sc-rAAV2.5IL-1Ra

▎药明康德内容团队编辑 近期，全球细胞和基因疗法（CGT）领域迎来系列进展。Sana Biotechnology公司开发的同种异体原代胰岛细胞疗法UP421在未对患者进行免疫抑制的情况下，被成功移植到一名1型糖尿病患者身上，实现了成功存活并能正常发挥功能。体内基因编辑领域迎来重要里程碑，首个通过基因编辑，在肝脏细胞的DNA中插入整个基因的婴儿在接受治疗后12周获得完全临床缓解。RheumaGen公司宣布完成1500万美元的A轮融资，所得资金将用于支持其用于治疗类风湿性关节炎的一次性、治愈性疗法RG0401的1期临床试验。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。 图片来源：123RF ———✦研发进展✦——— ◇ Capricor Therapeutics公司宣布，已完成向美国FDA提交生物制品许可申请（BLA），寻求完全批准其在研细胞疗法deramiocel（CAP-1002），用于治疗确诊为杜氏肌营养不良（DMD）相关心肌病的患者。根据新闻稿，如果获批，deramiocel将成为用于治疗DMD相关心肌病的首个获批疗法。 Deramiocel是一种同种异体细胞疗法，它们由心脏来源细胞（CDCs）组成，CDCs是一种含有心脏祖细胞的特殊细胞群。它们通过释放包含微RNA、非编码RNA和蛋白的外泌体，改善DMD患者肌肉瘢痕或纤维化、以及心脏功能。Deramiocel还显示出有效的免疫调节活性，可能促进细胞再生。支持此次BLA的HOPE-2开放标签扩展（OLE）研究的三年数据显示，接受deramiocel治疗患者的心脏和骨骼肌功能在三年期间趋于稳定，且deramiocel长期治疗具有良好的安全性。 ◇ Sana Biotechnology公司宣布，其研究者发起的“first-in-human”试验取得了初步成果。分析显示，一名1型糖尿病患者在未接受免疫抑制的情况下，成功移植了该公司开发的同种异体原代胰岛细胞疗法UP421。移植后四周的分析显示，胰岛β细胞成功存活并正常发挥功能，通过检测血液中C肽（C-peptide）的存在得以验证。 UP421是一款利用Sana的低免疫性（HIP）技术，对同种异体原代胰岛细胞进行工程化改造后生成的细胞疗法。根据新闻稿，这项研究首次证明，同种异体移植物可在具有完全免疫能力的个体中实现存活，无需依赖免疫抑制或免疫保护装置。 ◇ iECURE公司报告了正在进行中的1/2期临床试验OTC-HOPE中的首例婴儿患者的初步研究结果。该研究旨在评估体内基因编辑疗法ECUR-506，用于治疗新生儿发病的鸟氨酸氨甲酰转移酶（OTC）缺乏症的疗效和安全性。试验结果显示，这名婴儿接受ECUR-506治疗后总体耐受性良好。在单次给药最低剂量的ECUR-506治疗后12周获得完全临床缓解。该患者停止使用氨清除药物，且每日平均蛋白质摄入量增加至符合年龄标准的水平。增加蛋白质摄入后患者耐受良好，平均氨水平保持在正常范围内，并较治疗前水平有所降低。新闻稿表示，该患者是首个通过基因编辑，在肝脏细胞的DNA中插入整个基因的婴儿，代表着体内基因编辑领域的重要里程碑。 ECUR-506依赖于使用两种携带不同有效载荷但具有相同衣壳的腺相关病毒（AAV）载体。一种携带ARCUS核酸酶，靶向已被充分研究的PCSK9基因位点进行基因编辑，另一种是供体载体，用于插入功能性OTC基因。iECURE已从Precision BioSciences获得ARCUS核酸酶的许可，用于开发ECUR-506。在PCSK9位点的基因编辑为安全插入OTC基因提供了潜在路径，从而可能实现功能性基因的永久表达。 ◇ BrainChild Bio公司宣布推进其针对免疫检查点B7-H3的自体嵌合抗原受体（CAR）T细胞疗法BCB-276的临床开发计划，用于治疗一种无法治愈的小儿脑肿瘤——弥漫性内源性脑桥胶质瘤（DIPG）。发表在《自然-医学》杂志上的1期临床试验结果显示，21名DIPG患者从确诊到死亡的中位时间为19.8个月。3名在疾病进展前开始接受CAR-T治疗的患者分别在确诊后的44.6个月、45.6个月和52.5个月时仍然存活。这些初步数据表明，与目前的标准治疗相比，DIPG患者（包括进展前和进展后接受BCB-276治疗的患者）的总生存期有了显著提高。目前的标准治疗仅限于姑息性局灶性放射治疗，中位生存期为8-11个月。初步安全性分析表明，作为一种门诊治疗方案，重复的脑室内（ICV）BCB-276给药的耐受性良好，且无需进行淋巴清除化疗。 ◇ Genascence公司宣布，其用于评估在研基因疗法GNSC-001治疗膝骨关节炎（OA）的1b期临床试验DONATELLO取得了积极的安全性和生物标志物中期结果。随访6个月的分析数据显示，所有测试剂量的安全性和耐受性均可接受。生物标志物数据也令人鼓舞，滑液中平均白细胞介素1受体拮抗剂（IL-1Ra）的表达达到并维持了目标水平。此外，短期（3天）类固醇治疗的免疫调节支持更高水平的转基因表达。 GNSC-001是一款使用重组AAV表达编码IL-1Ra蛋白转基因的基因疗法。IL-1Ra蛋白是一种阻断IL-1信号传导的天然蛋白。IL-1被认为是介导骨关节炎疾病发生的关键因子，可导致炎症、关节痛和软骨损害。GNSC-001旨在通过一次注射到关节中，提供长期持久的IL-1信号通路抑制。 ◇ Solid Biosciences公司宣布，美国FDA已批准其用于治疗弗里德赖希共济失调（FA）的新型基因治疗候选药物SGT-212的IND申请。SGT-212是一种基于重组AAV的基因替代疗法，旨在通过双重给药途径（齿状核内输注和静脉输注）递送全长人共济蛋白（frataxin，Fxn），以分别提高小脑齿状核和心肌细胞中的Fxn水平。Fxn水平的恢复有望修复神经元和心肌细胞中潜在的线粒体功能障碍，从而解决该病的神经和心脏表现。该公司预计于2025年下半年启动1b期临床试验。 ◇ 中国国家药监局（NMPA）官网宣布，通过优先审评审批程序附条件批准铂生卓越生物科技（北京）有限公司申报的艾米迈托赛注射液（商品名：睿铂生）上市，用于治疗14岁以上消化道受累为主的激素治疗失败的急性移植物抗宿主病（aGVHD）。公开资料显示，艾米迈托赛注射液是铂生生物研发的人脐带间充质干细胞注射液。该项上市申请此前已经被NMPA药品审评中心（CDE）纳入优先审评。 ◇ EsoBiotec公司与Pregene Biopharma共同宣布，其在中国进行的一项由研究者发起的临床试验（IIT）已完成首例患者给药，该研究旨在评估ESO-T01治疗复发/难治性多发性骨髓瘤的安全性、耐受性和初步临床活性。ESO-T01是一种免疫屏蔽（immune shielded）的慢病毒载体，能在体内特异性地将T淋巴细胞重编程为高效的BCMA靶向CAR-T细胞。初步的临床观察表明，在不进行淋巴细胞清除的情况下，初始剂量的ESO-T01即可实现高水平的T细胞重编程和CAR-T细胞扩增。在单次给药ESO-T01后的第28天，首例接受治疗患者的骨髓中未检测到癌细胞。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ A2 Biotherapeutics公司宣布完成8000万美元的C轮融资，所得资金将用于资助公司的三项临床开发计划，以及推进基于其专有Tmod平台技术的CAR-T细胞疗法产品线。Tmod平台是一种结合了两种受体（激活剂和阻断剂）的精准靶向细胞系统，激活剂能够识别触发肿瘤细胞破坏的抗原，而阻断剂则识别保护正常细胞的抗原。该技术能够精确区分肿瘤与正常组织，确保免疫细胞仅攻击肿瘤细胞，从而实现个体化和高效的治疗效果。 该公司目前正在开展两项1/2期临床研究，一项是靶向癌胚抗原（CEA）的A2B530用于治疗胰腺癌、肺癌和结直肠癌的EVEREST-1研究，还有一项是靶向间皮素（MSLN）的A2B694用于治疗胰腺癌、卵巢癌、肺癌、结直肠癌和间皮瘤的EVEREST-2研究。这两项研究的患者都是经过BASECAMP-1研究筛选后被纳入的。BASECAMP-1研究利用人工智能（AI）支持的精准诊断，能够作为一种具有成本效益的高收益方法，为该公司所有的临床研究确定合格的患者。 ◇ XyloCor Therapeutics公司宣布完成6750万美元的B轮融资，所得款项将用于支持其一次性基因疗法XC001（encoberminogene rezmadenovec）的两项随机、双盲2期临床试验。XC001旨在促进心脏中的新血管，这些血管将绕过患病血管并改善血液流动。该疗法通过一次性局部给药策略来避免在其他基因疗法中观察到的毒性问题。这种方法使XC001能够在心脏中实现更高的基因表达，同时最大限度地减少全身载体循环和相关副作用。 该公司近期发表的1/2期临床试验（EXACT-1）的积极结果表明，XC001具有通过减少缺血负担来缓解难治性心绞痛患者胸痛的潜力。该公司计划于2025年开展两项研究，一项为XC001治疗难治性心绞痛的2b期临床试验EXACT-2，该试验采用了通过新型注射导管进行心内膜给药的非手术新方法，还有一项为XC001作为冠状动脉旁路移植手术（CABG）的辅助治疗的2期试验。 ◇ 致力于编辑人类白细胞抗原（HLA），以改变自身免疫力的RheumaGen公司宣布完成了1500万美元的A轮融资。这笔资金将支持公司完成其用于治疗耐药或难治性类风湿性关节炎的主打项目RG0401的1期临床试验。作为一次性的治愈性治疗，RG0401通过对HLA基因的DNA标记进行精确编辑，从而将患者有害的HLA等位基因转变为健康的HLA等位基因，以防止T细胞激活慢性自身免疫反应。该疗法不仅能阻止细胞和组织退化，还能阻止整个疾病进程。 ◇ Senti Biosciences公司宣布从Celadon Partners获得约1000万美元的额外投资，作为之前宣布的PIPE融资轮的补充。这笔追加资金使本轮融资总额达到4760万美元，所得款项将用于资助其主打项目SENTI-202的继续开发和扩大生产、其他研发活动以及一般公司用途。SENTI-202是一种逻辑门控的现货型CAR-NK细胞疗法候选产品，旨在选择性靶向并清除表达CD33和/或FLT3的血液恶性肿瘤，同时避免杀伤健康骨髓细胞。在截至2024年9月19日的初步临床数据中，3名急性髓系白血病（AML）患者中有2名达到了完全缓解（CR）。 ◇ 超小型基因剪刀技术生物科技公司GenKOre宣布获得了100亿韩元投资。自2019年9月成立以来，GenKOre已累计吸引307亿韩元投资，其中包括种子轮（36亿韩元）和A轮（171亿韩元）。GenKOre正在开发基于TaRGET（微小核酸酶、基于增强RNA的基因组编辑技术）的罕见疾病基因治疗方法，TaRGET是一种超小基因剪刀技术。TaRGET基因剪刀比现有的Cas9小40%，在药物递送方面具有潜在优势。 ◇ Verismo Therapeutics公司宣布与一家非营利性基金会——滤泡淋巴瘤创新研究所（IFLI）建立战略合作伙伴关系，IFLI将在未来三年内投资405万美元（59亿韩元），用于加速其候选CAR-T细胞疗法SynKIR-310的临床开发。SynKIR-310用于治疗复发/难治性B细胞非霍奇金淋巴瘤的1期临床试验正在美国开展，预计将招募18名患者，持续到2028年12月。 ◇ 艺妙神州宣布正式完成数亿元人民币的E轮融资，所得资金将用于支持公司首款原研CAR-T新药IM19在治疗晚期淋巴瘤方面的商业化布局，为患者提供全新的治疗选择，此外还将加快公司包括血液肿瘤、实体肿瘤以及自身免疫性疾病等重大疾病领域新工艺新产品的研发和临床进程。 ◇ 金唯科宣布完成数千万元阶段性融资，所得资金将加速推进AAV基因治疗管线的临床转化与商业化进程，为遗传疾病患者带去治愈希望。该公司的产品管线布局在眼科疾病、神经肌肉疾病、遗传代谢疾病等领域，首发管线JWK001项目已获批注册临床并已进入临床1/2期研究阶段，另有多个在研管线进入IIT阶段。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料（可上下滑动查看） [1] Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy. Retrieved January 2, 2025 from https://www.globenewswire.com/news-release/2025/01/02/3003570/0/en/Capricor-Therapeutics-Completes-Submission-of-Biologics-License-Application-to-the-U-S-FDA-for-Deramiocel-for-the-Treatment-of-Duchenne-Muscular-Dystrophy.html [2] Solid Biosciences Announces FDA IND Clearance for First-In-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich’s Ataxia. Retrieved January 7, 2025 from https://www.globenewswire.com/news-release/2025/01/07/3005854/0/en/Solid-Biosciences-Announces-FDA-IND-Clearance-for-First-In-Industry-Dual-Route-of-Administration-Gene-Therapy-to-Treat-Both-Neurologic-and-Cardiac-Manifestations-of-Friedreich-s-At.html [3] Sana Biotechnology Announces Positive Clinical Results from Type 1 Diabetes Study of Islet Cell Transplantation Without Immunosuppression. Retrieved January 8, 2025 from https://www.globenewswire.com/news-release/2025/01/07/3005841/0/en/Sana-Biotechnology-Announces-Positive-Clinical-Results-from-Type-1-Diabetes-Study-of-Islet-Cell-Transplantation-Without-Immunosuppression.html [4] BrainChild Bio, Inc. to Clinically Advance BCB-276, an Autologous B7-H3 Targeting CAR T-cell Therapy for Incurable Pediatric Brain Tumors. Retrieved January 8, 2025 from https://www.businesswire.com/news/home/20250106302628/en/BrainChild-Bio-Inc.-to-Clinically-Advance-BCB-276-an-Autologous-B7-H3-Targeting-CAR-T-cell-Therapy-for-Incurable-Pediatric-Brain-Tumors [5] EsoBiotec Doses First Patient in Investigator Initiated Trial of In Vivo BCMA CAR-T Candidate ESO-T01 for Multiple Myeloma. Retrieved January 8, 2025 from https://www.globenewswire.com/news-release/2025/01/08/3006092/0/en/EsoBiotec-Doses-First-Patient-in-Investigator-Initiated-Trial-of-In-Vivo-BCMA-CAR-T-Candidate-ESO-T01-for-Multiple-Myeloma.html [6] iECURE Reports Complete Clinical Response in First Infant Dosed with its In Vivo Gene Editing Candidate ECUR-506 in an Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC) Deficiency. Retrieved January 9, 2025, from https://iecure.com/news/iecure-reports-complete-clinical-response-in-first-infant-dosed-with-its-in-vivo-gene-editing-candidate-ecur-506-in-an-ongoing-phase-1-2-clinical-trial-in-ornithine-transcarbamylase-otc-deficiency/ [7] Genascence Announces Six-Month Results from Phase 1b DONATELLO Trial Indicating GNSC-001 Was Safe and Well Tolerated Across Multiple Dosing Arms. Retrieved January 9, 2025, from https://www.prnewswire.com/news-releases/genascence-announces-six-month-results-from-phase-1b-donatello-trial-indicating-gnsc-001-was-safe-and-well-tolerated-across-multiple-dosing-arms-302345061.html [8] 国家药监局附条件批准艾米迈托赛注射液上市 . From https://www.nmpa.gov.cn/zhuanti/cxylqx/cxypxx/20250102192741146.html [9] A2 Biotherapeutics Closes $80 Million Series C Financing to Advance Clinical Development of Its Precision Cell Therapies Using Its Proprietary Tmod™ Technology Platform. Retrieved January 9, 2025, from https://www.businesswire.com/news/home/20250108230204/en/A2-Biotherapeutics-Closes-80-Million-Series-C-Financing-to-Advance-Clinical-Development-of-Its-Precision-Cell-Therapies-Using-Its-Proprietary-Tmod%E2%84%A2-Technology-Platform [10] XyloCor Therapeutics Raises $67.5 Million in Series B Financing to Advance Clinical Development of Novel Gene Therapy in Cardiovascular Disease. Retrieved January 7, 2025, from https://xylocor.com/pdfs/XyloCor-Series-B-Financing-Release-010725.pdf [11] RheumaGen, Inc. Launches with $15 Million Series A Financing to Advance a New Class of HLA Gene-Editing Therapies for Major Autoimmune Diseases. Retrieved January 9, 2025, from https://www.prnewswire.com/news-releases/rheumagen-inc-launches-with-15-million-series-a-financing-to-advance-a-new-class-of-hla-gene-editing-therapies-for-major-autoimmune-diseases-302346361.html [12] Senti Bio Announces Additional $11.5 Million of Financing. Retrieved January 6, 2025, from https://www.globenewswire.com/news-release/2025/01/06/3004672/0/en/Senti-Bio-Announces-Additional-11-5-Million-of-Financing.html [13] Verismo draws $4 million to speed up developing small cell lymphoma drug. Retrieved January 8, 2025, from https://www.koreabiomed.com/news/articleView.html?idxno=26253 [14] 艺妙神州宣布完成E轮融资，开启十周年发展新征程. Retrieved January 8, 2025, from https://mp.weixin.qq.com/s?__biz=Mzg2NTY2MTMwMQ==&mid=2247485287&idx=1&sn=92076fe20f0b52b0e396f0b55bc7e1c0&chksm=cf0e96f8cadbcbc3a8d39dd823aada44c0678245d8c48c6d08a9f0bc6359d2578349363c8e57&scene=0&xtrack=1&ascene=7&devicetype=android-31&version=28003339&nettype=cmnet&abtest_cookie=AAACAA%3D%3D&lang=zh_CN&session_us=gh_313f6f7d6ba5&countrycode=NO&exportkey=n_ChQIAhIQNDiDr30xZ2UJW%2FbRh8gBsBLwAQIE97dBBAEAAAAAAAgPAdW%2FBPcAAAAOpnltbLcz9gKNyK89dVj0eQQxaost%2FdGJZv0jh9cvUvWvOLXPjBTQCHVQ2AFXEjnJ5GK0SS%2BIiLlyCBt [15] 金唯科完成数千万元阶段性融资，加速推进AAV基因治疗药物开发. Retrieved January 7, 2025, from https://www.vbdata.cn/1519002689 [16] '유전자가위' 진코어, 인터베스트서 브릿지 100억 유치. Retrieved January 3, 2025, from https://www.biospectator.com/news/view/23969 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

Mean IL-1Ra expression reached and maintained target levels in synovial fluid throughout six-month follow-up period Immune-conditioning with short steroid course supported higher levels of transgene expression Results will guide design of Phase 2 study and discussion with regulatory authorities PALO ALTO, CA, USA I January 9, 2025 I Genascence Corporation (“Genascence”), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced positive interim safety and biomarker results from the Phase 1b DONATELLO clinical trial evaluating GNSC-001. GNSC-001 is an investigational AAV gene therapy product for knee osteoarthritis (OA) that encodes interleukin 1 receptor antagonist (IL-1Ra), a potent inhibitor of interleukin 1 (IL-1). Data from the six-month analysis demonstrated acceptable safety and tolerability across all doses tested, along with encouraging biomarker data. “These six-month interim results mark a critical milestone in our mission to transform the treatment paradigm for osteoarthritis,” said Jeymi Tambiah, M.D., chief medical officer of Genascence. “The safety profile and sustained IL-1Ra expression data underscore the promise of GNSC-001 as a potential one-time gene therapy for this debilitating condition. The results from DONATELLO will guide discussions with regulatory authorities as Genascence prepares to initiate a Phase 2 trial focused on clinical efficacy.” “The ability to locally and sustainably inhibit IL-1 activity in the joint has been a long-standing goal in osteoarthritis research,” said Annahita Keravala, Ph.D., chief scientific officer of Genascence. “GNSC-001 is the first IL-1 inhibitor that has been shown to generate IL-1Ra expression levels that reach and maintain therapeutic thresholds in the synovial fluid long-term following a single administration. This highlights the potential of our gene therapy approach to address a key mediator of inflammation and disease progression in osteoarthritis and offers hope for a transformative treatment option for patients.” The DONATELLO Phase 1b clinical trial (NCT05835895) is a double-blind, placebo-controlled dose-ranging study designed to evaluate the safety, tolerability, and pharmacodynamics of a single intra-articular injection of GNSC-001 in patients with OA of the knee. The study enrolled 67 participants with OA at 10 centers across the U.S. The first five groups were randomized to receive GNSC-001 at doses of 1×10 12 vg or 1×10 13 vg, with or without a short course of oral steroids for immune-conditioning, or a placebo (saline) injection. The trial was expanded to enroll an additional, non-randomized arm. In this arm, pre-treatment synovial fluid sampling was required for entry, and subjects received 1×10 13 vg GNSC-001 with an abbreviated three-day course of oral steroids plus a local, intra-articular steroid injection. The six-month interim analysis will help guide the selection of the optimal dose of GNSC-001 as well as immune-conditioning regimens for future studies designed to evaluate clinical efficacy. Twelve-month data from DONATELLO are expected in the second quarter of 2025. Data and Safety Summary The primary endpoints of the DONATELLO clinical trial are safety and tolerability. Through six months of follow-up, data show that GNSC-001 was well tolerated, with no treatment-emergent or treatment-related deaths, serious adverse events (SAEs), or adverse event (AE)-related withdrawals reported. The most common target knee AEs included arthralgia, joint swelling, and joint effusion. The study’s secondary endpoints include expression levels of interleukin-1 receptor antagonist (IL-1Ra) in synovial fluid at Month 6, as well as change from baseline to Month 6. The interim results revealed that mean expression of IL-1Ra reached target therapeutic levels in multiple arms of the study and remained above the target threshold throughout the six-month follow up period. Immune-conditioning with a short course of steroids generally supported higher levels of prolonged IL-1Ra expression. About GNSC-001 GNSC-001 is a genetic medicine – a recombinant adeno-associated viral vector expressing an optimized form of IL-1Ra, a naturally occurring protein that blocks interleukin-1 (IL-1) signaling. IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation, joint pain, as well as cartilage destruction. GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single injection into the affected joint. GNSC-001 has received Fast Track Designation with the U.S. Food and Drug Administration. About Osteoarthritis (OA) of the Knee Osteoarthritis (OA) is a progressive joint disease that is a leading cause of disability. It is characterized by destruction of cartilage and structural changes in bone within the joint, which contribute to pain and loss of joint function. Osteoarthritis affects more than 30 million Americans and is increasing as a result of the aging population and increasing prevalence of obesity. Osteoarthritis represents a major economic burden, owing to direct medical costs and loss of productivity. Each year, millions of patients are treated for knee OA with NSAIDs, opioids, and steroid injections into the knee to manage their knee pain. There are no currently available therapies known to alter or slow down OA progression. About Genascence Corporation Genascence, a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, is developing life-changing treatments for highly prevalent conditions affecting millions of people. The company was founded in 2017 with technology licensed from three leading U.S. research institutions: Mayo Clinic, University of Florida, and NYU Langone Health. Headquartered in Palo Alto, California, Genascence’s founders and leadership team have deep experience in the design, development, and manufacturing of successful gene therapies and biological medicines. For more information, please visit www.genascence.com . SOURCE: Genascence