Nystagmus, Pathologic

本次直播免费参加，点击上方链接马上报名！上世纪20年代初期的美国一片欣欣向荣。美国工业生产指数由1921年的67飙升至1929年6月的126，带动美国股市全面走牛。赚钱效应的影响下，投资者信心爆棚，甚至美国财政部长安德鲁·梅隆在1929年9月直接向公众保证：“现在，没有担心的理由，这一繁荣的高潮将会继续下去。”事与愿违。在梅隆发表演说的第二个月，美国股市突然暴跌，谁也没有想到这一跌就是十年，由此美国陷入人类历史上最严峻的一次大萧条。工人失业率飙升，投资者信仰丧失，极度压力之下美国精神病人大幅增长。如今历史钟摆又再次回荡到当初的位置。纷繁复杂的国际局势下，焦虑、担忧、抑郁等负面情绪频繁出现，悄然之间极有可能就进入“精神病”范畴。但在我国，对于自己可能患有精神疾病这件事，多数人都是抗拒的，既不进行精神检查，也不进行心理疏导，持续累加之下，往往造成较为严重的后果。这是一个痛苦的赛道，却正迎来它的大时代。1非正式精神病人精神疾病的范畴远超人们想象。2008年，中国疾控中心精神卫生中心公布了一条“骇人听闻”的数据：我国各类精神疾病患者人数在1亿人以上，重性精神病患人数超过1600万。彼时，中国人口数刚突破13亿人，就有超过1亿人属于惊人病，那岂不是每十个人中就有一个是“精神病”？时至今日，人们依然怀疑当初数据的准确性，但“精神病”患者数远超认知却是一个不争的事实。精神疾病，又称精神障碍，是一大类疾病的总称，分为器质性精神障碍和非器质性精神障碍两大类。主要是由于各种生物、心理和社会环境因素引起的脑功能紊乱，导致认知、情感、意志和行为等精神活动出现不同程度的障碍，可以大致分为10种精神障碍，细分之下，更是多达400余种疾病类型。图：心理健康与精神障碍分类，来源：亿欧智库当然，这400余种疾病中，大多都是轻微或短暂症状，通过心理疏导和自我调节就可缓解。但如果不及时诊疗，极有可能让病情进一步加剧。目前我国在数据库里登记在册的重性精神障碍患者数量高达660万，包含精神分裂症、分裂情感性障碍、持久的妄想症(偏执性精神病)、双相(情感)障碍、癫痫所致精神障碍、精神发育迟滞伴发精神障碍等6种。其中，精神分裂症和抑郁症是精神科最大的两种重性疾病，占平时就诊量的70%-80%。受“病耻感”以及家庭支持等因素影响，我国精神病患者就诊率偏低，其中有相当一部分患者是由于未能及时发现或者治疗所导致的重性精神障碍。不同于国内市场的沉寂，对于MNC（跨国医药集团）来说，精神疾病实则是一条极为重视的赛道。先是艾伯维宣布将现金收购Cerevel Therapeutics以获得其强大的神经科学产品管线，包括精神分裂症、帕金森病（PD）和情绪障碍等多种疾病中的潜力药物。此次交易总股权价值高达87亿美元。随后，百时美施贵宝（BMS）和Karuna Therapeutics宣布达成最终收购协议。BMS同意以140亿美元交易总价收购Karuna公司。BMS将获得Karuna的潜在“First-in-class”疗法KarXT，此药物用于治疗成人精神分裂症，上市申请已被美国FDA受理，极有可能成为近几十年来首个用于治疗精神分裂症的新机制药物。精神类疾病在国内的重视程度偏低，大多数人对于精神病人也多是歧视、嘲讽，这不仅加重了患者病情，同时也抑制了产业发展。对精神问题的关注，不仅是自身问题，也是一个社会问题。鉴于精神类疾病的特殊性，因此它不应简单划分为生病与否，而是应该按照严肃医疗和消费医疗两条赛道展开研究。严肃医疗针对重性精神障碍患者，消费医疗针对症状较轻的患者。精神类疾病患者既需要治疗，但同时更需要抚慰或引导。2严肃医疗场景严肃医疗场景，针对的都是较为严重的病人，抗精神分裂药物和抗抑郁药物约占整个市场80%以上的份额，这两款药物也是目前产业价值最大的精神类药物。01抗精神分裂药物抗精神分裂药物方面，经过多年的研发，已经经历了三代迭代。第一代抗精神分裂药物也称典型抗精神病药，主要作用于中枢D2受体。D2受体拮抗剂通常要占据超过60%的受体才能发挥作用。该类药物由于较大的锥体外系不良反应（帕金森症、肌张力障碍、静坐障碍等），临床上已被二代和三代精神病药物取代，但由于价格低廉，仍有一定市场。主要包括吩噻嗪类的氯丙嗪、硫杂蒽类的氯哌噻吨等。第二代抗精神分裂药物多通过双重阻断5-羟色胺（5-HT）和多巴胺受体而产生作用，目前常用的包括氯氮平、利培酮、奥氮平、喹硫平、鲁拉西酮和齐拉西酮等。与第一代抗精神分裂药物不同，第二代抗精神分裂药物由于特殊的神经递质受体作用机制，从而减少了不良反应，在临床上有更广阔的应用前景。第三代抗精神分裂药物是部分多巴胺受体激动剂，也作为5-HT受体的拮抗剂或弱的部分激动剂，包括阿立哌唑、依匹哌唑和卡利拉嗪。聚焦整个抗精神分裂药物市场，第二代药物由于药品数量大以及更好的安全性和疗效，占据市场最大份额。根据药渡数据，2023年上半年，国内抗精神分裂药物Top5品种占据整体销售额八成以上，分别为奥氮平（二代）销售8.42亿元，帕利哌酮（二代）销售5.96亿元，富马酸喹硫平（二代）销售3.73亿元，阿立哌唑（三代）销售3.65亿元，利培酮（二代）销售2.81亿元。不过，目前的抗精神分裂药物由于上市时间较早，专利早已过期，国内仿制药竞争充分，因此商业价值并不高。其中奥氮平各类剂型上市厂家达40家之多，利培酮31家，阿立哌唑24家，富马酸喹硫平19家，帕利哌酮5家。虽然抗精神分裂药物国内药企销量占据总市场规模的九成以上，但体现在销售额上，外企以一成的销量却获得几乎一半的销售额。随着国家集采的深入推进，外企普通剂型的市场份额被慢慢挤占，只能转为聚焦难度更高的长效剂型药物。抗精神病药长效制剂可提高患者依从性，显著降低精神分裂症患者的住院率，是预防复发的重要治疗策略，也是延长专利过期药物生命周期的重要手段。图：国内在研和上市抗精神分裂长效剂型药物，来源：兴业证券02抗抑郁症药物作为精神类疾病的另一个大分支，抑郁症近些年的关注度明显更高，但治疗缺口却更大。根据《2022年国民抑郁症蓝皮书》，我国抑郁症患病人数高达9500万，但治疗率仅为9.5%。抗抑郁症整体市场规模约170亿元，行业集中度较高，2017年以前主要被辉瑞、丹麦灵北和礼来等几家外企占据。目前，针对中度以上抑郁发作的主要治疗措施是药物治疗。临床上，抗抑郁药主要分为五类，有单胺氧化酶抑制剂（MAOI）、选择性去甲肾上腺素再摄取抑制剂（NARI）、选择性5-HT再摄取抑制剂（SSRI）、选择性5-HT及去甲肾上腺素再摄取抑制剂（SNRI）和其他类如植物药、中药类等。具体品种方面，根据药渡数据，2023年上半年销售额TOP5的抗抑郁药均为SSRI/SNRI抑制剂，分别是阿戈美拉汀片（5.78亿元）、草酸艾司西酞普兰片（4.75亿元）、盐酸舍曲林片（3.64亿元）、盐酸文拉法辛（3.51亿元）和米氮平片（3.41亿元）。这其中，草酸艾司西酞普兰片、米氮平和盐酸舍曲林片已纳入集采，其余国内仿制药争夺也日趋激烈，如马来酸氟伏沙明片报产厂家多达9家。尽管如此，外企依然占据相当可观的市场份额，如2023年上半年，辉瑞依然分别占有盐酸舍曲林和盐酸文拉法辛48.17%、23.92%份额，雅培制药占有马来酸氟伏沙明25.54%份额等。创新药方面，绿叶制药再次展现出精神疾病领域研发非凡的实力。2022年11月，绿叶制药的盐酸托鲁地文拉法辛缓释片获国家药监局批准上市，成为中国首个自主研发并拥有自主知识产权的用于治疗抑郁症的化药1类创新药，标志着我国在高失败率的CNS（中枢神经系统）创新药研制领域正走向世界前沿。2023年以来，国内抗抑郁药也先后有多款国产药获批上市，包括盐酸文拉法辛缓释片、草酸艾司西酞普兰片、盐酸氟西汀分散片、氢溴酸伏硫西汀片等，涉及立方制药、圣济堂制药、扬子江、施美药业、江北药业、欧意药业等。此外，据不完全统计，国内目前还有16款抗抑郁1类新药处于获批临床及以上阶段，涉及东阳光药（磷酸嘧替佐酮，II期）、广为医药（GW117，II期）、华海药业（盐酸羟哌吡酮，II期）、吉贝尔（JH201501，II期）、石药集团（盐酸阿姆西汀肠溶，II期）等多家药企，涵盖包括GABAA受体、NMDA受体、mGluR5等新靶点。图：在研抑郁症创新药一览，来源：锦缎研究院尽管精神类药物潜力极大，但需要用到药物治疗的精神疾病，已经是相当严重的程度，大部分轻度精神疾病可通过非药物进行前期治疗，包括心理治疗等渠道进行疏导。从投资角度考量，大多数人可能都会遭到轻度抑郁困扰，拥有更多潜在用户的消费医疗或是一个更大的市场。3消费医疗场景对于心理健康和精神卫生建设，我国可谓下足功夫。2023年底，中央财政投资106亿元，改扩建540余所医疗卫生机构，为600余所医疗卫生机构配置精神专科基本医疗设备；职工医保、居民医保政策范围内住院费用报销比例分别达到80%、70%左右；持续开展精神科医师转岗培训，累计培训1.6万余人，其中1.2万余人已加注精神科执业资质，1万余人转岗到精神科工作；支持抑郁症、睡眠障碍、焦虑障碍及药物成瘾等方向21个项目，合计国拨经费5.18亿元，建设3家国家精神疾病临床医学研究中心，促进创新和转化等等。反映到市场层面，国内资本近十年虽一直关注心理健康领域，但企业数量少、金额小，当前获得融资的企业多集中在靠前的轮次，资本多点尝试，产业发展先发优势不明显。借势2015年互联网的兴起以及海外心理健康赛道的起步，国内相关投资随之增长并出现小高峰，但此后产业内仍在探索可持续的业务模式，资本投入没有明显增长。从市场竞争梯队来看，第一梯队主要为头部互联网科技企业布局互联网医院，结合线上线下资源，提供在线心理咨询、在线处方等全流程心理诊疗服务，代表性企业如平安好医生、阿里健康等；第二梯队为数字心理健康领域独角兽企业，如好心情、壹点灵等专业心理咨询平台，拥有较多专业心理咨询师、心理医师等资源，平台具有较高知名度；第三竞争梯队为其他中小型数字心理健康服务企业，主要产品为小型心理健康应用，平台研发能力较弱，用户基数小，竞争力相对较差。图：2021-2022年心理健康领域企业融资情况，来源：亿欧智库研报人工智能技术、虚拟现实技术、脑机接口技术等的发展为心理疾病与精神障碍的治疗手段提供了更多可能性。如基于人工智能技术的智能心理机器人、以VR/AR技术为基础的虚拟现实暴露疗法，可应用于治疗创伤后精神压力症、焦虑障碍和恐惧症等。如科大讯飞、汤姆猫等科技公司均推出了基于AI技术的心理咨询业务。在线下企业中，我国早在2015年就诞生了精神病院第一股”康宁医院。根据半年度报告显示，2023年上半年，康宁医院总营收达7.78亿元，同比增长5.4%，自有医院30家，其中包括一家独立设置的互联网医院（怡宁心理互联网医院），运营床位数高达10578张。高度的政策扶持，与薄弱的产业基础间，已经形成较为明显的预期差。在人们生活水平持续提高的情况下，付费用户数或将持续攀升，市场的扩容只是时间的问题。4正视精神疾病国内精神病领域关注度低，并不是由于国内患者少，而是因为太多的忽视。现实中，每个人或多或少都对自己的身体状况感到过焦虑或者抑郁，但并不是所有人都可以借助自身力量克服的。对于那些无法凭借个人力量战胜焦虑后者抑郁的人而言，及时进行干预治疗是最好的选择。心理健康是与身体健康同等重要的事情。太多人过分关注身体健康，但对心理健康却漠不关心。无论是自己还是他人，过于漠视心理问题，极有可能造成严重的后果。这种对于心理问题的轻视，会导致对于其他精神疾病患者的歧视。对抗精神疾病最好的方法就是正视它。这种改变不仅需要发生在个人层面，同样也需要发生在社会层面，需要每个人的持续努力与坚持，并非一朝一夕的事情。近期直播推荐:

[01-November-2023] HG004 is the first China-developed AAV gene therapy medicine to be evaluated in a multinational, master-protocol clinical trial for patients with inherited retinal dystrophies caused by mutations in RPE65 gene HG004 is also first China independently-developed gene therapy drug granted both ODD and RPDD by the U.S. FDA HG004 is Company's first product candidate from robust pipeline of ophthalmology and neurology advances into the clinic SHANGHAI and CLINTON, N.J., Nov. 1, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因，"HuidaGene"), a global clinical-stage biotechnology company focused on developing CRISPR-based programmable genomic medicines, today announced the first patient has been dosed in the multinational, master-protocol Phase 1/2a clinical trial (NCT05906953; CTR20232920) of HG004 gene therapy drug, or the STAR (Safety and efficacy Trial of HG004 for leber congenital Amaurosis related to Rpe65 gene mutations) study, an adeno-associated virus 9 (AAV9) gene replacement therapy candidate for the treatment of inherited retinal dystrophies caused by RPE65 mutations (RPE65-IRD). "Commonly used AAV2 for RPE65-related retinal diseases requires high total vector dose and large injection volume emerging to safety concerns such as retinal detachment etc," said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and Chief Executive Officer of HuidaGene. "The efficient retinal transduction in the targeted RPE layers using HG004 for retinal disease allowing to lower both the total vector dose and injection volume has been validated in the HG00401 clinical trial for treating adults and children with LCA2 and makes it likely that this therapy can also potentially treat the IRD caused by RPE65 gene, as suggested by the U.S. FDA. The unmet need is huge for over 55,000 patients living with RPE65-IRD worldwide. Everyone at HuidaGene is excited about this significant milestone as we're working closely with the sites in China and U.S. to develop a safer and more efficacious 'once-and-done' therapy for the RPE65-IRD patient community globally." HG004 is an investigational gene replacement therapy product independently developed by HuidaGene. This HG004 therapy uses the recombinant AAV9 (AAV9) vector to deliver a functional human RPE65 gene to the retina and to restore, treat, and prevent blindness of children and adults with RPE65-IRDs. HG004 was granted both orphan drug designation ( ) and rare pediatric disease designation ( ) by the U.S. Food and Drug Administration (FDA) this year. The HG00401 clinical trial (NCT06088992) for Leber congenital amaurosis type 2 (LCA2) using the same product of HG004 in this multinational, multi-center, master-protocol clinical trial (HG00402; NCT05906953) for RPE65-IRDs also recently announced that the last-patient-dose has been completed with significant visual improvement and no safety concerns in both adult and pediatric populations. "Following the completion of last-patient-dose in the IIT study on October 24, the successful completion of first-patient-dose in the HG004 multinational trial this week means that that company has reached another important milestone, particularly for the global footprint. We'll work diligently to advance this global multinational, master-protocol Phase 1/2a clinical trial to support simultaneous drug marketing approval and commercialization in the future," said Xuan Yao, Ph.D., Co-founder, President, and GM for Greater China of HuidaGene. "This milestone highlights our commitment to developing novel genetic medicines to patients with high, unmet needs and the strong execution capabilities of our team. We hope that this multi-regional trial with the potential to be registrational globally can bring new, transformative therapeutic options for individuals living with RPE65-IRD worldwide." About RPE65 Mutation-Associated Inherited Retinal Dystrophies (RPE65-IRD) Inherited retinal dystrophies (IRDs) are a group of rare blinding conditions caused by mutations in any 1 of more than 250 genes. Leber's congenital amaurosis (LCA), severe early childhood-onset retinal dystrophy (SECORD), early-onset severe retinal dystrophy (EOSRD), and retinitis pigmentosa (RP) which may all be grouped under the heading of IRDs caused by the mutations in RPE65 gene (RPE65-IRDs), are considered to represent a phenotypic continuum of the same disease. The RPE65-IRDs with a typical onset between birth and 5 years of age exhibit several common clinical findings, chiefly night blindness (light staring with profound nyctalopia and nystagmus), progressive loss of visual fields, and loss of central vision. Given the often severe and early visual loss associated with RPE65-IRDs, other areas of development including speech, social skills, and behavior may also be delayed. About Multinational, Master-Protocol Clinical Trial of HG004 (NCT05906953; CTR20232920) This multinational, multicenter, multiple-cohort, dose-finding, and master-protocol clinical trial is designed to evaluate the safety, tolerability, and efficacy of HG004 in patients with inherited retinal dystrophies caused by RPE65 mutations. The primary endpoints include adverse events, certain laboratory measures, and ophthalmic examinations. This HG00402 study will also assess visual function via. multi-luminance mobility test (MLMT) where subjects will be navigating a mobility course under a variety of light levels. Following the completion of the primary study period, subjects will continue to be assessed in a long-term follow-up study of HG004. For more information, please contact: HG00402@huidagene.com or visit About HuidaGene - 辉大基因 HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing novel gene-editing tools and gene therapies to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patients' needs globally with various preclinical therapeutic programs covering ophthalmology and neurology. We are currently advancing clinical programs of HG004 in inherited retinal disease caused by RPE65 mutations (which has been granted both ODD and RPDD by U.S. FDA), HG202 CRISPR/Cas13Y RNA-editing in neovascular age-related macular degeneration (nAMD), and our preclinical pipeline, including programs HG301 CRISPR/Cas12 DNA-editing in retinitis pigmentosa, HG204 CRISPR/Cas13Y RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (MDS) (which has been granted RPDD and ODD by U.S. FDA), and HG302 CRISPR/Cas12 DNA-editing in neuromuscular diseases of Duchenne muscular dystrophy (DMD) etc. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. HuidaGene is committed to transforming the future of genome-editing medicine. For more information, please visit or follow us on LinkedIn at

HG004 is the first China-developed AAV gene therapy medicine to be evaluated in a multinational, master-protocol clinical trial for patients with inherited retinal dystrophies caused by mutations in RPE65 gene HG004 is also first China independently-developed gene therapy drug granted both ODD and RPDD by the U.S. FDA HG004 is Company's first product candidate from robust pipeline of ophthalmology and neurology advances into the clinic SHANGHAI and CLINTON, N.J., Oct. 31, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因，"HuidaGene"), a global clinical-stage biotechnology company focused on developing CRISPR-based programmable genomic medicines, today announced the first patient has been dosed in the multinational, master-protocol Phase 1/2a clinical trial (NCT05906953; CTR20232920) of HG004 gene therapy drug, or the STAR (Safety and efficacy Trial of HG004 for leber congenital Amaurosis related to Rpe65 gene mutations) study, an adeno-associated virus 9 (AAV9) gene replacement therapy candidate for the treatment of inherited retinal dystrophies caused by RPE65 mutations (RPE65-IRD). "Commonly used AAV2 for RPE65-related retinal diseases requires high total vector dose and large injection volume emerging to safety concerns such as retinal detachment etc," said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and Chief Executive Officer of HuidaGene. "The efficient retinal transduction in the targeted RPE layers using HG004 for retinal disease allowing to lower both the total vector dose and injection volume has been validated in the HG00401 clinical trial for treating adults and children with LCA2 and makes it likely that this therapy can also potentially treat the IRD caused by RPE65 gene, as suggested by the U.S. FDA. The unmet need is huge for over 55,000 patients living with RPE65-IRD worldwide. Everyone at HuidaGene is excited about this significant milestone as we're working closely with the sites in China and U.S. to develop a safer and more efficacious 'once-and-done' therapy for the RPE65-IRD patient community globally." HG004 is an investigational gene replacement therapy product independently-developed by HuidaGene. This HG004 therapy uses the recombinant AAV9 (AAV9) vector to deliver a functional human RPE65 gene to the retina and to restore, treat, and prevent blindness of children and adults with RPE65-IRDs. HG004 was granted both orphan drug designation ( ) and rare pediatric disease designation ( ) by the U.S. Food and Drug Administration (FDA) this year. The HG00401 clinical trial (NCT06088992) for Leber congenital amaurosis type 2 (LCA2) using the same product of HG004 in this multinational, multi-center, master-protocol clinical trial (HG00402; NCT05906953) for RPE65-IRDs also recently announced that the last-patient-dose has been completed with significant visual improvement and no safety concerns in both adult and pediatric populations. "Following the completion of last-patient-dose in the IIT study on October 24, the successful completion of first-patient-dose in the HG004 multinational trial this week means that that company has reached another important milestone, particularly for the global footprint. We'll work diligently to advance this global multinational, master-protocol Phase 1/2a clinical trial to support simultaneous drug marketing approval and commercialization in the future," said Xuan Yao, Ph.D., Co-founder, President, and GM for Greater China of HuidaGene. "This milestone highlights our commitment to developing novel genetic medicines to patients with high, unmet needs and the strong execution capabilities of our team. We hope that this multi-regional trial with the potential to be registrational globally can bring new, transformative therapeutic options for individuals living with RPE65-IRD worldwide." About RPE65 Mutation-Associated Inherited Retinal Dystrophies (RPE65-IRD) Inherited retinal dystrophies (IRDs) are a group of rare blinding conditions caused by mutations in any 1 of more than 250 genes. Leber's congenital amaurosis (LCA), severe early childhood-onset retinal dystrophy (SECORD), early-onset severe retinal dystrophy (EOSRD), and retinitis pigmentosa (RP) which may all be grouped under the heading of IRDs caused by the mutations in RPE65 gene (RPE65-IRDs), are considered to represent a phenotypic continuum of the same disease. The RPE65-IRDs with a typical onset between birth and 5 years of age exhibit several common clinical findings, chiefly night blindness (light staring with profound nyctalopia and nystagmus), progressive loss of visual fields, and loss of central vision. Given the often severe and early visual loss associated with RPE65-IRDs, other areas of development including speech, social skills, and behavior may also be delayed. About Multinational, Master-Protocol Clinical Trial of HG004 (NCT05906953; CTR20232920) This multinational, multicenter, multiple-cohort, dose-finding, and master-protocol clinical trial is designed to evaluate the safety, tolerability, and efficacy of HG004 in patients with inherited retinal dystrophies caused by RPE65 mutations. The primary endpoints include adverse events, certain laboratory measures, and ophthalmic examinations. This HG00402 study will also assess visual function via. multi-luminance mobility test (MLMT) where subjects will be navigating a mobility course under a variety of light levels. Following the completion of the primary study period, subjects will continue to be assessed in a long-term follow-up study of HG004. For more information, please contact: HG00402@huidagene.com or visit About HuidaGene - 辉大基因 HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing novel gene-editing tools and gene therapies to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patients' needs globally with various preclinical therapeutic programs covering ophthalmology and neurology. We are currently advancing clinical programs of HG004 in inherited retinal disease caused by RPE65 mutations (which has been granted both ODD and RPDD by U.S. FDA), HG202 CRISPR/Cas13Y RNA-editing in neovascular age-related macular degeneration (nAMD), and our preclinical pipeline, including programs HG301 CRISPR/Cas12 DNA-editing in retinitis pigmentosa, HG204 CRISPR/Cas13Y RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (MDS) (which has been granted RPDD and ODD by U.S. FDA), and HG302 CRISPR/Cas12 DNA-editing in neuromuscular diseases of Duchenne muscular dystrophy (DMD) etc. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. HuidaGene is committed to transforming the future of genome-editing medicine. For more information, please visit or follow us on LinkedIn at View original content to download multimedia: SOURCE HuidaGene Therapeutics