Sangamo Pioneering and Delivering the Future of Genomic Medicines April 2023 Forward-Looking Statements This presentation contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to our focus on key strategic priorities, the potential to develop, obtain regulatory approvals for and commercialize durable, safe and effective therapies to treat certain diseases and the timing, availability and costs of such therapi ...

Sanaam Pioneering and Delivering the Future of Genomic Medicines February 2023 Forward-Looking Statements This presentation contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize durable, safe and effective therapies to treat certain diseases and the timing, availability and costs of such therapies, the potential to use ZFP, ZFP-TR, ...

Sangamo Therapeutics, Inc. (NASDAQ:SGMO) Q4 2022 Results Conference Call February 22, 2023 4:30 PM ET Company Participants Louise Wilkie - VP of IR and Corporate Communications Sandy Macrae - Chief Executive Officer Mark McClung - Chief Operating Officer Prathyusha Duraibabu - Chief Financial Officer Jason Fontenot - Chief Scientific Officer Nathalie Dubois-Stringfellow - Chief Development Officer Bettina Cockroft - Chief Medical Officer Andy Ramelmeier - Head of Technical Operations Conference Call Partici ...

公司概况 - 公司是一家临床阶段的生物技术公司，尚无已批准的产品或产品收入[11] - 公司已自成立以来承担了巨额运营亏损，并预计在可预见的未来将继续亏损[14] - 公司需要大量额外资金来执行其运营计划，并继续作为一个持续经营的实体运作[15] - 公司依赖于与更大的生物制药公司的合作来产生收入，并开发、获得监管批准和商业化其许多产品候选药物[16] 产品研发与治疗效果 - 2022年11月15日的补充截止日期显示，剂量递增阶段的九名患者的α-Gal A活性持续升高，范围从正常平均值的3.7倍到67.6倍，最后一次测量时。[36] - 在剂量递增阶段，首次接受5x1013 vg/kg剂量的扩展阶段的前三名患者在给药后α-Gal A活性迅速增加，持续时间长达14周。[37] - 在剂量扩增和递增阶段接受治疗的13名患者的结果显示，基线lyso-Gb3水平高于80 ng/mL的患者经历了40-65%的水平降低。[38] - BIVV003治疗SCD的初步临床数据已在2022年12月的ASH会议上公布[59] - BIVV003治疗SCD的患者在治疗后的临床改善情况已公布[60][61][62] - BIVV003在2022年9月30日截止日期前总体上耐受性良好，大部分不良事件与SCD相关[62] - BIVV003治疗后的严重血管阻塞危机（VOC）发生率已公布[68][69] 技术平台与合作关系 - 公司的技术策略是利用ZF技术平台创造具有临床潜力的产品候选药物[71] - ZFP是人类中天然存在的特异性DNA结合蛋白，可用于基因组编辑和表观遗传调控[72][73] - 公司的ZF平台提供了多种基因组工程工具，包括ZF核酸酶和ZF转录调节因子[75][76] - 公司正在评估多种用于中枢神经系统疾病的CNS靶向治疗方案[79] - 公司开发了专有的AAV外壳发现平台SIFTER™，旨在改进CNS转导[80] - 公司在基因组医学领域开发了一种新方法，即基因组工程-碱基编辑[81] - 公司认为ZF平台提供了开发新型人类治疗药物的机会[82] - 公司正在开发递送策略，包括使用mRNA、AAV、腺病毒等，以扩展ZF治疗试剂的临床范围[83] - 公司的CAR-Tregs项目专注于治疗自身免疫和炎症性疾病[84] - CAR-Tregs具有潜力诱导长期免疫耐受[85] - CAR-Tregs预计通过静脉注射后会迁移到炎症组织[86] - CAR-Tregs预计会与特定抗原结合，从而发挥抗炎和免疫抑制作用[87] 合作与发展 - Sangamo正在评估BIVV003，这是用于SCD的ZF核酸酶基因编辑细胞疗法产品候选药物[96] - Sangamo与Sanofi合作进行有序过渡，将BIVV003的权利和义务转回给Sangamo[96] - Sangamo继续推进CAR-Treg细胞疗法项目，用于治疗炎症性肠病[98] - Sangamo继续推进CAR-Treg细胞疗法项目，用于治疗多发性硬化症[99] - Sangamo正在推进在朊病领域的全资前期基因工程项目[100] - Sangamo计划在2023年底分享新的CNS表观遗传调控项目的数据[101] - Sangamo与Pfizer继续开发giroctocogene fitelparvovec，或称为SB-525，用于治疗血友病A[102] - Sangamo与Biogen继续开发基因调控疗法，用于治疗神经系统疾病[105] - Sangamo与Novartis合作开发基因调控疗法，用于治疗神经发育障碍[106] - Sangamo与Pfizer合作开发基因工程产品候选药物，用于治疗ALS和前颞叶变性[107] 知识产权与竞争 - 公司拥有约170个专利家族，涵盖了ZF蛋白、ZF核酸酶、ZF-转录抑制因子等技术[127] - 公司的专利地位存在不确定性，可能会受到专利局、法院和监管机构的影响[128] - 公司在全球范围内保护知识产权的成本高昂，可能会选择不在某些国家寻求专利保护[130] - 公司面临来自其他生物制药公司、学术研究机构和政府机构的激烈竞争[131] - 公司的产品候选可能与已有长期安全有效使用历史的产品竞争[132] 法规与监管 - 公司在美国受FDA监管，包括基因治疗和人类细胞疗法产品[142] - 公司的产品候选品必须在FDA批准前进行临床试验[143] - 临床试验通常分为三个阶段：第一阶段、第二阶段和第三阶段[149] - 临床试验的监控和审计要求非常严格，需要向FDA提交年度进展报告[153] - 在欧盟，药品只能在获得相关MA后才能上市[161] - 在欧盟，集中授权程序要求欧洲委员会授予单一MA，适用于特定产品[161] - 在欧盟，分散MA程序要求在每个欧盟成员国的主管当局进行单独申请和批准[162] - 在欧盟，相互认可程序允许已在一个欧盟成员国授权的药品获得其他欧盟成员国主管当局的认可[162] - 在欧盟，药品获得

Pioneering the Future of Genomic Medicines November 2022 Forward-Looking Statements This presentation contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize durable, safe and effective therapies to treat certain diseases and the timing, availability and costs of such therapies, the potential to use ZFP, ZFP-TF, CAR-Treg and other te ...

Sangamo Therapeutics, Inc. (NASDAQ:SGMO) Q3 2022 Earnings Conference Call November 3, 2022 4:30 PM ET Company Participants Louise Wilkie - Investor Relations Sandy Macrae - Chief Executive Officer Nathalie Dubois-Stringfellow - Chief Development Officer Jason Fontenot - Chief Scientific Officer Prathyusha Duraibabu - Chief Financial Officer Bettina Cockroft - Chief Medical Officer Mark McClung - Chief Operating Officer Conference Call Participants Gena Wang - Barclays Yanan Zhu - Wells Fargo Ritu Baral - Co ...

Sangamo Therapeutics, Inc. (NASDAQ:SGMO) Q2 2022 Earnings Conference Call August 4, 2022 4:30 PM ET Company Participants | --- | |------------------------------------------------| | | | | | Louise Wilkie - Investor Relations | | Sandy Macrae - Chief Executive Officer | | Rob Schott - Head of Development | | Jason Fontenot - Chief Scientific Officer | | Prathyusha Duraibabu - Chief Financial Officer | | Bettina Cockroft - Chief Medical Officer | | Mark McClung - Chief Operating Officer | | Conference Call Pa ...

Sangam Pioneering the Future of Genomic Medicines August 2022 Forward-Looking Statements This presentation contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize durable, safe and effective therapies to treat certain diseases and the timing, availability and costs of such therapies, the potential to use ZFP, ZFP-TF, CAR-Treg and oth ...

Sangam Pioneering the Future of Genomic Medicines May 2022 Forward-Looking Statements This presentation contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize durable, safe and effective therapies to treat certain diseases and the timing, availability and costs of such therapies, the potential to use ZFP, ZFP-TF, ZFN, ZFP-Epi, CAR-T ...

Sangam Pioneering the Future of Genomic Medicines May 2022 Forward-Looking Statements This presentation contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize durable, safe and effective therapies to treat certain diseases and the timing, availability and costs of such therapies, the potential to use ZFP, ZFP-TF, ZFN, ZFP-Epi, CAR-T ...