使用警語:中文譯文來源為 Google 翻譯,僅供參考,實際內容請以英文原文為主
Operator
Operator
Good day and welcome to the Sangamo Therapeutics first-quarter 2024 teleconference call. (Operator instructions)
美好的一天,歡迎參加 Sangamo Therapeutics 2024 年第一季電話會議。(操作員說明)
Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker today, Louise Wilkie, Vice President of Investor Relations and Corporate Communications. Please go ahead.
請注意,今天的會議正在錄製中。現在我想將會議交給今天的發言人,投資者關係和企業傳播副總裁路易斯·威爾基 (Louise Wilkie)。請繼續。
Louise Wilkie - Investor Relations
Louise Wilkie - Investor Relations
Thank you. Good afternoon, everyone. Thank you for joining us on the call today.
謝謝。大家下午好。感謝您今天加入我們的電話會議。
On this call are several members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer; Prathyusha Duraibabu, Chief Financial Officer, Amy Pooler, Head of Research, Nathalie Dubois-Stringfellow, Chief Development Officer.
Sangamo 執行領導團隊的幾位成員參加了本次電話會議,包括執行長 Sandy Macrae; Prathyusha Duraibabu,財務官,Amy Pooler,研究主管,Nathalie Dubois-Stringfellow,首席開發長。
Slides from our corporate presentation can be found on our website, sangamo.com, under the presentations page of the Investors and Media section. This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements related to the therapeutic and commercial potential of our product candidates and engineered capsids and the potential of our next-generation genome engineering technology; the anticipated plans and timelines of Sangamo and our collaborators for regulatory submissions, initiating and conducting clinical trials, and presenting clinical data; advancement of our product candidates; anticipated regulatory submissions; advancement of preclinical programs to the clinic; our strategic reprioritization and reallocation of resources and the anticipated benefits thereof; plans to partner certain of our programs; the sufficiency of our resources, cash runway, and plans to seek additional capital; upcoming catalysts and milestones; and other statements that are not historical facts.
我們公司演示的幻燈片可以在我們的網站 sangamo.com 的「投資者和媒體」部分的演示頁面下找到。本次電話會議包括有關 Sangamo 目前預期的前瞻性陳述。這些聲明包括但不限於與我們的產品候選者和工程衣殼的治療和商業潛力以及我們的下一代基因組工程技術的潛力相關的聲明; Sangamo 和我們的合作者提交監管申請、啟動和進行臨床試驗以及提供臨床數據的預期計劃和時間表;我們的候選產品的進步;預期的監理提交;將臨床前項目推進到臨床;我們對資源的策略優先順序和重新分配及其預期效益;計劃與我們的某些項目合作;我們的資源、現金跑道是否充足,以及尋求額外資本的計畫;即將到來的催化劑和里程碑;以及其他不屬於歷史事實的陳述。
Actual results may differ materially from what we discuss today. These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC, specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2023, supplemented by our quarterly report on Form 10-Q for the quarter ended March 31, 2024, filed with the SEC. The forward-looking statements stated today are made as of this date, and we undertake no duty to update such information except as required by law. Please note that all forward-looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding.
實際結果可能與我們今天討論的結果大不相同。這些陳述受到我們向SEC 提交的文件中討論的某些風險和不確定性的影響,特別是在我們截至2023 年12 月31 日的財年的10-K 表格年度報告中,並由我們的10 -Q 表格季度報告進行了補充截至 2024 年 3 月 31 日的季度已向 SEC 備案。今天所述的前瞻性陳述是截至該日期作出的,除非法律要求,否則我們不承擔更新此類資訊的義務。請注意,有關我們未來計劃和期望的所有前瞻性陳述均取決於我們獲得足夠額外資金的能力。
Now I'll turn the call over to our CEO, Sandy Macrae.
現在我將把電話轉給我們的執行長桑迪·麥克雷 (Sandy Macrae)。
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
Thank you, Louise, and good afternoon to everyone joining the call.
謝謝你,路易絲,祝所有加入電話會議的人下午好。
I'm very pleased to be speaking to you today from the American Society of Cell & Gene Therapy (sic - American Society of Gene & Cell Therapy) annual meeting in Baltimore where we have been presenting important preclinical data from our epigenetic regulators, AAV capsid delivery platform, and next-generation genomic engineering platform.
我很高興今天在巴爾的摩舉行的美國細胞與基因治療學會(原文如此- 美國基因與細胞治療學會)年會上向大家發表講話,會上我們展示了來自我們的表觀遺傳調節因子AAV 衣殼的重要臨床前資料交付平台和下一代基因組工程平台。
These data showcased both the depth of Sangamo's neurology pipeline and the power of our scientific capabilities, which we believe provide strong opportunities to advance programs ourselves and with potential partners. In order to progress these compelling programs, Sangamo must be well capitalized. The leadership team and I have been laser focused on addressing our funding needs.
這些數據展示了 Sangamo 神經病學管道的深度和我們科學能力的力量,我們相信這為我們自己和潛在合作夥伴推進計畫提供了強有力的機會。為了推進這些引人注目的項目,桑加莫必須擁有充足的資本。我和領導團隊一直專注於解決我們的資金需求。
In late March, we were pleased to announce a registered direct offering with institutional shareholders, including an important existing investor that raised approximately $24 million in gross proceeds. This was a significant development, and we are thankful for their support of our science and our mission.
3 月下旬,我們很高興地宣布與機構股東進行註冊直接發行,其中包括一位重要的現有投資者,籌集了約 2,400 萬美元的總收益。這是一項重大進展,我們感謝他們對我們的科學和使命的支持。
That was, however, the first step in our current journey to securing additional funding. I would like to emphasize that we are resolutely focused on building upon this foundation to position Sangamo for long-term success. That continues to be my number one priority.
然而,這是我們目前獲得額外資金的第一步。我想強調的是,我們堅決致力於在此基礎上的發展,使 Sangamo 取得長期成功。這仍然是我的首要任務。
Business development is an important part of these efforts, and I'm pleased with the progress that is being made on this front. We are currently engaged in very encouraging conversations with multiple potential partners across our portfolio, including our Fabry disease program, our novel STAC-BBB engineered capsid, our preclinical neurology product candidates, and our next-generation genome engineering capabilities.
業務發展是這些努力的重要組成部分,我對這方面的進展感到高興。目前,我們正在與我們的產品組合中的多個潛在合作夥伴進行非常令人鼓舞的對話,包括我們的法布瑞氏症計畫、我們的新型STAC-BBB 工程衣殼、我們的臨床前神經病學候選產品以及我們的下一代基因組工程能力。
I understand your desire to hear more concrete news on this front, but we're unable to share more until any potential transaction is finalized. Be assured that we are encouraged by the progress of these discussions and hope to announce news of one or more transactions.
我理解您希望聽到這方面更多具體消息的願望,但在任何潛在交易最終確定之前,我們無法分享更多資訊。請放心,我們對這些討論的進展感到鼓舞,並希望宣布一項或多項交易的消息。
Over recent months, Sangamo has presented important preclinical data that solidify our sharpened focus in neurology, validate our differentiated science, and help contextualize why we made this important decision to dedicate ourselves to addressing neurological disorders.
近幾個月來,Sangamo 提供了重要的臨床前數據,這些數據鞏固了我們對神經病學的敏銳關注,驗證了我們的差異化科學,並幫助我們了解為什麼我們做出這項致力於解決神經系統疾病的重要決定。
In March, we were proud to share remarkable preclinical data from our new intravenously administered neurotrophic AAV capsid, which demonstrated industry-leading blood-brain barrier penetration and brain transduction in nonhuman primates. This novel capsid, STAC-BBB, showed robust penetration of the blood-brain barrier with 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9, an outperformed all other known published capsid variance evaluated in this study.
今年3 月,我們很自豪地分享了我們新型靜脈注射神經營養性AAV 衣殼的顯著臨床前數據,該數據在非人靈長類動物中證明了行業領先的血腦屏障穿透和腦轉導能力。這種新型衣殼STAC-BBB 表現出對血腦屏障的強大穿透能力,與基準衣殼AAV9 相比,神經元中的轉基因表達量高出700 倍,優於本研究中評估的所有其他已知已發表的衣殼變異。
Combined with our potent epigenetic regulation cargo, we showed robust STAC-BBB mediated expression of zinc finger cargo in neurons with potent and widespread repression of the prion and tau genes observed across all key brain areas, illustrating the exciting potential to modify disease progression in prion disease and various tauopathies.
結合我們有效的表觀遺傳調控貨物,我們展示了STAC-BBB 介導的鋅指貨物在神經元中的強大表達,並對所有關鍵大腦區域中觀察到的朊病毒和tau 基因進行了有效和廣泛的抑制,這說明了改變朊病毒疾病進展的令人興奮的潛力疾病和各種tau蛋白疾病。
These data support further advancement of our prion and tau programs, which we are on track for regulatory submission sent to the clinic by the end of 2025. Meanwhile, we continue to advance our lead candidate in chronic neuropathic pain, NaV1.7, which uses an established intrinsically administered capsid toward an IND submission expected in the fourth quarter of this year. We believe our ability to combine potent zinc finger epigenetic regulation payloads with exciting new industry leading capsid delivery technology could unlock significant potential for the treatment of devastating neurological diseases, indications for which delivery of treatments to the central nervous system has historically proved challenging.
這些數據支持我們的朊病毒和 tau 計畫的進一步推進,我們預計在 2025 年底之前將這些計畫提交給診所。同時,我們繼續推進慢性神經病理性疼痛的主要候選藥物 NaV1.7,該藥物使用已確定的內在管理衣殼,預計將於今年第四季度提交 IND 申請。我們相信,我們將有效的鋅指表觀遺傳調控有效載荷與令人興奮的行業領先的衣殼傳遞技術相結合的能力,可以釋放治療毀滅性神經系統疾病的巨大潛力,而向中樞神經系統傳遞治療方法歷來被證明具有挑戰性。
Building on this, we are proud to be presenting 3 platform presentations and 17 posters at this week's ASGCT annual meeting. These presentations showcase the depth of our neurology pipeline, including various applications for zinc finger epigenetic regulation platform, exciting advances in our capsid delivery technology, and the discovery of potentially transformative next-generation integrated technology engineered to enable large scale genome editing.
在此基礎上,我們很自豪能夠在本週的 ASGCT 年會上展示 3 個平台演示和 17 張海報。這些演講展示了我們神經病學管道的深度,包括鋅指表觀遺傳調控平台的各種應用、衣殼遞送技術的令人興奮的進展,以及發現旨在實現大規模基因組編輯的潛在變革性下一代集成技術。
In epigenetic regulation, we have shared advances and zinc finger activators and repressor for the potential treatment of a remarkable range of neurological diseases such as prion disease; tauopathies; Charcot-Marie-Tooth diseases, type 1A and 2A; Dravet Syndrome; SOD1-mediated amyotrophic lateral sclerosis or ALS; Phelan-McDermid syndrome; Parkinson's disease; Angelman syndrome; and many other neurological disorders.
在表觀遺傳調控方面,我們分享了鋅指激活劑和阻遏物的進展,可用於治療朊病毒病等一系列神經系統疾病; tau蛋白病;腓骨肌萎縮症,1A 型和 2A 型;德拉韋綜合症; SOD1介導的肌萎縮側索硬化症或ALS;費蘭-麥克德米德症候群;帕金森氏症;天使症候群;和許多其他神經系統疾病。
We have also demonstrated Sangamo's potent delivery capabilities developed through our AAV capsid engineering platform, SIFTER. Here we have presented additional STAC-BBB findings, including prion and tau repression data achieved via STAC-BBB delivery. We also presented for the first time exciting initial findings for a possible mechanism supporting how STAC-BBB may cross the blood-brain barrier.
我們也展示了 Sangamo 透過我們的 AAV 衣殼工程平台 SIFTER 開發的強大遞送能力。在這裡,我們提出了更多的 STAC-BBB 研究結果,包括透過 STAC-BBB 傳遞獲得的朊病毒和 tau 蛋白抑制數據。我們也首次提出了令人興奮的初步發現,說明了支持 STAC-BBB 如何穿過血腦屏障的可能機制。
Our SIFTER platform is designed to ensure capsid for various routes of administration such as intrathecal and intravenous delivery. SIFTER can also engineer capsids for in-vitro discovery. So in a platform presentation tomorrow, we will present findings from STAC-150, a novel capsid that has been shown to be highly potent in neurons and enables high throughput screening of neurology-focused transcriptional regulators. We anticipate the STAC-150, which we believe manufacturers easily at small scale, will help accelerate the discovery of potent and highly-specific epigenetic regulators.
我們的 SIFTER 平台旨在確保衣殼適用於各種給藥途徑,例如鞘內和靜脈內給藥。SIFTER 還可以設計衣殼以進行體外發現。因此,在明天的平台演示中,我們將介紹STAC-150 的研究結果,這是一種新型衣殼,已被證明在神經元中非常有效,並且能夠高通量篩選以神經病學為中心的轉錄調節因子。我們預計 STAC-150(我們相信製造商很容易小規模生產)將有助於加速有效且高度特異性的表觀遺傳調節劑的發現。
Finally, building on our deep expertise in the protein-DNA interactions derived from our zinc finger platform, we presented for the first time a potentially breakthrough new approach for integrating large sequences of DNA into the genome to potentially treat with a single medicine patients who have unique mutations in the same gene.
最後,基於我們在鋅指平台衍生的蛋白質-DNA相互作用方面的深厚專業知識,我們首次提出了一種潛在突破性的新方法,將大序列DNA整合到基因組中,從而有可能用單一藥物治療患有以下疾病的患者:同一基因的獨特突變。
Precisely integrating large synthetic DNA constructs into a desirable chromosomal site has been the dream for people working in this field for many years. Our Modular Integrase or MINT platform is a versatile protein-guided genome editing method that understands and engineers Bxb1, a serum serine recombinase to insert or replace entire genes and adds to Sangamo's toolbox of editing capabilities. We are hopeful that our MINT platform could be used to correct many disease-causing mutations in a diverse patient population by inserting a correct copy of the gene into its natural locus.
將大型合成 DNA 構建體精確地整合到理想的染色體位點一直是該領域工作人員多年來的夢想。我們的模組化整合酶或MINT 平台是一種多功能蛋白質引導基因組編輯方法,可理解和設計Bxb1(一种血清絲氨酸重組酶),用於插入或替換整個基因,並添加到Sangamo 的編輯功能工具箱中。我們希望我們的 MINT 平台可以透過將正確的基因副本插入其自然位點來糾正不同患者群體中的許多致病突變。
MINTs could be deployed internally for various neurological indications, but also provide potential partnering opportunities both for human disease and in agricultural biotech settings. We are already in active discussions with potential partners about our integrase capabilities and are hopeful that MINT could provide us with another potential non-dilutive funding opportunity.
MINT 可以在內部部署用於各種神經系統適應症,但也為人類疾病和農業生物技術環境提供潛在的合作機會。我們已經在與潛在合作夥伴就我們的整合酶能力進行積極討論,並希望 MINT 能為我們提供另一個潛在的非稀釋性融資機會。
Alongside our presentations in this topic at ASGCT this week, we have also published a manuscript in bioRxiv further outlining these next-generation integrase advancements, which is also available on the publications page of the website. I encourage you to learn more about this exciting development in the field of genomic medicines.
除了本週在 ASGCT 上就此主題進行的演講外,我們還在 bioRxiv 上發表了一份手稿,進一步概述了這些下一代整合酶的進展,該手稿也可以在該網站的出版物頁面上找到。我鼓勵您多了解基因組醫學領域這一令人興奮的發展。
Now looking at our clinical programs, we have made strong advances in the first quarter for our Fabry disease program. Having dosed the final patient in the Phase 1/2 STAAR study of isaralgagene civaparvovec, our investigational gene therapy for the treatment of Fabry disease with 33 patients now dosed, screening, enrollment, and dosing are complete. One additional patient has been able to stop enzyme replacement therapy or ERT, resulting in a total of 14 patients withdrawn from ERT to date. The 4 remaining patients dosed since February 2024, who began the study on ERT already have plans in place to withdraw ER (sic - ERT) treatment at the appropriate time.
現在看看我們的臨床項目,我們的法布瑞氏症項目在第一季取得了巨大進展。在isaralgagene civaparvovec 的1/2 期STAAR 研究中對最後一位患者進行給藥後,我們用於治療法布瑞氏症的研究性基因療法現已對33 名患者進行了給藥,篩選、入組和給藥均已完成。又有一名患者能夠停止酵素替代療法或 ERT,迄今共有 14 名患者退出 ERT。其餘 4 名自 2024 年 2 月起開始接受 ERT 研究的患者已製定計劃在適當的時間撤回 ER(原文如此 - ERT)治療。
At the 20th Annual WORLDSymposium in February, we presented compelling, updated, preliminary clinical data from the STAAR study showing sustained benefit and a differentiated safety profile. These results underscore the program's potential as a single administration treatment for Fabry disease. As a reminder, this quarter, we announced alignment with the US FDA on an abbreviated pathway to potential approval. We've also been granted PRIME eligibility by the European Medicines Agency and ILAP designation by the UK Medicines, and Healthcare products Regulatory Agency. We are engaged in active discussions with potential collaborator partners for our Fabry disease program and continue to defer additional investments in planning for a potential rich registrational trial until a collaboration partnership or financing for this program is secured.
在 2 月舉行的第 20 屆世界研討會上,我們展示了來自 STAAR 研究的令人信服的、最新的初步臨床數據,顯示出持續的益處和差異化的安全性。這些結果強調了該計劃作為法布瑞氏症單次給藥治療的潛力。提醒一下,本季度,我們宣布與美國 FDA 就潛在批准的簡化途徑達成一致。我們還獲得了歐洲藥品管理局授予的 PRIME 資格以及英國藥品和保健產品監管機構授予的 ILAP 資格。我們正在與我們的法布瑞氏症計畫的潛在合作夥伴進行積極的討論,並繼續推遲對潛在的豐富註冊試驗的計劃的額外投資,直到確保該計畫的合作夥伴關係或融資。
Moving to our partner clinical program. We look forward to the pivotal readout expected in mid-2024 in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigator with gene therapy we're developing with Pfizer for patients with moderately severe to severe hemophilia A. Pfizer anticipates submitting a biologics license application and a marketing authorization application in early 2025 if the pivotal readout is supportive.
轉向我們的合作夥伴臨床計劃。我們期待在2024 年中期對giroctocogene fitelparvovec 進行的3 期AFFINE 試驗中獲得關鍵結果,giroctocogene fitelparvovec 是我們與輝瑞合作開發的一種基因療法研究人員,用於治療中重度至重度A 型血友病患者。輝瑞預計將提交生物製劑許可申請如果關鍵數據支援的話,將在 2025 年初提交行銷授權申請。
As a reminder, we are eligible to earn up to $220 million in milestone payments and up to 14% to 20% royalties on potential sales from this program. We ended the quarter with approximately $54 million in available cash and cash equivalents, which includes funds from the aforementioned registered direct offering. We believe these resources; in combination with the cost savings expected from the recent restructurings, workforce reduction, and other potential cost reductions; will be sufficient to fund our planned operations into the third quarter of 2024.
謹此提醒,我們有資格從該計劃的潛在銷售額中獲得高達 2.2 億美元的里程碑付款以及高達 14% 至 20% 的特許權使用費。本季結束時,我們擁有約 5,400 萬美元的可用現金和現金等價物,其中包括上述註冊直接發行的資金。我們相信這些資源;結合最近的重組、勞動力減少和其他潛在成本削減預計可節省的成本;將足以為我們計劃在 2024 年第三季的營運提供資金。
As I outlined earlier, we are actively pursuing a range of different options to raise important additional capital and are encouraged by the business development discussions ongoing across our Fabry program, capsid engineering, and next-generation genome engineering efforts. We believe our company has the science required to potentially transform the lives of patients living with devastating neurological conditions and are committed to raising the funding required as we seek to make our vision a reality.
正如我之前概述的那樣,我們正在積極尋求一系列不同的選擇來籌集重要的額外資金,並受到我們法布里計劃、衣殼工程和下一代基因組工程工作中正在進行的業務發展討論的鼓舞。我們相信,我們公司擁有改變患有毀滅性神經系統疾病的患者的生活所需的科學,並致力於籌集所需的資金,以實現我們的願景。
Operator, please open the lines for questions.
接線員,請打開提問線。
Operator
Operator
(Operator instructions)
(操作員說明)
James Stamos, Jefferies.
詹姆斯·斯塔莫斯,杰弗里斯。
James Stamos - Analyst
James Stamos - Analyst
Hi, this is James on for Maury Raycroft. Congrats on the progress, and thanks for taking our question.
大家好,我是莫里‧雷克羅夫特的詹姆斯。恭喜您的進展,並感謝您提出我們的問題。
I just wanted to ask on where are you with the preclinical and GLP tox studies with STAC-BBB and what are your latest thoughts on which targets or indications would make the most sense to partner or keep in-house?
我只是想問一下,您對 STAC-BBB 的臨床前和 GLP 毒物研究進展如何?
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
We have made one of the reasons that we like STAC-BBB so much is that our manufacturing team have been able to work with it and found that it is very manufacturable. They can take it so far up to 50-liter scale and are going higher. They can use the same purification and assay techniques and that it feels like a capsid that will be able to give us great yield and also be able to use many of the processes in CMC work that really is underappreciated in the importance of capsid selection.
我們如此喜歡 STAC-BBB 的原因之一是我們的製造團隊已經能夠使用它並發現它非常具有可製造性。目前他們的容量可以達到 50 公升,而且還在進一步提高。他們可以使用相同的純化和測定技術,感覺就像衣殼一樣,能夠為我們帶來巨大的產量,並且還能夠使用CMC 工作中的許多過程,而這些過程在衣殼選擇的重要性方面確實被低估了。
We've said publicly that we are moving ahead with tau and with prion. You could imagine the amount of external interest in tau, not just because it's a high unmet medical need but because the capsid gives you the brain penetration and widespread delivery that's needed and our zinc finger repressors are really unique in being able to almost switch off tau in the cells that they can get to.
我們已經公開表示,我們正在推進 tau 蛋白和朊病毒的研究。您可以想像外部對tau 蛋白的興趣有多大,不僅因為它是一個未滿足的醫療需求,還因為衣殼為您提供了所需的大腦滲透和廣泛傳遞,而我們的鋅指抑制蛋白在幾乎能夠關閉tau 蛋白方面確實是獨一無二的在他們可以到達的細胞中。
We are having many discussions with companies about our capsid, and we'll choose wisely, I believe, which ones we partner and which ones we take forward ourselves. What we understand and I want to say this again and repeat what I said in the chosen words is that we understand the need to bring in money to the company. And that's always a balance between the desire that we have to take things forward and the money that's available from many of the partnerships, I'm sure we will achieve in the coming weeks.
我們正在與公司就我們的衣殼進行多次討論,我相信我們會明智地選擇與哪些公司合作以及我們自己推進哪些公司。我們的理解是,我想再說一次並重複我在所選詞語中所說的話,我們理解為公司帶來資金的必要性。這始終是我們推動事情向前發展的願望與許多合作夥伴提供的資金之間的平衡,我相信我們將在未來幾週內實現。
James Stamos - Analyst
James Stamos - Analyst
Great. That's very helpful. And just to go on to like some of the more specialized parts of your ASGCT presentation. Can you talk about the importance of targeting the pons and motor neurons in Alzheimer's? And are there any other companies that you're aware of that have the ability to target these sites?
偉大的。這非常有幫助。只是為了繼續喜歡 ASGCT 演示中的一些更專業的部分。您能談談針對阿茲海默症中的腦橋和運動神經元的重要性嗎?您知道有其他公司有能力定位這些網站嗎?
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
Thank you for your question.
謝謝你的問題。
It's really been a remarkable ASGCT, and the field moves closer to these tools being able to address such important disease. Amy, can you talk a little about this? I know this is one of your passions.
這確實是一次了不起的 ASGCT,而這些工具能夠解決如此重要的疾病又更近了一步。艾米,你能談談這個嗎?我知道這是你的興趣之一。
Amy Pooler - Head of Research
Amy Pooler - Head of Research
Yeah, I would love to. Thank you for the great question.
是的,我很樂意。謝謝你提出這個好問題。
What's really important here is being able to target brain regions that would be traditionally challenging to target using something like a direct injection approach. The pons being part of the brainstem, it's not a region that you would necessarily want to reach using a direct injection, a guided approach, even for the most severe of diseases. So we were so excited to see the biodistribution that was able to be achieved with STAC-BBB, especially for what it means for the tau program. The pons is critical in progressive supranuclear palsy, but also other tauopathies like Alzheimer's disease.
這裡真正重要的是能夠瞄準傳統上使用直接注射方法等方法難以瞄準的大腦區域。腦橋是腦幹的一部分,即使對於最嚴重的疾病,您也不一定希望透過直接注射(引導方法)到達該區域。因此,我們非常高興看到 STAC-BBB 能夠實現生物分佈,特別是它對 tau 計畫的意義。腦橋對於進行性核上性麻痺以及阿茲海默症等其他 tau蛋白疾病也至關重要。
In addition, as you noticed, the motor cortex, which is part of the frontal cortex, we use as an example of how STAC-BBB is able to transduce those cortical neurons. Really, we believe that this kind of widespread brain targeting is critical for halting or slowing the progression of the disease.
此外,正如您所注意到的,運動皮質是額葉皮質的一部分,我們用它作為例子來說明 STAC-BBB 如何能夠轉導這些皮質神經元。事實上,我們相信這種廣泛的大腦靶向對於阻止或減緩疾病的進展至關重要。
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
Thanks, Amy.
謝謝,艾米。
James Stamos - Analyst
James Stamos - Analyst
Great. Thank you for taking our questions. I'll hop back in the queue.
偉大的。感謝您接受我們的提問。我會跳回到隊列中。
Operator
Operator
Gena Wang, Barclays.
王吉娜,巴克萊銀行。
Gena Wang - Analyst
Gena Wang - Analyst
I have two. The first one was about the STAC-BBB, and it seems like very exciting new capsids. What is the highest dose you used in nonhuman primates? And what is the limiting dose toxicity there?
我有兩個。第一個是關於 STAC-BBB,它看起來是非常令人興奮的新衣殼。您對非人靈長類動物使用的最高劑量是多少?那裡的極限劑量毒性是多少?
And then the second question quickly regarding the hem A. I know it's your partner, Pfizer, is in charge, but just wondering for the mid '24 Phase 3 update or would that be just a press release from Pfizer? And then for the $220-million milestone payment, the first milestone is that the drug approval? And is that a big portion of the $220 million?
然後是關於下擺 A 的第二個問題。那麼對於2.2億美元的里程碑付款,第一個里程碑是藥物批准?這是 2.2 億美元中的很大一部分嗎?
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
Gena, thank you for your questions. And I know you've been following hem A for a great deal of time, so it's good to be coming closer to the point where it gets to registration and approval. So let me pass this on to two people. Amy, can you talk about the dose that we've used and whether there was any toxicity, please?
吉娜,謝謝你的提問。我知道您已經關注下擺 A 很長一段時間了,所以很高興能夠更接近註冊和批准的階段。讓我把這個轉告給兩個人。艾米,您能談談我們使用的劑量以及是否有任何毒性?
Amy Pooler - Head of Research
Amy Pooler - Head of Research
Yeah, absolutely.
是的,絕對是。
As part of the STAC-BBB study that we discussed yesterday, and we also will discuss later today at the poster session at ASGCT. We performed a dose-range finding study with our tau and zinc -- clinical lead zinc finger, and we did three different doses, and the top dose was 1e14. We're really happy to see that with the histopathology results that we didn't see any dose-limiting toxicity and really no findings in the brain, the spinal cord, or the liver, or the peripheral organ. So really, really positive and encouraging data.
作為我們昨天討論的 STAC-BBB 研究的一部分,我們也將在今天晚些時候在 ASGCT 的海報會議上進行討論。我們用 tau 蛋白和鋅(臨床鉛鋅指)進行了劑量範圍發現研究,我們進行了三種不同的劑量,最高劑量是 1e14。我們非常高興地看到,根據組織病理學結果,我們沒有看到任何劑量限制性毒性,並且在大腦、脊髓、肝臟或周圍器官中確實沒有發現任何發現。非常非常正面和令人鼓舞的數據。
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
Gena, does that answer your question? Is there any follow-up (multiple speakers)--
吉娜,這能回答你的問題嗎?還有後續嗎(多位發言者)-
Gena Wang - Analyst
Gena Wang - Analyst
Yeah, like more thinking like, other than the dose finding like what is the highest dose you tested for the safety toxicology perspective?
是的,就像更多的思考一樣,除了劑量發現之外,例如您從安全毒理學角度測試的最高劑量是多少?
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
1e14, so 1e14 (multiple speakers)--
1e14,所以 1e14(多個揚聲器)--
Gena Wang - Analyst
Gena Wang - Analyst
That's 1e14. Okay. That's it (multiple speakers)--
那是1e14。好的。就是這樣(多位發言者)——
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
-- (multiple speakers) is the highest we've -- so we went there in the efficacy study that we just reported and we really saw nothing -- we saw nothing that would give us any concern. We will now go into the GMP tox study, which is the only thing remaining to complete the IND-enabling studies for that. But we will clearly start at a lower dose in the clinical study because that's the right thing to do, and that's where the agency will ask us to do. But thus far, we've -- it's a very clean capsid, which is great, great news.
- (多個演講者)是我們所達到的最高水平 - 所以我們在剛剛報告的功效研究中去了那裡,我們真的沒有看到任何東西 - 我們沒有看到任何會讓我們擔心的東西。我們現在將進入 GMP 毒物研究,這是完成 IND 支持研究的唯一剩下的事情。但我們顯然會在臨床研究中從較低劑量開始,因為這是正確的做法,也是該機構要求我們這樣做的地方。但到目前為止,我們已經——這是一個非常乾淨的衣殼,這是一個非常非常好的消息。
And then can we talk about hem A, Nathalie? I know you've been speaking to your friends at Pfizer regularly.
那我們可以談談下擺 A 嗎,Nathalie?我知道您經常與您在輝瑞的朋友交談。
Nathalie Dubois- Stringfellow - Senior Vice President, Chief Development Officer
Nathalie Dubois- Stringfellow - Senior Vice President, Chief Development Officer
Sure, sure.
一定一定。
Yeah, we're very excited that the mid-2024 pivotal readout in MA is coming up soon. And we -- the partnership allows for $220 million in potential milestone and 14% to 20% royalty on potential sale. But the specific amount of each milestone is not publicly disclosed, but this could start as early as the start of 2025 if the pivotal readout is positive, and if Pfizer would like to see regulatory approval to the program.
是的,我們非常高興 2024 年中期 MA 的關鍵數據即將發布。我們—該合作夥伴關係允許潛在的里程碑價值 2.2 億美元,以及潛在銷售的 14% 至 20% 的特許權使用費。但每個里程碑的具體金額並未公開披露,但如果關鍵數據是積極的,並且輝瑞希望看到監管部門批准該計劃,那麼最早可能會在 2025 年初開始。
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
And Gena, just to reflect that, and we're limited from what we can say. When you sign these deals, there's always an agreement about who talks about what. But the regulatory milestones, which are -- we get one for the US, one for Europe, and one for Japan are not for approval. They are for submission of the package. And then the commercial milestones, which are for first patient dosed rather than for reaching a certain sales threshold, so they're very, very favorable milestones that will fund the company in '25 and '26.
Gena,只是為了反映這一點,我們能說的有限。當您簽署這些協議時,總是會就誰談論什麼達成一致。但監管里程碑——我們為美國制定了一項,為歐洲制定了一項,為日本製定了一項,但尚未獲得批准。它們用於提交包裹。然後是商業里程碑,這是針對第一位患者給藥而不是達到某個銷售閾值,因此它們是非常非常有利的里程碑,將為公司在 25 年和 26 年提供資金。
Gena Wang - Analyst
Gena Wang - Analyst
Very helpful. Thank you (multiple speakers)--
很有幫助。謝謝(多位發言者)——
Nathalie Dubois- Stringfellow - Senior Vice President, Chief Development Officer
Nathalie Dubois- Stringfellow - Senior Vice President, Chief Development Officer
Yeah. I need to mention that the significant portion of the $220 million is near term and we could also choose to monetize them now.
是的。我需要指出的是,2.2 億美元中的很大一部分是近期的,我們現在也可以選擇將其貨幣化。
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
Yeah, so the -- so Gena the closer we get to when the data is revealed, and then when Pfizer submits this, we have the option of monetizing those royalties, but we could also choose to wait and use them to fund '25 and '26. And what will make that decision is the non-dilutive funding from business development that we do now will tell us whether we need to make that decision. We are -- I know that there's a concern from people that followed us for a long time about our near-term cash runway. We feel that we are fortunate to be able to do both capsid deals, Fabry deals, technology deals, and to have this large amount of cash next year that we can bring forward if necessary.
是的,所以- 所以吉納,當數據披露時,我們越接近,然後當輝瑞提交此數據時,我們可以選擇將這些特許權使用費貨幣化,但我們也可以選擇等待並使用它們來資助' 25和'26。做出這個決定的是我們現在所做的業務開發的非稀釋資金將告訴我們是否需要做出這個決定。我知道長期關注我們的人們對我們的近期現金跑道感到擔憂。我們覺得我們很幸運能夠進行衣殼交易、法布里交易、技術交易,並在明年擁有大量現金,如有必要,我們可以提前支付。
Gena Wang - Analyst
Gena Wang - Analyst
Thank you very much.
非常感謝。
Operator
Operator
Patrick Trucchio, H.C. Wainwright.
特魯基奧 (Patrick Trucchio),H.C.溫賴特。
Luis Santos - Analyst
Luis Santos - Analyst
Hi, everyone. This is Luis Santos for Patrick. Congratulations on the recent progress and your presence at -- impressive presence at ASGCT.
大家好。我是派崔克的路易斯桑托斯。恭喜您最近的進展以及您在 ASGCT 上的精彩亮相。
I am interested in knowing if you already guided to the tau program. And if it's going to be in Alzheimer's, do you know which patients you're going to be treating? And which other indications you are going to push forward here with the tau program?
我有興趣知道您是否已經指導過 tau 計劃。如果是針對阿茲海默症,您知道要治療哪些患者嗎?您還將透過 tau 計劃推動哪些其他跡象?
And then I have a follow-up question.
然後我有一個後續問題。
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
So thank you for your question. We haven't guided on that. There's been an enormous amount of interest in our presentation yesterday at ASGCT. It clearly was, I think, the most remarkable presentation of all of the many people that are moving forward in blood-brain barrier penetrant capsids. We feel that the tau asset, the cargo that's within it, double stone, makes us even more interesting.
謝謝你的提問。我們還沒有對此進行指導。我們昨天在 ASGCT 上的演講引起了極大的興趣。我認為,這顯然是所有在血腦屏障滲透衣殼領域取得進展的人中最引人注目的演講。我們覺得 tau 資產,其中的貨物,雙石,讓我們更有趣。
The role of tau in Alzheimer's, I think, is becoming increasingly clear. The Biogen data is very encouraging. The idea that it's the thing that is relevant to the clinical stages of tau rather -- of Alzheimer's rather than in the early and presymptomatic ones makes us a much more easy lift. However, we are very conscious that Alzheimer's studies are large programs and that we need to make sure that we either do ourselves well or partner with someone who has the real expertise in Alzheimer's.
我認為,tau 在阿茲海默症中的作用正變得越來越明顯。百健(Biogen)的數據非常令人鼓舞。認為它與 tau 蛋白的臨床階段有關,而不是阿茲海默症的臨床階段,而不是早期和症狀前的階段,這個想法讓我們更容易擺脫困境。然而,我們非常清楚,阿茲海默症研究是一項大型項目,我們需要確保我們要么做好自己,要么與在阿茲海默症方面擁有真正專業知識的人合作。
Luis Santos - Analyst
Luis Santos - Analyst
On the follow-up -- thank you. That was helpful.
關於後續行動——謝謝。這很有幫助。
On the follow-up on partnerships but for the MINT platform, you mentioned that there are business development opportunities there. Do you have any potential collaborators in mind? And what diseases would you be targeting?
關於後續合作夥伴關係,但對於MINT平台,您提到那裡有業務發展機會。您心中有潛在的合作者嗎?您將針對哪些疾病?
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
The MINT integrase is remarkable. The presentation at ASGCT was just 57 minutes ago and there was a lot of excitement in the room about it. We have already been -- we've been speaking to people for three months on this. And I want to put the business development discussions into perspective.
MINT 整合酶非常優異。ASGCT 的演講剛過 57 分鐘,房間裡充滿了興奮。我們已經就此事與人們進行了三個月的交談。我想正確看待業務發展討論。
We had the registry clarity for Fabry just in February. We had the capsid results just in March. We finally taken MINT to a place that we feel we can share it in May, so I understand the desire for signed deals, but the results are going to lead to them have only come out over the past two or three months, which is MINT is at the moment, you could imagine our business development group is incredibly busy in talking to lots of people about this.
就在二月份,我們就明確了法布里的登記情況。我們在三月就得到了衣殼結果。我們終於把 MINT 帶到了一個我們覺得可以在 5 月分享的地方,所以我理解簽署交易的願望,但結果將導致他們在過去的兩三個月裡才出來,這是 MINT目前,您可以想像我們的業務開發團隊非常忙於與許多人討論這個問題。
Now integrase is something that has the potential for really disruptive genomic engineering. It's the thing that David Liu, when he was asked, said was his dream of what the ideal genomic medicine was, which was an integrase. It could be engineered to go to your site of choice and put in pieces of DNA -- gene-size pieces of DNA that would replace all of the mutations downstream. And the team at Sangamo, because of their expertise in understanding the interaction between proteins and DNA from the zinc finger platform, have been able to do this remarkable engineering and the whole AlphaFold and understanding artificial intelligence of how you understand proteins has helped us.
現在,整合酶具有真正顛覆性基因組工程的潛力。當 David Liu 被問到時,他說他夢想的理想基因組醫學就是整合酶。它可以被設計成進入你選擇的位點並放入DNA片段——基因大小的DNA片段,它將取代下游的所有突變。Sangamo 的團隊憑藉在從鋅指平台了解蛋白質和 DNA 之間的相互作用方面的專業知識,能夠完成這項非凡的工程和整個 AlphaFold,並且了解人工智慧如何理解蛋白質對我們很有幫助。
We feel that this is something Sangamo can use, but it also has attracted great interest from the hardcore science pharma companies that you could imagine who all see this as the future of genomic engineering. And so we have responsibility to put it in as many people's hands as possible. I think you will hear more about this MINT Platform for some time to come.
我們認為這是 Sangamo 可以使用的東西,但它也引起了核心科學製藥公司的極大興趣,你可以想像,他們都將其視為基因組工程的未來。因此,我們有責任將其交到盡可能多的人手中。我想您在未來一段時間內將會聽到更多有關這個 MINT 平台的資訊。
Luis Santos - Analyst
Luis Santos - Analyst
Thank you.
謝謝。
Operator
Operator
Luca Issi, RBC.
盧卡·伊西,加拿大皇家銀行。
Lisa Walter - Analyst
Lisa Walter - Analyst
This is Lisa on for Luca. I just wanted to ask another question on the MINT technology. Just wondering if you can give us a sense of what target here would be good candidates, For instance, would it be possible to integrate something like a full-length CFTR gene, which is around roughly 190 kilobases? Or should we think about something larger like full-length dystrophin which is measuring closer to over 2,000 kilobases? Just any color here would be helpful. Thanks.
這是盧卡的麗莎。我只是想問另一個關於 MINT 技術的問題。只是想知道您是否能讓我們了解這裡的哪些目標是好的候選目標,例如,是否有可能整合像全長 CFTR 基因(大約 190 kilobase)之類的東西?或者我們應該考慮一些更大的東西,例如全長肌肉營養不良蛋白,其長度接近 2,000 千鹼基?這裡任何顏色都會有幫助。謝謝。
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
So you ask really important and interesting questions. And as always, with Sangamo, we saw about that size because, as you know, zinc fingers are an eighth of the size of CRISPR. And even the small mammoth CRISPR are four times as big as the zinc finger, and that's important because of delivery. It's the same with MINT because we try to keep the technology as small as possible. So as we could put both the integrase and a cargo in an AAV.
所以你問了非常重要和有趣的問題。像往常一樣,我們在 Sangamo 中看到了這個大小,因為如您所知,鋅指的大小是 CRISPR 的八分之一。即使是小型猛獁象的 CRISPR 也有鋅指的四倍大,這對於傳遞來說很重要。MINT 也是如此,因為我們試圖使技術盡可能小。這樣我們就可以將整合酶和貨物都放入 AAV 中。
Now when you get to the size of some of the genes that you are talking about, it comes down to what kind of delivery you're using. My dream for MINT is to be able to drop a cDNA copy of the gene or of the exons or mutated into intron 1 of the genome. It would then be run off of the promoter to have all the locus control elements.
現在,當您了解您正在談論的某些基因的大小時,這取決於您使用的傳遞方式。我對 MINT 的夢想是能夠刪除基因或外顯子的 cDNA 副本,或突變為基因組的內含子 1。然後它會脫離啟動子以獲得所有基因座控制元件。
And it would alleviate the requirement to edit all the individual mutations that usually are the cause of genomic disease. Now to put in a whole genome copies would be unnecessary, I would argue and then we need to look at what are the diseases where the cDNA is packageable into the delivery mechanisms that are available because the MINT should be sized agnostic and has so far shown great potential.
而且它將減輕編輯通常導致基因組疾病的所有個體突變的要求。我認為,現在放入整個基因組拷貝是不必要的,然後我們需要看看哪些疾病可以將 cDNA 打包到可用的傳遞機制中,因為 MINT 的大小應該是不可知的,並且迄今為止已經表明巨大的潛力。
Amy, is there anything else? Amy, I'm aware that you're on the line and more -- and as our Head of Research, perhaps you have other things to add to that.
艾米,還有什麼事嗎?艾米,我知道您在線上以及更多資訊 - 作為我們的研究主管,也許您還有其他需要補充的內容。
Amy Pooler - Head of Research
Amy Pooler - Head of Research
No, that's fantastic explanation, Sandy.
不,這是很棒的解釋,桑迪。
I would just add that there are specific genes as well that are involved in some neurological disorders, and I'm thinking of things like Rett syndrome or MeCP2 needs to be fine-tuned to a very specific level, something like an integrates where you could integrate in perhaps the healthy copy of exon 1 or even a bigger portion of the gene so that it's under control of the endogenous promoter could be really transformative for a disease like that, so I think it's incredibly exciting to see what are the opportunities around this new MINT platform.
我想補充一點,某些神經系統疾病也涉及特定的基因,我認為像 Rett 綜合徵或 MeCP2 這樣的基因需要微調到一個非常特定的水平,例如整合,你可以整合到外顯子1 的健康副本甚至基因的更大部分中,使其處於內源啟動子的控制之下,這可能會對此類疾病產生真正的變革,所以我認為看到這方面的機會是非常令人興奮的新的MINT平台。
Operator
Operator
Ritu Baral, TD Cowen.
裡圖·巴拉爾,TD·考恩。
Ritu Baral - Analyst
Ritu Baral - Analyst
Just a quick question on the Fabry program actually dosed 33 patients. We saw the data at WORLD plus you have the Phase 3 path going forward agreed upon with FDA. What is the next data update that we're going to get from that? And also, I guess is there a data point or follow-up point in a subset of patients that's gating to potential business development around or out-licensing of this program?
一個關於 Fabry 計劃的簡單問題實際上對 33 名患者進行了給藥。我們在 WORLD 上看到了數據,而且你們已經與 FDA 商定了第三階段的進展路徑。我們將從中獲得的下一個數據更新是什麼?而且,我猜想在一部分患者中是否存在一個數據點或後續點,可以促進該計畫的潛在業務發展或向外許可?
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
Thank you, Ritu. I'm going to pass this on to Nathalie, but just remind you again, the whole business development conversation around Fabry changed with Peter's new way of thinking about this at the agency and the conversations that we had in February. And so it's like a new conversation we're having with potential partners.
謝謝你,瑞圖。我將把這個轉達給 Nathalie,但再次提醒您,圍繞 Fabry 的整個業務發展對話隨著 Peter 在該機構對此問題的新思考方式以及我們二月份的對話而改變。這就像我們與潛在合作夥伴進行的新對話。
But Nathalie, can you address some of the technical pieces, please?
但是 Nathalie,你能談談一些技術問題嗎?
Nathalie Dubois- Stringfellow - Senior Vice President, Chief Development Officer
Nathalie Dubois- Stringfellow - Senior Vice President, Chief Development Officer
Yeah, sure. So we've completed the dosing of all the patients in the Phase 1/2, so a total of 33 patients. We continue to analyze the data, but the body of data that we have thus far is not gate limiting for any partnership and any of the Phase 2b preparation or registrational trial preparation.
好,當然。所以我們已經完成了1/2期所有患者的給藥,總共33名患者。我們繼續分析數據,但迄今為止我們擁有的數據主體並不限制任何合作夥伴關係以及任何 2b 期準備或註冊試驗準備。
So the continue -- we continue to analyze data, but we have agreed with the FDA that we have enough data to move on to a Phase 2b. And that's what we're discussing with partners -- potential partners.
因此,我們繼續分析數據,但我們已經與 FDA 達成一致,認為我們有足夠的數據可以進入 2b 階段。這就是我們正在與合作夥伴——潛在的合作夥伴——討論的內容。
Ritu Baral - Analyst
Ritu Baral - Analyst
Sandy, when you say that the discussion has changed, are you talking about the structure of the deal that is in Sangamo's best interests? Like is it -- do you think it could be like a COCO going forward? Or is it just really valuation terms that are different now?
桑迪,當你說討論發生了變化時,你是在談論符合桑加莫最佳利益的交易結構嗎?就像它一樣——你認為它未來會像 COCO 一樣嗎?還是只是估值條款現在不一樣了?
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
I think it's much simpler than that, Ritu. There was a concern that there wasn't clarity about what the regulators were wanting. And there was this worry that we've all talked about that it was going to be a head-to-head against ERT. And the agency now saying -- no, it's a simple single study and that we can -- they've named the number of patients and they've said we can come back halfway through the study and show them the data we've got. We feel it's compelling. I think that is completely changed how the various companies are going to -- and I think great credit to Peter. He is walking the talk in this, and we have benefited greatly from it as being almost like the poster child for what it is approval of a rare disease gene therapy is.
我認為這比那簡單得多,Ritu。有人擔心監管機構的要求並不明確。我們都曾擔心這將是一場與 ERT 的正面交鋒。該機構現在說——不,這是一項簡單的單一研究,我們可以——他們已經列出了患者的數量,他們說我們可以在研究進行到一半時回來並向他們展示我們得到的數據。我們覺得它很有說服力。我認為這完全改變了各個公司的發展方向——我認為這要歸功於彼得。他在這方面言行一致,我們也從中受益匪淺,因為我們幾乎就像罕見疾病基因療法獲得批准的典型代表。
Operator
Operator
One moment for our next question (multiple speakers)--
請稍等一下我們的下一個問題(多位發言者)——
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
And if I may reiterate -- we've started the -- we've initiated the conversations with the Europeans and uniquely the FDA is volunteering to send one of their staff with us to that conversation because they feel that it's the way forward for these therapies is for a common approach to them. And I think it reflects the support that we have from the FDA in these discussions.
如果我可以重申一下——我們已經開始了——我們已經開始與歐洲人進行對話,而且 FDA 自願派出一名工作人員與我們一起參加對話,因為他們認為這是這些計畫的前進方向。這些問題的通用方法。我認為這反映了 FDA 在這些討論中給予我們的支持。
Operator
Operator
Nicole Germino, Truist.
妮可·傑米諾,真理主義者。
Unidentified Participant
Unidentified Participant
This is Bill on for Nicole.
這是比爾為妮可代言的。
We have a question. Given the uptake for Roctavian, what is the read-through to potential uptake for Fabry disease gene therapy?
我們有一個問題。鑑於 Roctavian 的採用率,法布瑞氏症基因治療的潛在採用率是什麼?
Alexander Macrae - President, Chief Executive Officer, Director
Alexander Macrae - President, Chief Executive Officer, Director
So the Roctavian launch, I know, has been a subject of great debate. We can't comment on how BioMarin launched it. All I know is the reason that we chose Pfizer to be our partner is I have great confidence in when Pfizer chooses to launch something. They do it well and effectively, and that follows through to our choice of partners for Fabry disease. There are several companies and there are some that are more obvious as being great commercialization engines and great launch companies.
因此,據我所知,羅克塔維安號的發射一直是一個備受爭議的話題。我們無法評論 BioMarin 如何推出它。我所知道的是,我們選擇輝瑞作為合作夥伴的原因是我對輝瑞選擇推出某些產品充滿信心。他們做得很好、很有效,這也影響了我們對法布瑞氏症夥伴的選擇。有幾家公司,其中一些更明顯是偉大的商業化引擎和偉大的發射公司。
What I can tell you is in our conversations with these companies, we have had notes from the patient support groups. If you have been very keen to make sure that the pharma partners understand how supportive they are of the results that they have seen in our Fabry program. Because you and I will talk about the biochemistry and the biopsies.
我可以告訴你的是,在我們與這些公司的對話中,我們收到了病患支持小組的記錄。如果您非常渴望確保製藥合作夥伴了解他們對我們法布里計劃中看到的結果有多麼支持。因為你和我將討論生物化學和活組織檢查。
What the patients have said is how much better they feel on it, how delighted that they are to be off of ERT. So now we have 14 patients off of ERT and the 4 that are remaining on ERT in the study have already booked when they're going to come off of the therapy, so this has enormous support from the patient. And I think that is what is going to make it a very successful launch for the partner that can take it forward and make a difference.
患者所說的是他們對 ERT 的感覺好多了,他們對停止 ERT 感到多麼高興。現在我們有 14 名患者停止了 ERT,而研究中仍在接受 ERT 的 4 名患者已經預訂了停止治療的時間,因此這得到了患者的大力支持。我認為這對合作夥伴來說將是一次非常成功的發布,能夠推動它向前發展並產生影響。
Nathalie Dubois- Stringfellow - Senior Vice President, Chief Development Officer
Nathalie Dubois- Stringfellow - Senior Vice President, Chief Development Officer
And if I may add, we also have RMAT and PRIME, which are designations that really analogize an unmet medical need for Fabry. So the Fabry patient, even though can have access to ERT, are not satisfied with the current medicine and standard of care.
如果我可以補充一下,我們還有 RMAT 和 PRIME,這些名稱真正模擬了 Fabry 未滿足的醫療需求。因此,法布里患者儘管可以接受 ERT,但對目前的藥物和護理標準並不滿意。
Unidentified Participant
Unidentified Participant
Thank you so much.
太感謝了。
Operator
Operator
Thank you very much. At this time, I'm showing no further questions. And I would now like to turn the call back to Louise Wilkie for closing remarks.
非常感謝。目前,我沒有再提出任何問題。現在我想將電話轉迴路易絲·威爾基 (Louise Wilkie),讓其致閉幕詞。
Louise Wilkie - Investor Relations
Louise Wilkie - Investor Relations
Thank you very much, and thanks to everyone for joining us on the call today. We look forward to speaking to you soon.
非常感謝,也感謝大家今天加入我們的電話會議。我們期待盡快與您交談。
Operator
Operator
Thank you for your participation in today's conference. This does conclude the program, and you may now disconnect. Thank you.
感謝您參加今天的會議。這確實結束了程序,您現在可以斷開連接。謝謝。