Passage Bio Receives FDA Clearance Of IND Application For PBML04 For Treatment Of Metachromatic Leukodystrophy
Passage Bio Receives FDA Clearance Of IND Application For PBML04 For Treatment Of Metachromatic Leukodystrophy
PBML04 represents the company's fourth program to receive IND clearance
PBML04是該公司第四個獲得IND批准的項目
PHILADELPHIA, June 08, 2022 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for PBML04, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of Metachromatic Leukodystrophy (MLD). MLD is a rare, fatal, pediatric, lysosomal storage disease that currently has limited available treatment options.
費城，2022年6月8日(環球網)--Passage Bio，Inc.(納斯達克代碼：PASG)，一家專注於為中樞神經系統疾病開發變革性療法的臨牀階段基因藥物公司，今天宣佈，美國食品和藥物管理局(FDA)已經批准了其PBML04的研究新藥申請，PBML04是一種腺相關病毒遞送基因療法，正在研究用於治療異染性腦白質營養不良(MLD)。MLD是一種罕見的、致命的兒科溶酶體儲存性疾病，目前可供選擇的治療方法有限。
"We are thrilled to have received IND clearance for our program in metachromatic leukodystrophy, marking our fourth IND clearance as a company and our third pediatric lysosomal storage disorder program to reach clinical development," said Edgar (Chip) Cale, interim chief executive officer of Passage Bio. "Clearance of this IND is a testament to the quality preclinical data supporting PBML04 through our partnership with the University of Pennsylvania's Gene Therapy Program and the strong CMC and analytics capabilities we have developed internally. MLD, GM1 gangliosidosis and Krabbe disease are similar diseases each caused by a single gene mutation leading to deficiency of a critical enzyme. Our approach and clinical development plans across these three programs also share similarities, including use of our next-generation, proprietary AAVhu68 capsid and ICM delivery. We are excited by the promise of PBML04 to offer a potentially transformative treatment for this devastating disease."
Passage Bio臨時首席執行官埃德加(奇普)·凱爾説：“我們很高興我們的異色性腦白質營養不良項目獲得了IND許可，這標誌着我們作為一家公司獲得了第四次IND許可，也是我們第三次獲得兒科溶酶體儲存障礙項目的臨牀開發。通過與賓夕法尼亞大學基因治療計劃的合作，以及我們內部開發的強大的CMC和分析能力，該IND的批准證明瞭支持PBML04的高質量臨牀前數據。MLD、GM1神經節苷脂沉着症和Krabbe病都是類似的疾病，每種疾病都是由單個基因突變導致關鍵酶缺乏引起的。我們的方法和這三個計劃的臨牀開發計劃也有相似之處，包括使用我們的下一代專利AAVhu68衣殼和ICM交付。我們對PBML04有望為這種毀滅性疾病提供潛在的變革性治療感到興奮。
MLD is a fatal inherited disease that is caused by mutations in the arylsulfatase-A (ARSA) gene which reduces enzyme activity, leading to progressive build-up of toxic sulfatides in the central and peripheral nervous system. We are targeting infantile-onset MLD, which is characterized by progressive muscle weakness, rigidity, gait disorder, developmental delays and is typically fatal by five years of age. The estimated worldwide incidence of MLD is approximately 1 in 100,000 live births.
MLD是一種致命的遺傳性疾病，由基因突變引起芳基硫酸酯酶-A (ARSA)基因，它降低酶活性，導致有毒硫脂在中樞和外周神經系統中逐漸積聚。我們的目標是嬰兒期的MLD，它的特徵是進行性肌肉無力、僵硬、步態障礙、發育遲緩，通常在五歲時致命。據估計，全世界MLD的發病率約為每100,000名活產兒中就有1名。
The Phase 1 clinical trial will utilize intra-cisterna magna (ICM) administration to deliver an AAVhu68 capsid to express ARSA and potentially address both central nervous system and peripheral manifestations of this devastating disease.
第一階段臨牀試驗將利用大腦池內(ICM)給藥來傳遞AAVhu68衣殼，以表達ARSA，並可能解決這種毀滅性疾病的中樞神經系統和外周表現。
譯文內容由第三人軟體翻譯。