Capsida Biotherapeutics to Present New Preclinical Data for Potential Best-in-Class Friedreich's Ataxia Next-Generation Gene Therapy
Capsida Biotherapeutics to Present New Preclinical Data for Potential Best-in-Class Friedreich's Ataxia Next-Generation Gene Therapy
Friedreich's ataxia is a rare hereditary disease with limited treatment options. It is caused by a mutation that reduces frataxin (FXN) protein expression, leading to progressive neurodegeneration, cardiac dysfunction, and sensory deterioration. CAP-004 is designed to target and address the key pathophysiological features of the disease by delivering therapeutically meaningful FXN expression in the CNS, cardiac tissues, and sensory regions.NHP data demonstrate potential for CAP-004 to treat CNS, cardiac, and sensory manifestations of Friedreich's ataxia
NHP數據展示CAP-004有潛力治療弗裏德雷希共濟失調的中樞神經系統、心臟和感覺表現
THOUSAND OAKS, Calif., Nov. 12, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced new preclinical data for CAP-004, a potential best-in-class systemically administered next-generation gene therapy for the treatment of Friedreich's ataxia. CAP-004 is Capsida's third wholly owned program and utilizes one of the company's proprietary engineered capsids to target central nervous system (CNS), cardiac, and sensory manifestations of the disease while limiting effects in off-target tissues, especially the liver. CAP-004 is now entering Investigational New Drug (IND) application enabling studies.
2024年11月12日，加州千橡市/PRNewswire/-- Capsida生物治療（"Capsida"）今天宣佈CAP-004的新的臨床前數據，CAP-004是一種潛力領先的全身給藥的下一代基因療法，用於治療弗裏德雷希共濟失調。 CAP-004是Capsida的第三個完全擁有的項目，利用公司專有的改良殼體之一來靶向該疾病的中樞神經系統（CNS）、心臟和感覺表現，同時限制在非靶組織，尤其是肝臟的影響。 CAP-004現已進入新藥申請（IND）可行性研究階段。
Data highlighting the therapeutic potential of CAP-004 will be presented in a late-breaking poster presentation at the International Congress for Ataxia Research (ICAR), being held November 12-15, 2024 in London, UK.
CAP-004療效潛力的數據將在2024年11月12日至15日於英國倫敦舉行的國際共濟失調研究大會（ICAR）上通過一份晚報告海報展示。
Friedreich's ataxia is a rare hereditary disease with limited treatment options. It is caused by a mutation that reduces frataxin (FXN) protein expression, leading to progressive neurodegeneration, cardiac dysfunction, and sensory deterioration. CAP-004 is designed to target and address the key pathophysiological features of the disease by delivering therapeutically meaningful FXN expression in the CNS, cardiac tissues, and sensory regions.
弗裏德雷希共濟失調是一種罕見的遺傳性疾病，治療選擇有限。 它是由導致減少FXN蛋白表達的突變引起的，導致進行性神經退行性、心臟功能障礙和感覺惡化。 CAP-004旨在通過在中樞神經系統、心臟組織和感覺區域提供治療意義的FXN表達來靶向和解決該疾病的關鍵病理生理特徵。
In the study conducted in non-human primates (NHPs), results showed that CAP-004 demonstrated:
在對非人靈長類動物（NHPs）進行的研究中，結果顯示CAP-004表現出：
- Brain-wide biodistribution, achieving more than 80% transduction in key cell types, such as Purkinje cells in the cerebellum, neurons in the dentate nucleus, and motor neurons in the cortex and spinal cord.
- 100 times higher RNA expression compared with AAV9 in the cerebellum, motor cortex, and spinal cord.
- Significant cardiac tissue transduction, covering nearly 30% of the left ventricle tissue area.
- Bulk hFXN protein levels in treated NHPs were up to 8.2 times higher than endogenous levels in the motor cortex and 1.7 times higher in the left ventricle, highlighting the therapeutic potential in both CNS and cardiac regions.
- Potential in treating sensory vision loss, with significant RNA expression detected in the retina.
- Significant detargeting of the liver and other non-target tissues contributed to the favorable safety profile characterized by no adverse immunogenicity, clinical pathology, and histopathology findings.
- 腦部廣泛的分佈，在小腦的普金細胞、腦的小腦核神經元以及皮層和脊髓的運動神經元等關鍵細胞類型中實現了80%以上的轉導。
- 與AAV9相比，在小腦、運動皮質和脊髓中的RNA表達高出100倍。
- 顯著的心臟組織轉導，覆蓋了左心室組織區域的近30%。
- 治療過的非人靈長類動物中，hFXN蛋白水平在運動皮層高出內源水平多達8.2倍，在左心室中高出1.7倍，突顯了治療潛力，涵蓋CNS和心臟區域。
- 在治療感覺視力喪失方面具有潛力，視網膜中檢測到顯著的RNA表達。
- 對肝臟和其他非靶組織的重點排除有助於無任何不良免疫原性、臨床病理學和組織病理學結果的有利安全性特點。
"These preclinical data demonstrate CAP-004's potential to be a best-in-class gene therapy for Friedreich's ataxia, a devastating disease with few treatment options," said Peter Anastasiou, CEO of Capsida Biotherapeutics. "With CAP-004 delivered as a single IV injection, Capsida has the potential to address neurodegenerative, cardiovascular, and sensory components of this disease."
"這些臨床前數據展示了CAP-004在治療弗氏共濟失調症中可能成爲最佳基因療法，這是一種幾乎沒有治療選擇的破壞性疾病，"Capsida生物療法公司的首席執行官Peter Anastasiou表示。"通過CAP-004作爲單一靜脈注射的途徑，Capsida有望解決這種疾病的神經退行性、心血管和感覺元素。"
Details about the CAP-004 ICAR poster presentation:
關於CAP-004 ICAR海報展示的詳細信息：
Systemic AAV Gene Therapy with Next Generation Engineered Capsids for Treatment of CNS and Cardiac Symptoms in Friedreich's Ataxia
Date: Wednesday, November 13, 2024, 6:00 PM GMT
Session: Emerging and existing therapeutics – preclinical research
Poster Number: 477
Location: Leonardo Royal Hotel London Tower Bridge, Minories Suite
Presenter: Celeste Stephany, Ph.D., Director of CNS and Ophthalmology Preclinical Research, Capsida
使用下一代工程化膠囊病毒進行全身AAV基因療法，治療弗氏共濟失調症中的中樞神經系統和心臟症狀
日期：2024年11月13日，週三，晚上6點，GMT
會議：新興和現有療法-臨床前研究
海報編號: 477
地點: 倫敦塔橋倫敦Leonardo Royal Hotel, Minories Suite
演講者: Celeste Stephany, 博士, 腦中樞系統和眼科醫療臨床前研究董事, Capsida
About Capsida Biotherapeutics
Capsida Biotherapeutics is a fully integrated gene therapy company with a central nervous system (CNS) pipeline consisting of disease modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida's intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including a potential first-in-class treatment for STXBP1 developmental and epileptic encephalopathy and a best-in-class treatment Parkinson's disease associated with GBA mutations, both of which are in IND-enabling studies and on track to enter the clinic in the first half of 2025. Capsida's third program is a potential best-in-class therapy for Friedreich's ataxia and is in IND-enabling studies. In addition to its wholly owned programs, the Company has validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at .
關於Capsida生物治療
Capsida生物治療是一家完全一體化的基因療法公司，具有中樞神經系統(CNS)管道，包括罕見病和更常見疾病的病症修正和潛在治癒治療，適用於各個年齡段。Capsida的靜脈內(IV)給藥基因療法利用專利的工程化衣殼，實現對期望組織和細胞的高轉導水平，同時限制朝性不靶器官(如肝臟)的傳染。Capsida擁有三個全資計劃，包括可能是STXBP1發育性癲癇性腦病的一類潛在首創療法，以及用於GBA基因突變相關帕金森病的一類最佳療法，這兩類療法目前正在進行IND授權研究，並計劃在2025年上半年進入臨床應用。Capsida的第三項計劃是適用於弗裏德雷希共濟失調的一個潛在最佳療法，正在進行IND授權研究。除了全資計劃外，該公司還與艾伯維公司，Lilly，crispr therapeutics等合作伙伴在CNS進行驗證性合作，而艾伯維公司的合作伙伴關係擴展到了眼科疾病。Capsida成立於2019年，由Versant Ventures和西湖村生物合夥人的領投者創立，源自於加州理工學院神經科學教授Viviana Gradinaru, 博士實驗室的開創性研究。訪問我們的網站: 。
SOURCE Capsida Biotherapeutics
資訊 來源 Capsida生物治療