Sangamo Therapeutics (NASDAQ:SGMO) stock is rising higher on Tuesday after the clinical-stage genomic medicine company announced a new license agreement. Sangamo Therapeutics has signed a license agreement Genentech for the development of intravenously administered genomic medicines to treat "certain neurodegenerative diseases." That includes the company's proprietary zinc finger repressors for Alzheimer's disease. This agreement will see Sangamo handle technology transfer and some preclinical activities. G ...

文章核心观点 - 公司拥有3个潜在价值高达10亿美元的资产 [2][3] - 公司正处于生死存亡的关键时刻 需要尽快推进这3个资产的进展 [8][10] - 公司有望在未来12-24个月内实现10亿美元的估值 [11] 资产1: Giroctocogene fitelparvovec (giro-vec) - 用于治疗血友病A的基因疗法 [4][5] - 已完成I/II期临床试验 显示疗效和安全性良好 [5] - 正在进行III期临床试验 预计2024年中期公布结果 [5] - 如果III期试验成功 公司将获得最高2.2亿美元的里程碑付款 [5] 资产2: Isaralgagene civaparvovec (isa-vec) - 用于治疗法布里病的基因疗法 [5][6] - 已完成I/II期临床试验 显示部分患者可停用酶替代疗法 [6] - 只需进行一个25人的关键性试验即可获批 [6] - 预计可为公司带来数亿美元的授权金和里程碑付款 [6] 资产3: STAC-BBB - 一种可穿过血脑屏障递送基因的独特载体 [6][7] - 在非人灵长类动物实验中表现出显著优于其他载体 [6] - 有望用于治疗阿尔茨海默病、帕金森病等中枢神经系统疾病 [6][7] - 公司已有10家公司对此表现出浓厚兴趣 有望在今年内达成合作 [7]

财务数据和关键指标变化 - 公司在2024年第一季度结束时拥有约5,400万美元的现金和现金等价物,包括之前的定向增发所得资金 [31][32] - 公司认为这些资源加上预期的成本节约,将足以为计划的运营提供资金,直到2024年第三季度 [31][32] 各条业务线数据和关键指标变化 - 公司的Fabry病基因疗法项目已完成33名患者的给药,其中14名患者已停用酶替代疗法(ERT) [27][28] - 公司与辉瑞合作开发的血友病A基因疗法项目预计将于2024年中期进行关键性临床试验读数,如果结果支持,辉瑞计划于2025年初提交生物制品许可申请和上市申请 [30] 各个市场数据和关键指标变化 - 公司的Fabry病基因疗法项目已获得欧洲药品管理局的PRIME资格和英国药品监管机构的ILAP指定,表明该疗法具有重大医疗需求 [29] 公司战略和发展方向及行业竞争 - 公司将重点发展神经系统疾病领域,并正在就Fabry病、新型AAV载体和下一代基因编辑技术与多家潜在合作伙伴进行积极讨论 [14][15][25] - 公司在ASGCT年会上展示了神经系统疾病领域的多项重要临床前数据,包括新型AAV载体STAC BBB的优异脑穿透性和转染效率,以及基于锌指蛋白的表观遗传调控技术在多种神经系统疾病中的应用 [12][16][17][21][22][23][24] - 公司还展示了一种新型的整合酶技术MINT,可实现大片段DNA的精准整合,有望用于治疗存在不同突变的同一基因疾病患者 [24][25] 管理层对经营环境和未来前景的评论 - 公司CEO表示,公司正全力解决资金需求,并对与潜在合作伙伴的讨论持乐观态度,希望能够在未来几周内宣布相关交易消息 [14][15][33] - 公司认为,其科技平台有望为治疗多种神经系统疾病带来变革性的机会,正致力于筹集所需资金以实现这一愿景 [19][33] 问答环节重要的提问和回答 问题1 **James Stamos 提问** 询问公司对STAC BBB的临床前和GLP毒理学研究进展,以及未来哪些适应症会选择自主开发或寻求合作 [36][37][38] **Sandy Macrae 回答** 公司正在推进STAC BBB的GMP毒理学研究,初步结果显示该载体安全性良好。公司正在就STAC BBB与多家公司进行合作讨论,将根据资金需求等因素决定自主开发或寻求合作的适应症,如tau蛋白相关疾病等 [37][38][39] 问题2 **Gena Wang 提问** 询问STAC BBB在非人灵长类动物试验中使用的最高剂量,以及公司与辉瑞合作的血友病A项目的里程碑付款情况 [48][49] **Sandy Macrae 和 Nathalie Dubois-Stringfellow 回答** 公司在STAC BBB的剂量范围研究中使用了最高1x10^14 vg/kg的剂量,未观察到任何剂量限制性毒性。 关于血友病A项目,公司有权获得最高2.2亿美元的里程碑付款,其中包括监管里程碑和商业里程碑,具体金额未披露 [50][51][57][59][60] 问题3 **Luis Santos 提问** 询问公司是否已确定tau蛋白项目的适应症,以及MINT整合酶技术的潜在合作伙伴和适应症 [67][68] **Sandy Macrae 回答** 公司尚未确定tau蛋白项目的具体适应症,但对其在阿尔茨海默病等疾病中的潜力持乐观态度。 MINT整合酶技术已引起多家制药公司的浓厚兴趣,公司正在与潜在合作伙伴就各种适应症进行积极讨论 [68][69][72][73]

Sangamo Therapeutics (SGMO) came out with a quarterly loss of $0.27 per share versus the Zacks Consensus Estimate of a loss of $0.22. This compares to earnings of $0.12 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -22.73%. A quarter ago, it was expected that this drug developer would post a loss of $0.25 per share when it actually produced a loss of $0.34, delivering a surprise of -36%.Over the last four quarters, the compa ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ________________________________________________ FORM 10-Q ________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 000-30171 ______ ...

Exhibit 99.1 SANGAMO THERAPEUTICS REPORTS RECENT BUSINESS HIGHLIGHTS AND FIRST QUARTER 2024 FINANCIAL RESULTS • Showcased 20 presentations at the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting demonstrating progression of neurology-focused preclinical pipeline, including additional data from novel delivery capsid, STAC-BBB, that demonstrated industry-leading blood-brain barrier (BBB) penetration in non-human primates (NHPs) following intravenous administration. • Announced discovery of ...

RICHMOND, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced preclinical data from its proprietary adeno-associated virus (AAV) capsid variant, STAC-BBB, that demonstrated robust penetration of the blood-brain barrier (BBB) and strong transgene expression throughout the central nervous system (CNS) of NHPs when administered intravenously at clinically-relevant doses, outperforming results obtained by Sangamo for other known neurotropic capsid vari ...

RICHMOND, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, announced today that the company has scheduled the release of its fourth quarter and full year 2023 financial results before the market opens on Wednesday, March 13, 2024. The Company will hold a conference call at 4:30 p.m. Eastern on Wednesday, March 13, which will be open to the public. During the conference call, the company will review its financial results and provide business updates. Participa ...

Exhibit 99.1 SANGAMO THERAPEUTICS REPORTS RECENT BUSINESS HIGHLIGHTS AND FOURTH QUARTER AND FULL YEAR 2023 FINANCIAL RESULTS • Data from novel proprietary neurotropic adeno-associated virus (AAV) delivery capsid, STAC-BBB, demonstrated industry- leading blood-brain barrier (BBB) penetration in non-human primates (NHPs) following intravenous administration, with capsid-enabled delivery of zinc finger payloads targeting prion disease and tauopathies resulting in potent and widespread repression of target gene ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _______________________________________________________________________________________________ Form 10-K _______________________________________________________________________________________________ ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the trans ...