend the collaboration for an additional two years to date, Bristol-Myers Squibb has opted into 13 different programs across 11 gene targets.
謝謝你,琳達,大家早安。我很高興與您交談,並且很高興能夠提供有關我們 2024 年第一季業務發展成就、智慧財產權和財務業績的最新資訊。首先,在業務發展方面,正如吉爾在三月提到的,我們宣布將與百時美施貴寶的合作延長兩年,以研究、開發和商業化用於治療癌症和癌症的自體和同種異體α -β T細胞藥物。到目前為止,我們還可以選擇將合作再延長兩年,百時美施貴寶已選擇參與 11 個基因標靶的 13 個不同項目。
To date. New programs are currently in IND-enabling studies and four programs are in late stage discovery. As a reminder, for each new experimental medicine, the Bristol Myers Squibb develops and commercializes using opted into genome editing tools, investors, Bristol-Myers Squibb will pay Editas Medicine and potential future milestone payments following the approval of any products resulting from the collaboration. Editas Medicine is also eligible to receive tiered royalties on net sales.
迄今為止。新計畫目前正在進行 IND 支持研究,四個計畫正處於後期發現階段。提醒一下,對於每一種新的實驗藥物,百時美施貴寶都會使用選擇的基因組編輯工具進行開發和商業化,投資者在合作產生的任何產品獲得批准後,百時美施貴寶將向Editas Medicine 支付以及未來潛在的里程碑付款。Editas Medicine 也有資格獲得淨銷售額的分級特許權使用費。
So we are pleased that our Bristol-Myers collaboration has proved to be a productive partnership, and we are committed to future collaborations and partnerships that will allow for the continued access and advancement of gene editing and in IP. As Joe mentioned yesterday, the oral arguments were held before the US Court of Appeals for the Federal Circuit.
因此,我們很高興我們的百時美施貴寶合作被證明是一種富有成效的夥伴關係,我們致力於未來的合作和夥伴關係,這將使基因編輯和智慧財產權領域的持續發展和進步成為可能。正如喬昨天提到的,口頭辯論是在美國聯邦巡迴上訴法院進行的。
Regarding the CBC.'s appeal of the PTAB.'s decision involving patents for crisper cash, nine editing in human cells. As you know, the Broad Institute has previously prevailed three times against the CBC twice with the PTAB. And once at the Federal Circuit, the Federal Circuit's review will determine whether the PTAB. correctly applied the law. It is important to remember, the court will not hear new evidence, an appellate court decision in the broad favor would reaffirm Editas position as the exclusive licensor of the patents covering [cash nine] used in human medicines in the US.
關於 CBC 對 PTAB 涉及保鮮現金、九項人體細胞編輯專利的裁決的上訴。如您所知,布羅德研究所此前曾與 PTAB 兩次戰勝 CBC 3 次。一旦到達聯邦巡迴法院,聯邦巡迴法院的審查將決定 PTAB 是否同意。正確適用法律。重要的是要記住,法院不會聽取新的證據,上訴法院廣泛支持的裁決將重申 Editas 作為美國人類藥物中使用的 [現金九] 專利的獨家許可人的地位。
Is also important to remember that only a small fraction of the IP we licensed from the road are involved in the ongoing USPTO interference proceedings. We expect a decision on the case in the second half of 2024, we remain confident that the growth will once again prevail our IP portfolio of foundational US and international patents covering cash, nine and cast 12 used in human medicines are a source of meaningful value as we believe that globally there are more than 100 cash noncash 12 A.
同樣重要的是要記住,我們從道路上獲得許可的智慧財產權中只有一小部分涉及正在進行的美國專利商標局干涉程序。我們預計該案將於2024 年下半年做出裁決,我們仍然相信增長將再次佔上風,我們的美國和國際基礎專利知識產權組合涵蓋現金、9 項和12 項用於人類藥物的專利,是有意義的價值來源因為我們相信全球有超過 100 種現金非現金 12 A.
Programs in development worldwide with the majority of the programs being developed by 10 companies. We believe these potential fields represent a potential material source of non-dilutive capital as evidenced by our deal in the fourth quarter of 2023 that extended our cash runway by two quarters. We look forward to future discussions and now I'd like to refer you to our press release issued earlier today for a summary of our financial results for the first quarter of 2024.
全球正在開發的程序,其中大部分程序由 10 家公司開發。我們相信這些潛在領域代表了非稀釋性資本的潛在物質來源,我們在 2023 年第四季的交易將我們的現金跑道延長了兩個季度,就證明了這一點。我們期待未來的討論,現在我想請您參閱今天早些時候發布的新聞稿,以了解我們 2024 年第一季的財務業績摘要。
I'll take this opportunity to briefly review a few items for the quarter. Our cash cash equivalents and marketable securities as of March 31, $377 million compared to $427 million as of December 31st, 2023 we expect our existing cash, cash equivalents and marketable securities, together with near term annual license fees and contingent upfront payments payable under our license agreement with Vertex to fund our operating expenses and capital expenditures into 2026.
我將藉此機會簡要回顧本季的一些項目。截至3 月31 日,我們的現金、現金等價物和有價證券為3.77 億美元,而截至2023 年12 月31 日為4.27 億美元,我們預計我們現有的現金、現金等價物和有價證券,加上近期年度許可費和根據我們的規定應付的或有預付款與 Vertex 簽訂許可協議,為我們到 2026 年的營運費用和資本支出提供資金。
Revenue for the first quarter of 2024 was $1.1 million compared to $9.9 million for the same period in 2023. A decrease relates to the January 2023 one-time sale of the Company's wholly owned oncology assets and related licenses. R&D expenses this quarter increased by $11 million to $49 million from the first quarter of 2023.
2024 年第一季的營收為 110 萬美元,而 2023 年同期的營收為 990 萬美元。減少與 2023 年 1 月公司全資腫瘤學資產和相關許可證的一次性出售有關。本季研發費用較 2023 年第一季增加 1,100 萬美元,達到 4,900 萬美元。
This increase relates to additional clinical and manufacturing costs that support the continued progression of the Company's red cell program. The increase is also attributable to one-time payments related to sublicense and license obligations and costs will continue to incur these types of payments as we and our collaboration partners advanced certain license programs in the gene editing space.
這一增長與支持公司紅血球項目持續進展的額外臨床和製造成本有關。這一增長還歸因於與分許可和許可義務相關的一次性付款,隨著我們和我們的合作夥伴在基因編輯領域推進某些許可計劃,這些類型的費用將繼續產生。
G&A expenses for the first quarter of 2024 were $19 million, which decreased from $23 million in the first quarter of '23. The decrease in expenses primarily attributable to one-time professional service expenses related to the 2023 strategic initiatives and business development activities, as well as reduced legal and patent costs with our BD. and IT. activity and a cash runway into 2026.
2024 年第一季的一般管理費用為 1,900 萬美元,較 2023 年第一季的 2,300 萬美元有所下降。費用減少主要歸因於與 2023 年策略性舉措和業務發展活動相關的一次性專業服務費用,以及我們的 BD 法律和專利成本的減少。和資訊科技。活動和到 2026 年的現金跑道。
Editas remains in a strong financial position. We have ample resources to continue the advancement of our Renaissance program, support the progression of our in vivo capabilities to develop our pipeline and leverage our strong IP position for additional business development and licensing opportunities. For that, I'll hand the call back to Gilmore.
Editas 的財務狀況依然強勁。我們擁有充足的資源來繼續推進我們的復興計劃,支持我們體內能力的發展,以開發我們的產品線,並利用我們強大的智慧財產權地位來獲得更多的業務發展和許可機會。為此,我會將電話轉回吉爾摩。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thank you, Eric. We are proud of our progress in the first quarter of 2024. I look forward to continue to accelerate the momentum in 2024 as we continue to evolve from a development stage technology platform company into a commercial stage gene editing company. We look forward to continuing our transformation and sharing our progress with you. As a reminder of our 2024 strategic objectives for any sale, we will provide a clinical update from the Renaissance Ruby trial for severe sickle cell disease and the NHL trial for transfusion dependent beta-thalassemia in mid-2024 and year end 2024.
謝謝你,埃里克。我們對 2024 年第一季的進展感到自豪。我期待在 2024 年繼續加速這一勢頭,我們繼續從開發階段的技術平台公司發展成為商業階段的基因編輯公司。我們期待繼續轉型並與您分享我們的進步。為了提醒我們 2024 年任何銷售的策略目標,我們將在 2024 年中期和 2024 年底提供針對嚴重鐮狀細胞疾病的 Renaissance Ruby 試驗和針對輸血依賴性 β 地中海貧血的 NHL 試驗的臨床更新。
We have now completed the adult cohort enrollment and have started enrolling patients in the adolescent cohort and Ruby. We will also continue enrollment in FL and dosing in both trials. For our in vivo pipeline, we will establish in vivo preclinical proof of concept for an undisclosed indication. And for BG., we will leverage our robust IP portfolio and business development capabilities to drive value and complement core gene editing technology capabilities.
我們現已完成成人隊列入組,並開始招募青少年隊列和 Ruby 患者。我們也將繼續在 FL 進行招募並在兩項試驗中進行給藥。對於我們的體內管道,我們將為未公開的適應症建立體內臨床前概念證明。對於 BG.,我們將利用我們強大的智慧財產權組合和業務開發能力來推動價值並補充核心基因編輯技術能力。
As we shared today, we are making significant progress in all three pillars of our strategy this quarter, including red cell in vivo and business development, including intellectual property. We entered 2024 with great momentum, and I am proud of the Editas team significant progress towards becoming a commercial-stage company and on developing clinically differentiated transformational medicines for people living with serious previously untreatable diseases. As always, we could not achieve our objectives without support of our patients, caregivers, investigators, employees, corporate partners, and you.
正如我們今天分享的那樣,本季度我們在策略的所有三大支柱方面都取得了重大進展,包括體內紅血球和業務發展,包括智慧財產權。我們以強勁的勢頭進入 2024 年,我為 Editas 團隊在成為一家商業階段的公司以及為患有嚴重的以前無法治療的疾病的人們開發臨床差異化轉化藥物方面取得的重大進展感到自豪。一如既往,如果沒有患者、照護者、研究人員、員工、企業合作夥伴和您的支持,我們就無法實現我們的目標。
Thanks very much for your interest and attention, and we're happy to answer questions.
非常感謝您的興趣和關注,我們很樂意回答問題。
Operator
Operator
(Operator Instructions) Samantha Semenkow, Citi.
(操作員指示)Samantha Semenkow,花旗銀行。
Samantha Semenkow - Analyst
Samantha Semenkow - Analyst
Hi, good morning and thanks very much for taking my questions. And I'm wondering about your in vivo pipeline and the proof of concept that you're expecting by the end of this year, a what will be the bar for success that you're looking for in this program?
你好,早安,非常感謝你回答我的問題。我想知道你們的體內管道以及你們期望在今年年底獲得的概念驗證,你們在這個計畫中尋求成功的標準是什麼?
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thank you.
謝謝。
Thanks very much, Samantha. I'm going to ask Linda to address that.
非常感謝,薩曼莎。我要請琳達解決這個問題。
Linda Burkly - EVP, Chief Scientific Officer
Linda Burkly - EVP, Chief Scientific Officer
Thank you, Sam. Thank you for the question. We are looking for a proof of concept for high efficiency delivery and editing for our target of interest in vivo in this preclinical POC that will give us confidence in our ability to target the target of interest. We are going to be sharing more information on this at a future date.
謝謝你,山姆。感謝你的提問。我們正在臨床前 POC 中尋找體內感興趣標靶的高效傳遞和編輯的概念證明,這將使我們對靶向感興趣靶標的能力充滿信心。我們將在未來分享更多相關資訊。
Thank you for the question.
感謝你的提問。
Operator
Operator
Joon Lee, Truist Securities.
李俊,Truist 證券公司。
Joon Lee - Analyst
Joon Lee - Analyst
And congrats on the progress and thanks for taking our question. An interesting update on your disclosure. Is there a plan to identify large ceiling combinations, which implies sort of a newer approach to many of you that many of the peers are also developing some call it team adding some call it pasting does the size of the Company's media and insertion allow for you starting a coding sequence for dystrophin, for example, and are you able to comment on whether TMD is out of the question regarding your in vivo aspirations? Thank you.
恭喜您的進展,並感謝您提出我們的問題。關於您的披露的一個有趣的更新。是否有計劃確定大型天花板組合,這對你們許多人來說意味著一種更新的方法,許多同行也在開發一些稱為團隊的添加一些稱為粘貼的公司媒體和插入的大小是否允許您例如,開始抗肌萎縮蛋白的編碼序列,您能否評論一下TMD 是否不符合您的體內願望?謝謝。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much, June. I'm going to have Linda sector.
非常感謝,六月。我將擁有琳達部門。
Linda Burkly - EVP, Chief Scientific Officer
Linda Burkly - EVP, Chief Scientific Officer
Yes, thank you for commenting. We're excited about the LSRI. technology that we're disclosing here. We are identifying many different novel LSR. We're not disclosing at the moment. The size of the integrations that can be accommodated by these large Jeremy companies is, but we have quite a few novel LSR.s that we've identified and we're further characterizing them. And thank you for the question.
是的,謝謝您的評論。我們對 LSRI 感到非常興奮。我們在這裡公開的技術。我們正在鑑定許多不同的新型 LSR。我們目前不便透露。這些大型傑里米公司可以容納的整合規模是,但我們已經確定了相當多的新穎 LSR,並且我們正在進一步描述它們的特徵。謝謝你的提問。
Operator
Operator
Mary Kate Davis, Bank of America.
瑪麗·凱特·戴維斯,美國銀行。
Mary Kate Davis - Analyst
Mary Kate Davis - Analyst
Good morning. Thanks for taking my question. I'm looking at the red cell program here. How are you guys looking at the midyear? Any follow-up data compared to the year-end update here as far as time progresses, what should we look for from treated patients in terms of safety and efficacy moving forward.
早安.感謝您提出我的問題。我正在看這裡的紅血球計畫。大家對年中的事情怎麼看?隨著時間的推移,與年終更新相比的任何後續數據,我們應該從治療患者的安全性和有效性方面尋找什麼。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thank you.
謝謝。
Thanks, Mary Kay song is going to take that one.
謝謝,玫琳凱的歌曲會選擇那一首。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Thanks, Mary. And so we're in the middle year, our release we expect to have at least 18 patient data for Ruby study and within the 18 patients, and 4 of them will have 12 or longer 12 to 21 months of exposure and seven were out 5 months to 12 months exposure, another 7 with 2 to 5 months exposure. With that, we are we feel this data is very meaningful to see that direction, not only the increase of total hemoglobin normalization of our top total hemoglobin increase of fetal globin.
謝謝,瑪麗。因此,我們正處於年中,我們的發布預計將有至少18 名患者數據用於Ruby 研究,並且在18 名患者中,其中4 名將有12 或更長的12 至21 個月的暴露時間,7名超出 5幾個月到 12 個月的暴露時間,另外 7 個月的暴露時間為 2 到 5 個月。有了這個,我們覺得這個數據非常有意義,看到了這個方向,不僅是總血紅蛋白的增加,我們頂部總血紅蛋白的正常化還有胎兒珠蛋白的增加。
But also the durability of the study and impact our from an efficacy perspective for, for example, the free of big vessel currency events for the Adacel study, we'll have patients perform at least seven. Those patients. We have four to 12 months of exposure, which also where we have a meaningful variance compared to the December release, we have 11 will be patient and six editorial patients. So their substantial more data will help us to understand the program much better.
但從功效角度來看,研究的持久性也會影響我們,例如,在 Adacel 研究中,沒有大血管貨幣事件,我們會讓患者執行至少七次。那些病人。我們有 4 到 12 個月的曝光時間,與 12 月發布的版本相比,我們有一個有意義的差異,我們有 11 名將是患者和 6 名編輯患者。因此,他們提供的大量數據將幫助我們更好地理解該程式。
Operator
Operator
Jack Allen, Baird.
傑克艾倫、貝爾德。
Jack Allen - Analyst
Jack Allen - Analyst
And congratulations to the team on the progress, and thanks for taking the question. I'm going to stick with the red cell program. I was hoping you could provide some color on dosing of the pivotal cohort. I know you've commented on the adult cohort being fully enrolled but have all those patients receive therapy.
恭喜團隊的進展,並感謝您提出問題。我將堅持紅血球計劃。我希望你能提供一些關於關鍵隊列的劑量的資訊。我知道您曾評論過成人隊列已完全入組,但所有這些患者都接受了治療。
And any other comments you can provide as it relates to the size of the cohort that you've agreed to with regulators would be very helpful. Thanks so much.
您可以提供的與您與監管機構同意的群體規模相關的任何其他評論都會非常有幫助。非常感謝。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much, Jack. I'll answer the first part and then pass it to Basil to back, you know, maybe give us some more color to regulatory interactions we have obviously completed the adult enrollment, which are actually very excited about not least because that is in the context of two approved therapy. So it really is a very concrete reflection of the enthusiasm that they saw has found and described, indeed, increasing enthusiasm about our program with his visits to sites and conversations with investigators.
非常感謝,傑克。我將回答第一部分,然後將其傳遞給巴茲爾,你知道,也許會給我們更多的監管互動色彩,我們顯然已經完成了成人註冊,這實際上非常令人興奮,尤其是因為這是在上下文中兩種已批准的療法。因此,這確實非常具體地反映了他們所看到和描述的熱情,事實上,透過他對現場的訪問以及與調查人員的對話,人們對我們的專案的熱情不斷增加。
Our health care providers and indeed with patient advocacy groups, Tom, we have scheduled that many of those patients already for dosing, and we'll give you further updates on the progress of dosing at a later date. And with regard to the regulatory color, I think Basil, you might want to maybe just share a little more of that.
我們的醫療保健提供者以及患者倡導團體湯姆,我們已安排其中許多患者接受給藥,我們將在稍後向您提供有關給藥進展的進一步更新。關於監管顏色,我認為 Basil,你可能想分享更多。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Yes, for regulatory, as we shared that, we have alignment with FDA that there will be study is a Phase one two three study for PMA filing. And we also have alignment on the sample size, duration and study design of that. So we continue to have collaboration with FDA on the further discussion about this progress.
是的,對於監管而言,正如我們所分享的那樣,我們與 FDA 保持一致,將進行一項用於 PMA 備案的第一、二、三期研究。我們也就樣本量、持續時間和研究設計進行了協調。因此,我們將繼續與 FDA 合作,進一步討論這項進展。
Jack Allen - Analyst
Jack Allen - Analyst
Great. Thank you.
偉大的。謝謝。
Operator
Operator
Gena Wang, Barclays.
王吉娜,巴克萊銀行。
Gena Wang - Analyst
Gena Wang - Analyst
Thank you. If I may very quick two questions. First, should we read into your ASGCT. presentation for in-vivo indication such as glaucoma? And second, I know you mentioned also a little bit. But when we look at the current approved genomic therapy for sickle cell, we still have a 10% patient relapse with VOD events. What do you think is the key factors that we should look into for the potential of differentiated durability was hopefully 100% control.
謝謝。我可以很快問兩個問題。首先,我們應該了解您的 ASGCT。體內適應症(如青光眼)的介紹?其次,我知道你也提到了一點。但當我們查看目前批准的鐮狀細胞基因組療法時,仍有 10% 的患者因 VOD 事件而復發。您認為我們應該研究的關鍵因素是什麼,以實現差異化耐用性的潛力,希望是 100% 的控制。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much.
非常感謝。
Gena. I'm going to ask Linda to handle the first part of your question around the ASGCT. and then based can talk about durability, what we're seeing to date have been.
吉納。我將請 Linda 處理您關於 ASGCT 的問題的第一部分。然後我們可以談論耐用性,這是我們迄今為止所看到的。
Linda Burkly - EVP, Chief Scientific Officer
Linda Burkly - EVP, Chief Scientific Officer
Thank you very much, Gena, on primary open-angle glaucoma is obviously an area of very high unmet need. And while we conducted those studies because of that, we are not currently pursuing that indication, but we were able through those studies to really demonstrate impressive preclinical POC and showcase our in vivo capabilities with respect to three components are our ability to deliver lipid nanoparticles in vivo with our gene editing cargo, our proven capability of our proprietary enzyme and Kestrel way to edit in vivo and our guide modifications to enhance and gene editing potency. So these capabilities really position us well for delivering in vivo gene editing medicines, and we're thrilled pleased with the ASGCT. disclosures.
非常感謝,Gena,原發性開角型青光眼顯然是一個未滿足需求非常高的領域。雖然我們因此進行了這些研究,但我們目前並未追求這一適應症,但我們能夠透過這些研究真正證明令人印象深刻的臨床前POC,並展示我們在三種成分方面的體內能力,即我們在體內遞送脂質奈米顆粒的能力。因此,這些能力確實使我們能夠很好地提供體內基因編輯藥物,我們對 ASGCT 感到非常高興。披露。
Thank you.
謝謝。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Yes, I can. I will tick up tick up on the question about the BOE. relapse for sickle cell patients. And first, I would like to say I want to congratulate the entire field of effort, the treating sickle cell disease including the recent approval of the two molecules. And so I think what we see is that with the continued effort of all of us, we will continue to improve and transform the treatment for sickle cell disease patients with renal cell.
我可以。我會在有關英國央行的問題上打勾。鐮狀細胞疾病患者復發。首先,我想說我要祝賀整個領域的努力,包括治療鐮狀細胞疾病,包括最近批准的這兩種分子。所以我認為我們看到的是,在我們所有人的持續努力下,我們將不斷改進和改變腎細胞鐮狀細胞疾病患者的治療方法。
And we are still continuing to collect the clinical data. As I mentioned, we expected this is a different not only competitive but differentiated molecule with the normalization of the total hemoglobin correction of pneumonia. So we're looking forward to see our own data on the VOE. as we reported so far we have seen all those patients, those with Renault sale, the three BOEB.
我們仍在繼續收集臨床數據。正如我所提到的,我們預計這不僅是一種不同的競爭性分子,而且是一種差異化分子,可以使肺炎的總血紅蛋白校正正常化。所以我們期待在 VOE 上看到我們自己的數據。正如我們到目前為止所報告的,我們已經見過所有這些患者,那些雷諾銷售的患者,三個 BOEB。
Gena Wang - Analyst
Gena Wang - Analyst
Thank you.
謝謝。
Operator
Operator
Mani Foroohar, Leerink Partners.
Mani Foroohar,Leerink 合夥人。
Mani Foroohar - Analyst
Mani Foroohar - Analyst
Hi, good morning. This is CJ on for money and thanks for taking your question on ours is following the agreement with Vertex. Can you just how you're thinking about future IP monetization opportunity.
早安.這是 CJ 的錢,感謝您提出問題,我們的問題是按照與 Vertex 的協議進行的。您能否談談您對未來 IP 貨幣化機會的看法?
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much. I'm going to ask Eric to address that question.
非常感謝。我要請埃里克回答這個問題。
Erick Lucera - EVP, CFO
Erick Lucera - EVP, CFO
Yes, thanks for the question. Obviously, as we said in the transcript, we view the future potential royalty monetization and licensing activities as an integral and very important source of non-dilutive capital. As you know, these are foundational IP patents which are applied to just about everybody's projects in cash, non-cash 12 and we expect to have conversations with those folks as soon as we can.
是的,謝謝你的提問。顯然,正如我們在文字記錄中所說,我們將未來潛在的特許權使用費貨幣化和許可活動視為非稀釋資本的一個不可或缺的且非常重要的來源。如您所知,這些是基礎智慧財產權專利,幾乎適用於每個人的現金、非現金項目 12,我們希望盡快與這些人進行對話。
Mani Foroohar - Analyst
Mani Foroohar - Analyst
Thank you.
謝謝。
Operator
Operator
Brian Chang from JP Morgan.
摩根大通的布萊恩張。
Brian Chang - Analyst
Brian Chang - Analyst
Hey, guys, thanks for taking our question this morning. Can you just remind us what's your latest thinking around the timing of holding discussion with regulators on sickle cell? And, you know, just given the data that you're going to present mid year. Any update and color on the timing from holding a productive conversation with regulators would be appreciated. Thank you.
嘿,夥計們,感謝您今天早上提出我們的問題。您能否提醒我們您對與監管機構就鐮狀細胞疾病進行討論的時間安排有何最新想法?而且,你知道,只要給出你將在年中提供的數據。如果您能就與監管機構進行富有成效的對話的時間安排提供任何更新和信息,我們將不勝感激。謝謝。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much, Brian. Going to us based on to talk about that sort of. I think your two questions really, which is about what the data are in the middle of the year and how they integrate with our discussions with regulators.
非常感謝,布萊恩。來找我們就是為了談論這樣的事情。我認為你確實有兩個問題,即年中的數據是什麼,以及它們如何與我們與監管機構的討論結合。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
So Brian, we as I mentioned, we are very pleased with the data we're going to release the middle of the year and which is substantive and also give us good direction how much you will we will get and to have, for example, a dataset to have equivalent to the SGVIBOF. filing, for example. So that's kind of one part of that where they happy to see the amount of data and the patient outcome on the data.
所以布萊恩,正如我所提到的,我們對我們將在年中發布的數據感到非常滿意,這些數據是實質性的,也為我們提供了良好的方向,我們將獲得多少,例如,與SGVIBOF等效的數據集。例如,歸檔。因此,這就是他們很高興看到數據量和數據上的患者結果的一部分。
And then the other thing is by the regulatory engagement. As I mentioned, we already have a line of this is Phase three study to support the BLA. And we have then we have our continued engagement with the with FDA. We have not disclosed all those details of the interaction yet.
另一件事是監管參與。正如我所提到的,我們已經有一系列第三階段研究來支持 BLA。然後我們繼續與 FDA 合作。我們尚未透露互動的所有細節。
But as a reminder, we have our MET explanation and which allow us to have frequent interaction with agency, but also with a high level, our interaction with the agency. And this, of course, helps give us opportunity for potential priority review and rolling submission. So we have been excited to the direction we continue to have engagement and cooperation with FDA.
但提醒一下,我們有 MET 解釋,這使我們能夠與代理商進行頻繁的互動,並與代理商進行高水準的互動。當然,這有助於為我們提供潛在的優先審查和滾動提交的機會。因此,我們對繼續與 FDA 接觸和合作的方向感到興奮。
Operator
Operator
Eric Schmidt, Cantor Fitzgerald.
埃里克·施密特,康托·菲茨傑拉德。
Eric Schmidt - Analyst
Eric Schmidt - Analyst
Thanks for the question. And congrats on the progress on. Are you able to give the approximate number of patients who were enrolled in the Ruby trial with sickle cell disease? And then it sounds like you've been able to make pretty good progress in enrollment in that study despite the availability of commercial cell therapies. Just wondering if at centers that have both experimental and commercial cell therapy available may be based on could talk a little bit about what's driving the decision to use the Editas product over others. Thanks it.
謝謝你的提問。並祝賀取得的進展。您能否提供參加 Ruby 試驗的鐮狀細胞疾病患者的大概人數?聽起來,儘管有商業細胞療法,但您在該研究的入組方面取得了相當大的進展。只是想知道那些同時擁有實驗性和商業性細胞療法的中心是否可以談論一下是什麼促使我們決定使用 Editas 產品而不是其他產品。謝謝它。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much, Eric. And based on we can update you on where each of our clinical trials, trials.gov set as a target for the cohort in our trial and then obviously build on his perceptions of why we're doing so well with enrollment even in the context of commercial therapies being available.
非常感謝,埃里克。基於我們可以向您提供的最新信息,Trials.gov 為我們的試驗中的隊列設定了目標,然後顯然基於他對為什麼我們在入組方面做得如此出色的看法,即使在這樣的背景下商業療法可用。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Thanks, Kyle. We are very pleased with the momentum by the enrollment in both the adult cohort and the adolescent cohort. And for the adult cohort, we and we shared that in February, we dosed We enrolled 40 patients now in or slightly more than 40 patients and therefore, we close the the adult cohort you employment. And for adolescent cohort, we started like beginning of this year, we already enrolled a modification and have modification and screenings were very pleased with that.
謝謝,凱爾。我們對成人隊列和青少年隊列的入學勢頭感到非常高興。對於成人隊列,我們在 2 月分享了這一點,我們現在招募了 40 名患者或略多於 40 名患者,因此,我們關閉了您僱用的成人隊列。對於青少年隊列,我們從今年年初開始,我們已經註冊了一項修改,並進行了修改,篩選對此非常滿意。
Then, as I mentioned, I'm on the road all the time and to visit our investigators and the study size. And then they really feel that one is actually there, I believe on the Renault sale based on the MOA based on the data, we have continued to sharing on that. I also do credit the entire field working on that with a two gene therapy approved for sickle cell is also increased the interest in the direction of the gene therapy for sickle cell disease. So that's how we see you know, over the last year or so, we see really great momentum that for Renaissance enrollment, especially after we release our data.
然後,正如我所提到的,我一直在路上拜訪我們的研究人員和研究規模。然後他們真的覺得確實存在,我相信基於數據的 MOA 的雷諾銷售,我們一直在繼續分享這一點。我還相信整個領域的工作,批准用於鐮狀細胞疾病的兩種基因療法也增加了人們對鐮狀細胞疾病基因療法方向的興趣。這就是我們所看到的,在過去一年左右的時間裡,我們看到了文藝復興招生的巨大勢頭,特別是在我們發布數據之後。
Operator
Operator
Jay Olson Oppenheimer & Company,
傑·奧爾森·奧本海默公司,
Jay Olson - Analyst
Jay Olson - Analyst
Congrats on all the progress, and thank you for taking the question. Can you talk about the timing of the collaboration extension with Bristol? Was there some new data that that triggered the new collaboration? And is there any color on what new data Bristow may have seen? And then And separately, can you talk about any work that you've done on developing a milder conditioning agent.
恭喜所有進展,並感謝您提出問題。能談談與布里斯托延長合作的時間嗎?是否有一些新數據觸發了新的合作?布里斯托可能看到了哪些新數據?然後,您能單獨談談您在開發更溫和的調理劑方面所做的工作嗎?
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
And what I want to do is ask Eric to address the question about the BM. What I do want to say I can address the conditioning, which is that just at our last earnings call, we talked that we are going to continue monitoring the space. We have significant contacts in the academic and nonacademic world around the field, but we have actually really deployed our efforts and our resources internally to focusing on our in vivo pipeline, including developing hematopoietic stem cells.
我想做的是請 Eric 解決有關 BM 的問題。我想說的是,我可以解決這個問題,那就是,就在我們上次的財報電話會議上,我們談到我們將繼續監控這個領域。我們在該領域的學術和非學術界擁有重要的聯繫,但我們實際上在內部部署了我們的努力和資源,專注於我們的體內管道,包括開發造血幹細胞。
The rationale for that being that we see that where a milder conditioning therapy is actually approved, it would be used universally adopted universally in transplant centers across multiple indications, including on stem cell transplantation. Hemoglobinopathies. And with that software might I'm just going to pass to Eric just to talk about the BMS I deal.
這樣做的理由是,我們看到,如果溫和的調理療法實際上獲得批准,它將在移植中心普遍採用,涵蓋多種適應症,包括幹細胞移植。血紅蛋白病。有了這個軟體,我可能會轉給 Eric 來談談我處理的 BMS。
Erick Lucera - EVP, CFO
Erick Lucera - EVP, CFO
Yes, thanks for the question. With respect to the timing of the renegotiation or the extension. Obviously, we put out a press release in the very recent past a week or two ago, something like that. That would give you an update on the timing, I would say with respect to the data, Bristol Myers, as you know, recently completed a portfolio review and we were pleased to see that all of the projects that we're working on them are continuing to move forward. I think we would leave discussion of specific programs to them to talk about a thing that they're seeing in those programs.
是的,謝謝你的提問。關於重新談判或延期的時間。顯然,我們在一兩週前發布了一份新聞稿,類似的事情。這將為您提供最新的時間安排,我想說的是,就數據而言,正如您所知,百時美施貴寶最近完成了投資組合審查,我們很高興看到我們正在進行的所有項目都在繼續前進。我認為我們會將具體計劃的討論留給他們來討論他們在這些計劃中看到的事情。
I would highlight the fact that at their most recent R&D Day last September, which I think was the first one they've done in several years, they did mention six products on their pipeline chart which are using our technology. So I would refer you to their R & D disclosures from that meeting to get an update on the work that they're doing with us, but we are very excited about working with them. This has been a partnership that has survived several mergers and several portfolio reviews. So we're very excited about what we're seeing Thank you.
我要強調的事實是,在去年九月他們最近的研發日(我認為這是他們幾年來的第一次研發日)上,他們確實在其管道圖表上提到了六種使用我們技術的產品。因此,我建議您參閱該會議上他們披露的研發信息,以了解他們與我們合作的最新工作,但我們對與他們合作感到非常興奮。這種夥伴關係經歷了多次合併和多次投資組合審查而倖存下來。所以我們對所看到的感到非常興奮,謝謝。
Operator
Operator
Phil Nadeau, TV Cowen.
菲爾‧納多,考恩電視台。
Unidentified Participant
Unidentified Participant
This is Alex on for Phil. Thanks for taking my question. So given the association between total hemoglobin hemoglobin levels and organ function. Do you plan to utilize any quantitative endpoints on basically assessing and organ function in the REVEAL trial? And if so, what might those look like? And when could we maybe expect initial data? Thanks.
這是亞歷克斯為菲爾代言的。感謝您提出我的問題。鑑於總血紅素血紅素水平與器官功能之間的關聯。您是否計劃在 REVEAL 試驗中使用任何定量終點來基本評估和器官功能?如果是這樣,那些會是什麼樣子?我們什麼時候可以得到初始資料?謝謝。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much, Alex. I'm going to ask based on to address that question.
非常感謝,亞歷克斯。我將根據這個問題來提問。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Yes, thanks, Alex. We certainly have measurements for the end organ function are we looking to the several major organ system to monitor the function improvement? For example, we monitor the liver function, not only with these different lab values. We're looking to our pulmonary function to check to check the the respiratory system.
是的,謝謝,亞歷克斯。我們當然有終末器官功能的測量,我們是否希望透過幾個主要器官系統來監測功能的改善?例如,我們不僅使用這些不同的實驗室值來監測肝功能。我們正在檢查肺功能以檢查呼吸系統。
And we are we also have cardio ankle and other measures. You're talking to measure their cardiovascular system and that too. So we are looking forward to see more data on that and give us more understanding of the end organ. It's a function may be may behave after the treatment Just a reminder that we also, of course, look into that not only sickle cell but also other area in this in terms of the anemia, how that impact a function and how that correcting anemia may be able to improve that assumption after the after treatment in sickle cell.
我們還有有氧踝關節和其他措施。你說的是測量他們的心血管系統等等。因此,我們期待看到更多這方面的數據,讓我們對末端器官有更多的了解。這是一種治療後可能表現出來的功能,只是提醒一下,當然,我們不僅研究鐮狀細胞,還研究貧血方面的其他領域,它如何影響功能以及糾正貧血可能如何在鐮狀細胞病治療後能夠改善這一假設。
Specifically over the last couple of years, you already see more publications about end organ function even after the allogenic transplant to treating sickle cell patients. We are very excited on that. But just to be very honest to ourselves, right, this field is still fairly new and we see some really good publication and direction in this, and we're looking forward to our study as well as the literature on this field.
特別是在過去幾年中,即使在同種異體移植治療鐮狀細胞患者之後,您也已經看到了更多有關終末器官功能的出版物。我們對此感到非常興奮。但老實說,這個領域仍然相當新,我們在這方面看到了一些非常好的出版物和方向,我們期待我們的研究以及該領域的文獻。
Unidentified Participant
Unidentified Participant
Okay, thank you.
好的謝謝。
Operator
Operator
Yanan Zhu, Wells Fargo.
朱亞楠,富國銀行。
Yanan Zhu - Analyst
Yanan Zhu - Analyst
And thanks for taking our questions. So first, on the differentiation of total hemoglobin normalization, I was wondering, have you had feedback from sickle cell treaters on that differentiation and whether there's any hesitancy or pushback that perhaps the current level of hemoglobin achieved by the marketed product is sufficient and what how much of that kind of thinking is it out there? And on the in vivo side, was wondering are you focused on first-in-class targets or perhaps I not first-in-class targets, but hoping to have a differentiation on specificity and transduction efficiency, et cetera.
感謝您回答我們的問題。首先,關於總血紅蛋白正常化的差異,我想知道您是否從鐮狀細胞治療者那裡得到了關於這種差異的反饋,以及是否存在任何猶豫或阻力,也許上市產品目前達到的血紅蛋白水平是否足夠以及如何是不是有很多這樣的想法?在體內方面,我想知道您是否專注於一流的目標,或者也許我不是一流的目標,但希望在特異性和轉導效率等方面有差異。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much. And I'm so I'm going to ask a song to talk about the differentiation of total hemoglobin. And then I'll ask Linda, just to talk about our approach to first in class or clear differentiation and clear that we would see that with our approach to function of regulation. Thanks.
非常感謝。我想請一首歌來談談總血紅素的差異。然後我會問琳達,只是談談我們實現一流或明確差異化的方法,並明確我們將透過我們的監管功能方法看到這一點。謝謝。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Thanks, Alan. I mean, certainly we talk about the investigators as well, the KOLs and sickle cell treaters for differentiation and how we may be able to see and with the position of this molecule. And when we're talking to a loosely hematologist and sickle cell treaters, they see our data. And when once the hematologist mentioned that it is a no-brainer that it's better if you have a 16 gram, but that's the leader versus 10 gram, but definitely the of total hemoglobin.
謝謝,艾倫。我的意思是,我們當然也會談論研究人員、KOL 和鐮狀細胞分化治療師,以及我們如何能夠看到這種分子的位置。當我們與鬆散的血液學家和鐮狀細胞治療師交談時,他們會看到我們的數據。有一次血液學家提到,毫無疑問,如果你有 16 克會更好,但那是領先者與 10 克,但絕對是總血紅蛋白。
And then they also I mentioned the enthusiasm of our study and they have finally been knowledgeable hematologist. They see the difference among several molecules. And we investigator said that he was waiting for our trial and we did not anticipate other. So those other anecdotal examples on that. As I mentioned earlier, we certainly want to look forward to see the clinical data.
然後他們也提到了我們研究的熱情,他們終於成為了知識淵博的血液學家。他們看到了幾個分子之間的差異。我們調查員說他正在等待我們的審判,我們沒有預料到其他。關於這一點的其他軼事例子。正如我之前提到的,我們當然希望看到臨床數據。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
It's also worth highlighting, of course, that the FDA has recognized that a one gram per deciliter difference is meaningful or certainly likely to predict a clinically meaningful benefit in it. They used that threshold to be of an expenditure approvals to Oxbryta in the past Yes.
當然,也值得強調的是,FDA 已經認識到每分升一克的差異是有意義的,或者肯定有可能預測其中具有臨床意義的益處。他們過去使用該閾值作為 Oxbryta 支出批准的門檻。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
And talking about that at least is also when we communicate with FDA, this is also appointed. We have been discussion with FDA.
至少在我們與 FDA 溝通時,這也是指定的。我們一直在與 FDA 進行討論。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
So thanks very much based on, Linda, just VRD in vivo and where our focus is.
非常感謝 Linda,體內的 VRD 以及我們的重點。
Linda Burkly - EVP, Chief Scientific Officer
Linda Burkly - EVP, Chief Scientific Officer
Yes, in vivo, our in vivo approaches aimed at functional upregulation of gene expression, genetically determined diseases. And this strategy positions us very well to be differentiated from others in terms of our targets and our target editing strategies.
是的,在體內,我們的體內方法旨在基因表現的功能上調,即遺傳決定的疾病。這項策略使我們在目標和目標編輯策略方面與其他公司區分開來。
And what this means is that we can go after targets that others can't go after and so from an indication perspective, we can go after indications that perhaps others can go after. And so we could have a first-in-class strategy also within a given indication, we could devise a targeted strategy that would be it's best in class, if you will. So we can have first-in-class strategies as well as best in class opportunities. I hope that answers here.
這意味著我們可以追求別人無法追求的目標,因此從跡象的角度來看,我們可以追求別人可能可以追求的跡象。因此,我們可以在給定的適應症範圍內製定一流的策略,如果您願意的話,我們可以設計一個有針對性的策略,這將是同類中最好的。因此,我們可以擁有一流的策略和一流的機會。我希望答案在這裡。
Yanan Zhu - Analyst
Yanan Zhu - Analyst
Yes, thank you. Very helpful. Thanks for all the answers.
是的,謝謝。很有幫助。感謝所有的答案。
Operator
Operator
Luca Issi, RBC Capital.
盧卡·伊西,加拿大皇家銀行資本。
Luca Issi - Analyst
Luca Issi - Analyst
And congrats on the progress. And maybe just a quick one on red cell and the filing strategy. What's the vision here for DNA? Are you planning to file adults and adolescents concurrently or sequentially. Any color there?
並祝賀取得的進展。也許只是簡單介紹一下紅血球和歸檔策略。DNA 的願景是什麼?您打算同時或依序提交成人和青少年的申請嗎?那裡有什麼顏色嗎?
Much appreciated. And then maybe quickly on the Middle East, can you just talk about the opportunity for sickle cell disease in the Middle East on Vertex? Seems really, really excited about that market. So wondering what's your strategy there to potentially tap that market? Are you still focused on partnership? Can you potentially access that via distributor again, any thoughts there much appreciated. Happy to department.
非常感激。然後也許很快就會談到中東,您能在 Vertex 上談談中東鐮狀細胞疾病的機會嗎?看起來對這個市場真的非常非常興奮。那麼想知道您有什麼策略來潛在地開拓該市場?您仍然專注於合作嗎?您是否可以再次透過經銷商存取該內容,非常感謝您的任何想法。快樂部門。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much, Luca. So I'll ask based on just talk about our regulatory strategy as far as we have shared it. And then Karen can talk about just how we're looking at and thinking about the Middle East and frankly, in the context of the rest of world.
非常感謝,盧卡。因此,我會根據我們分享的監管策略來詢問。然後凱倫可以談談我們如何看待和思考中東,坦白說,在世界其他地區的背景下。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Yes. Yes. Thanks, Luka up, we are very pleased with the interaction with the agency and continued interaction with the agency about the rental sale. New for the there will be study as a Phase three study to support the BLA and of all the front of the Phase three study.
是的。是的。謝謝,盧卡,我們對與該機構的互動以及與該機構就租賃銷售的持續互動感到非常滿意。新的研究將作為第三階段研究來支持 BLA 和第三階段研究的所有前沿。
We have alignment of that and we have continuously a conversation with FDA on this route. We have not shared the specifics about the date of the BLA or the indication of it out of a alone or adolescent. But as we shared, we are very pleased with the enrollment for close our cohort as well as adolescent cohort. So that give us a great position for this market.
我們對此保持一致,並就這一途徑不斷與 FDA 進行對話。我們尚未透露有關 BLA 日期的具體信息,也沒有透露來自單身或青少年的 BLA 指示。但正如我們所分享的,我們對接近我們的隊列以及青少年隊列的註冊感到非常高興。這使我們在這個市場上佔據了有利的地位。
Luca Issi - Analyst
Luca Issi - Analyst
Thanks.
謝謝。
Linda Burkly - EVP, Chief Scientific Officer
Linda Burkly - EVP, Chief Scientific Officer
Great, Luca, thanks for the question about Middle East and certainly just the populations outside the United States. And there's absolutely a number of geographies where there is a significant population of sickle cell patients and really high unmet need of beta-thalassemia as well.
太好了,盧卡,感謝您提出有關中東以及美國以外人口的問題。絕對有許多地區存在大量鐮狀細胞疾病患者,而且β-地中海貧血的需求也未滿足。
What I'd say is our continued drive to execute in the U.S. and to move ourselves forward with differentiation just continues to support the opportunity for us to partner at the appropriate time. And that's certainly something that we've said we can we are open to, and we'll certainly provide more more color in the future as appropriate.
我想說的是,我們持續努力在美國執行任務,並以差異化的方式推動自己前進,這將繼續為我們在適當的時候提供合作機會提供支持。我們已經說過我們可以對此持開放態度,並且我們肯定會在未來酌情提供更多顏色。
Luca Issi - Analyst
Luca Issi - Analyst
Thanks so much.
非常感謝。
Operator
Operator
Steve Seedhouse, Raymond James.
史蒂夫席德豪斯,雷蒙德詹姆斯。
Unidentified Participant
Unidentified Participant
Thank you. This is Nick on for Steve. We actually have a longer-term question. To what extent can you leverage the infrastructure that Vertex is already building out for cash given will will added through I won't be able to plug into the existing authorized treatment centers once launched. Thank you.
謝謝。這是尼克替史蒂夫發言。我們實際上有一個更長期的問題。您可以在多大程度上利用 Vertex 已經用現金建造的基礎設施,因為一旦啟動,我將無法插入現有的授權治療中心。謝謝。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much and thanks very much. And Nick, I'm going to ask Karen to address that.
非常感謝,非常感謝。尼克,我要請凱倫解決這個問題。
Caren Deardorf - EVP, Chief Commercial and Strategy Officer
Caren Deardorf - EVP, Chief Commercial and Strategy Officer
Thank you, Nick, for the question. In a first, we'd say I'd say that I'm really pleased to see some of the initial progress for the other therapies and being able to get patients started. And we always anticipated that it would be a debate, many centers starting to dip their toes on as they build the infrastructure and they gain the confidence. So to answer your question, absolutely, we've always said that in this kind of market of the complexity of the ex vivo being a fast follower is absolutely an advantage.
謝謝尼克提出的問題。首先,我想說我真的很高興看到其他療法取得一些初步進展並且能夠讓患者開始使用。我們一直預計這將是一場辯論,許多中心在建設基礎設施時開始涉足並獲得信心。因此,為了回答你的問題,我們總是說,在這種複雜的市場中,快速追隨者絕對是一個優勢。
And we we also, on our own have a really strong base of over 20 clinical sites in the U.S. was very strong enrollment and relationships that we're leveraging and those relationships and the guidance they're giving us will be really pivotal for us as well. But this is a field that will benefit from the increase in education, which patients which based on mentioned also helps with our enrollment and just building the infrastructure. But we are engaged on the KOL, the patient advocacy as well as on the payer front on to ensure that we're prepared. Thanks for the questions.
我們自己也在美國擁有 20 多個臨床中心,擁有非常強大的基礎。但這是一個將受益於教育增加的領域,基於上述的患者也有助於我們的入學和基礎設施建設。但我們致力於 KOL、病患倡議以及付款人方面的工作,以確保我們做好準備。感謝您的提問。
Unidentified Participant
Unidentified Participant
Thank you.
謝謝。
Operator
Operator
Jack Allen, Baird.
傑克艾倫、貝爾德。
Jack Allen - Analyst
Jack Allen - Analyst
Great. Thanks so much for taking the quick follow-up here. I just wanted to touch on Renaissance one more time and ask when we might hear a little bit more about the differentiation as it relates to the treatment process. Have you provided any color on the number of E. three cycles and how editing efficiency of the cash flow the enzyme may allow you to be more efficient in manufacturing the process.
偉大的。非常感謝您在這裡進行快速跟進。我只是想再次談談文藝復興,並詢問我們什麼時候可以聽到更多關於與治療過程相關的差異化的信息。您是否提供了有關 E. 三個循環次數的任何顏色,以及酵素的現金流編輯效率如何使您在製造過程中更加高效。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
Thanks very much, Jack. So I'm going to ask Basil to cut.
非常感謝,傑克。所以我要請巴茲爾來剪。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Yes, then thank you, Al. We are, but we have not share specifics about the number of cycling the freezes when I mentioned before, Wes, since I joined, and we actually work together with our internal as well as a free seats, extra external freezes expert who actually have critical amendment to improve the process cycle and also provide assistance to study sites two for the PC cycle, which is significant because it's a reduced the patient burden is a smoother manufacturing process. We are very pleased to see the progress in that front. And just to add on that, we are hoping this clinical experience will be very much helpful for commercial and Dubai.
是的,那麼謝謝你,艾爾。我們是,但我們沒有分享關於循環凍結次數的具體信息,當我之前提到時,韋斯,自從我加入以來,我們實際上與我們的內部以及空閒席位、額外的外部凍結專家一起工作,他們實際上擁有關鍵的能力修正案旨在改進製程週期,並為 PC 週期的兩個研究中心提供幫助,這一點意義重大,因為它減少了患者負擔,使製造流程更加順暢。我們很高興看到這方面的進展。除此之外,我們希望這項臨床經驗將對商業和杜拜非常有幫助。
Operator
Operator
Mani Foroohar from Leerink Partners.
Leerink Partners 的 Mani Foroohar。
Mani Foroohar - Analyst
Mani Foroohar - Analyst
Thank you for taking a follow-up question kind of similar to the last question. You've previously talked about optimizing the vein-to-vein process. Would you be able to walk us through how when still could provide advantages from either both operational and logistics perspective? Thank you.
感謝您提出與上一個問題類似的後續問題。您之前討論過優化靜脈到靜脈的過程。您能否向我們介紹何時仍可以從營運和物流角度提供優勢?謝謝。
Gilmore OâNeill - CEO, President
Gilmore OâNeill - CEO, President
So based on I'm going to ask, I'll ask baseline to address that.
因此,根據我要問的問題,我會要求基線來解決這個問題。
Baisong Mei - EVP, Chief Medical Officer
Baisong Mei - EVP, Chief Medical Officer
Yes. Yes, heavy heavy to our we are over these process really working together that too, with the study size to optimize these process and that coming from multiple factors. Some of the factors just mentioned above Reese's. The other factor is the logistics, and we provide support on that.
是的。是的,對我們來說,我們正在對這些過程進行真正的合作,優化這些過程的研究規模來自多個因素。上面里斯提到的一些因素。另一個因素是物流,我們提供這方面的支援。
The first back is HE patient condition, as we know that we are treating this severe sickle cell disease and before that ran into a treatment that can have multiple BOE. per year. And that also can impact the vein-to-vein time.
第一個是 HE 患者的情況,因為我們知道我們正在治療這種嚴重的鐮狀細胞疾病,在此之前遇到了可能有多個 BOE 的治療。每年。這也會影響靜脈到靜脈的時間。
Caren Deardorf - EVP, Chief Commercial and Strategy Officer
Caren Deardorf - EVP, Chief Commercial and Strategy Officer
Manny, this is Karen. I would just add that again, in the fast follower position, one of the things it gives us the opportunity to do is to really understand as the first two therapies are commercialized, what's working, what's not working, what they need to see differently and really making sure that it is tough sets ourselves up as the partner of choice. And so we're working very hard on that, and we'll certainly talk about that at a later time.
曼尼,這是凱倫。我想再次補充一點,在快速追隨者的位置上,它讓我們有機會做的一件事就是真正了解前兩種療法的商業化,什麼有效,什麼無效,他們需要以不同的方式看待什麼以及真正確保這很艱難,讓我們成為首選的合作夥伴。所以我們正在努力解決這個問題,我們稍後會討論這個問題。
Mani Foroohar - Analyst
Mani Foroohar - Analyst
Great. Thanks, Dmitry, for your questions.
偉大的。謝謝德米特里的提問。
Operator
Operator
Ladies and gentlemen, this concludes today's call. Thank you once again for your participation. You may now disconnect.
女士們、先生們,今天的電話會議到此結束。再次感謝您的參與。您現在可以斷開連線。