ABO-505

▲5月30-31日 第八届广州生物医药创新者峰会 扫码立即报名 注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。4月22日，Abeona Therapeutics宣布：其细胞疗法 prademagene zamikeracel (pz-cel，EB-101) 收到了 FDA 的完整回复信 (CRL)，FDA拒绝了该疗法上市，并要求其提供治疗的有效信息，以及满足额外的CMC 要求才能批准申请。隐性营养不良性大疱性表皮松解症(RDEB)由COL7A1基因突变引起的，以广泛的水泡和严重的皮肤伤口为特征。Pz-cel是一种体外基因校正的自体细胞疗法，用于治疗RDEB患者。该疗法由表皮片组成，旨在将功能性COL7A1基因传递到患者自身的皮肤细胞中，以实现正常的VII型胶原蛋白表达，并促进伤口愈合。2023年11月27日，FDA同意优先审查pz-cel(，EB-101)用于RDEB的生物制剂许可申请(BLA)。试验表明，pz-cel可以帮助治愈伤口，并减轻RDEB患者的疼痛。然而，FDA 对其位于俄亥俄州克利夫兰的制造工厂的检查，因 pz-cel 制造相关的问题，FDA 驳回了pz-cel的上市申请，但没有要求提供额外的试验或数据来支持后续申请。pz-cel原定PDUFA 日期是2024年 5 月 25 日，现在将有所推迟。受此消息影响，Abeona股价大跌53%。目前市值达2.01亿美元。Abeona 表示，将在pz-cel批准之前向FDA提供该疗法的CMC信息，并承诺在今年年中之前提交有关其生产流程的完整验证报告。Abeona首席执行官Vishwas Seshadri 在一份声明中表示：公司计划在第三季度重新提交申请，之后将启动新的审核流程。Seshadri 补充道，FDA 已表示，一旦 Abeona 重新提交申请，他们将迅速审查”最终申请“，时间将不超过6个月。Abeona 2023年财报显示：全年营收350万美元，同比大增147.52%；净亏损为5418.80万美元，同比扩大36.51%。Abeona研发管线包括：用于Stargardt病(STGD1)的临床前候选产品ABO-504，旨在通过使用Cre-LoxP重组酶系统实施双AAV载体策略，有效地重建全长ABCA4基因。用于X连锁视网膜劈裂症（XLRS）的临床前候选产品ABO-503。用于常染色体显性遗传视神经萎缩(ADOA)临床前候选产品：ABO-505。研发管线值得注意的是，2023年5月，FDA 批准了 Krystal Biotech 的基因疗法 Vyjuvek (beremagene geperpavec)，用于治疗6个月或以上的营养不良性大疱性表皮松解症(DEB)患者和 COL7A1 基因突变的患者。如果Pz-cel获得批准，将为RDEB患者带来福音。关于Abeona TherapeuticsAbeona Therapeutics是一家临床阶段的生物制药公司，1974年成立，总部位于美国俄亥俄州，致力于开发针对危及生命的罕见遗传疾病的细胞和基因疗法，如圣菲利波综合征(SanfilippoSyndrome)、大疱性表皮松解症和巴藤病。最初名为Chemex Corporation，2014年12月19日在纳斯达克上市。Abeona的AAV9基因治疗载体能够特异进入心脏、肝脏和肌肉，通过血脑屏障，进入中枢神经系统，这对于治疗中枢袖经系统疾病非常有用。参考资料：https://investors.abeonatherapeutics.com/press-releases/detail/276/abeona-therapeutics-provides-regulatory-update-on-pz-cel版权声明：本文转自生物药大时代，如不希望被转载的媒体或个人可与我们联系，我们将立即删除 活动推荐  5月 • 第八届大湾区生物医药创新者峰会  关键词：  小分子，XDC，多肽及GLP-1热点话题，80+行业专家（点击下方图片查看详情）▼【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

Animal proof-of-concept for AAV-based gene therapies for Stargardt disease, X-linked retinoschisis and autosomal dominant optic atrophyCLEVELAND, May 16, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced new data regarding three internally developed preclinical gene therapy product candidates from its adeno-associated virus (AAV) ophthalmology program that will be presented at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place from May 16-20, 2023 in Los Angeles, CA. “The preclinical proof-of-concept data being presented at ASGCT provides early evidence of the potential of our proprietary AAV capsids and gene constructs to express the recombinant protein in target tissues and rescue mutant phenotypes in mouse disease models,” said Brian Kevany, Ph.D., Chief Technical Officer and Head of Research at Abeona. “We look forward to pre-Investigational New Drug Application meetings with the FDA for two of our programs to take place in the second quarter of 2023.” Details for the poster presentations are as follows. Wednesday, May 17, 2023 at 12:00 PM PT In the presentation, entitled, “In Vivo Production of Full-Length ABCA4 Protein Following Cre-Mediated Recombination from Dual AAV Vectors in ABCA4-/- Mice,” Dr. Paul Wille, Director, Product Development, Abeona Therapeutics will present key findings: A dual AAV vector strategy using Cre recombinase efficiently reconstituted the ABCA4 gene, leading to full-length hABCA4 protein expression both in vitro via viral transduction of HEK293 cells and in vivo in photoreceptor cells after subretinal injection into ABCA4-/- mouse eyes.Cre-mediated recombination of ABCA4 was confirmed by mRNA sequencing of RNA from transfected HEK293 cells and AAV-dosed animals.Immunohistochemistry results confirmed correct localization of recombinant hABCA4 protein in photoreceptor cells. Thursday, May 18, 2023 at 12:00 PM PT In the presentation, entitled, “ABO-503, a Novel Gene Therapy for Treatment of X-Linked Retinoschisis,” Dr. Joseph Fogerty, Senior Scientist, Product Development, Abeona Therapeutics will present key findings: Robust RS1 expression was observed in photoreceptor cells near the injection site and in the adjacent inner retina in mutant mice six months after treatment with ABO-503.RS1 expression was associated with improvement in cone photoreceptor density and increased thickness of the photoreceptor outer nuclear layer.Full-field flicker electroretinogram (ERG) analysis showed significant improvement in cone photoreceptor function. Friday, May 19, 2023 at 12:00 PM PT In the presentation, entitled, “AAV Gene Therapy for Autosomal Dominant Optic Atrophy Caused by Mutation in the Opa1 Gene,” Dr. Rachel Stupay, Senior Scientist, Product Development, Abeona Therapeutics will present key findings: Vectors expressing Opa1 showed robust expression at both RNA and protein levels both in vitro and in vivo.In Opa1 knockout mouse fibroblasts, isoform variants 1, 5 and 7 expressed both RNA and protein with each variant corresponding to the expected cleavage pattern.Following intravitreal injection in wild type mice, variants 1 and 7 showed robust protein expression. The posters presented at ASGCT are available on ASGCT’s ePoster site and the Events page of Abeona’s website at https://investors.abeonatherapeutics.com/events. About Abeona’s AAV Ophthalmology Program Abeona’s preclinical programs are investigating the use of novel adeno-associated virus (AAV) strategies in therapies for serious genetic eye diseases. The most common form of Stargardt disease is caused by mutations in the ABCA4 gene, which prevents removal of toxic compounds from photoreceptor cells that results in photoreceptor cell death and progressive vision loss. ABO-504 is designed to efficiently reconstitute the full-length ABCA4 gene by implementing a dual AAV-vector strategy using the Cre-LoxP recombination system. XLRS is a rare, monogenic retinal disease that results in the irreversible loss of photoreceptor cells and severe visual impairment. XLRS is caused by mutations in the RS1 protein, which is normally secreted by retinal photoreceptors and bipolar neurons and functions to mediate cell-cell adhesion. ABO-503, composed of a functional human RS1 packaged in the novel AIM™ capsid AAV204, has shown preclinical efficacy following delivery to the retina in a mouse model of XLRS. ADOA, a form of hereditary vision loss associated with retinal ganglion cell (RGC) death, is predominantly caused by mutations in the Opa1 gene. ABO-505 is designed to express a functional copy of human Opa1 in the retina following para-retinal injection. ABO-505 aims to take advantage of the robust optic nerve and RGC transduction ability of AAV204 to deliver its genetic payload to the cells most affected by ADOA. About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona’s lead clinical program is EB-101, its investigational autologous, engineered cell therapy currently in development for recessive dystrophic epidermolysis bullosa. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona’s fully integrated cell and gene therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL™ study and is capable of clinical and potential commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com. Forward-Looking Statements This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as “may,” “will,” “believe,” “anticipate,” “expect,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, the timing and outcome of our Biologics License Application submission to the FDA for EB-101; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company’s most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.