Beacon Therapeutics Ltd.

The VISTA Registrational Trial will assess the effect of AGTC-501 on the vision and other symptoms of X-Linked Retinitis Pigmentosa in additional patients with XLRP. AGTC-501 is an investigational gene therapy expressing the full length RPGR protein, thus addressing the full complement of photoreceptor damage caused by XLRP. VISTA will enroll patients in the United States, with additional sites expected to open in multiple countries. LONDON and CAMBRIDGE, Mass., June 12, 2024 /PRNewswire/ -- Beacon Therapeutics Holdings Limited ('Beacon Therapeutics' or 'the Company'), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced the treatment of the first patient in its registrational VISTA clinical trial with its lead asset, AGTC-501 (laruparetigene zovaparvovec). VISTA is a global randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 dose levels of AGTC-501 for the treatment of X-Linked Retinitis Pigmentosa (XLRP) compared to an untreated control group. Beacon Therapeutics plans to use the data generated from the VISTA trial, in combination with data from the Phase 1/2 HORIZON and Phase 2 SKYLINE studies, including all available long-term data, to support a BLA in the US and a MAA in Europe for the treatment of patients with XLRP. AGTC-501 has been awarded an Innovative Medicine Designation (ILAP) in the UK, Priority Medicine (PRIME) in the EU, and Fast Track in the US for the treatment of X-linked Retinitis Pigmentosa caused by mutations in the RPGR gene. Darin Curtiss, PharmD, Global Vice President of Clinical Development at Beacon Therapeutics, said, "This is an important milestone for the continued clinical development of AGTC-501, which offers the potential to transform the prognosis of patients with XLRP by enabling them to see better or maintain their current vision. We look forward to progressing AGTC-501 in the clinic and moving toward our goal of bringing a treatment to market for this life-altering disease." Dr. David Birch, Scientific Director at Retina Foundation of the Southwest, said, "We are delighted to start the final phase of this important trial of gene therapy for X-linked retinitis pigmentosa. It will bring hope to thousands of young men affected by this disease. The results so far have been very encouraging." XLRP is an orphan disease affecting approximately 17,000 patients in the US and EU5. XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age with no treatment options available. The disease is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein and is therefore expected to rescue function of both rod and cone photoreceptors, making it uniquely well-suited as a potential treatment to improve the lives of patients with XLRP. More information about the VISTA study can be found at . Contact: [email protected] Media: [email protected] About Beacon Therapeutics Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501, is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises. Find out more about Beacon Therapeutics at beacontx.com. SOURCE Beacon Therapeutics

欢迎观看，直播有奖！2022年的生物技术风险投资未能达到2021年的高度，那么2023年的融资情况如何？FiercePharma统计记录了生物技术公司超过3000万美元的融资项目，其中价值超过1亿美元的融资如下：01Septerna首席执行官：Jeffrey Finer， M.D.， Ph.D.融资轮次：B融资金额：1.5亿美元投资者：RA Capital Management，Third Rock Ventures，Samsara BioCapital，Invus，Catalio Capital Management，BVF Partners，Casdin Capital，Logos Capital，Deep Track Capital，Goldman Sachs Asset Management，Vertex Ventures HC，Mirae Asset Financial Group，Driehaus Capital Management，Woodline Partners LP，Soleus Capital和其他未公开的投资者。 Septerna的1.5亿美元B轮融资将对他们的两个新项目起到巨大推进作用，包括将其首个G蛋白偶联受体（GPCR）药物推向甲状旁腺功能减退症的临床研究。这家总部位于旧金山的生物技术公司于去年初推出，手头有1亿美元，其使命是重新设计GPCR药物。此后，Septerna又开发了第二个临床前项目，通过靶向促甲状腺激素受体来治疗格雷夫斯病。Beacon Therapeutics02首席执行官：David Fellows融资轮次：种子轮金额：9600万英镑（1.2亿美元）投资者：Syncona，Oxford Science Enterprises等Beacon融资获得9600万英镑，利用这笔资金将其新一代基因疗法引入视网膜疾病市场。他们将开发多样化的投资组合，包括名为AGTC-501的X连锁视网膜色素变性（XLRP）的2期项目，预计很快将问世。Beacon还有两个临床前项目，其中一个是在牛津大学开发的，用于治疗视锥杆营养不良症；其另一个项目适应症为干性年龄相关性黄斑变性。03Upstream Bio首席执行官：Samantha Truex融资轮次：B融资金额：2亿美元投资者：主要由Enavate Sciences和Venrock Healthcare Capital Partners投资，其他投资者包括Bain Capital Life Sciences，Wellington Management，以及A轮投资者OrbiMed，Access Biotechnology，Decheng Capital，Altshuler Shaham Provident Funds Ltd.，TCG X，HBM Healthcare Investments，Omega Funds和Samsara BioCapital专注于炎症的Upstream Bio已经获得了2亿美元的B轮融资，以支持其UPB-101的临床开发。UPB-101是一种针对哮喘的单克隆抗体，目前处于1b阶段。Upstream Bio计划利用这笔资金进行哮喘和慢性鼻-鼻窦炎伴鼻息肉的中期研究，同时为3期研究做准备。Bitterroot Bio04首席执行官：Pavan Cheruvu, M.D融资轮次：A融资金额：1.45亿美元投资者：ARCH Venture Partners，Deerfield Management，GV，Koch Disruptive Technologies，Alexandria Venture Investments和其他未具名的投资者。Bitterroot Bio在其1.45亿美元的A轮融资结束后扶摇直上，他们声称这是有史以来对以临床前心血管疾病为重点的生物技术公司的最大投资。这些资金将用于开发此类疾病的新疗法。其以肿瘤学和自身免疫学为切入点，以免疫疗法为基础，以动脉粥样硬化药物为起点进行开发。05Acepodia首席执行官：Sonny Hsiao, Ph.D.  融资轮次：D融资金额：1亿美元投资者：Digital Mobile Venture，以及未具名的投资者Acepodia在筹集了1亿美元的D轮融资后，准备加大对细胞疗法的开发。具体来说，该融资将支持Acepodia实体瘤和血液癌的药物管线项目，包括两种先导T细胞疗法：一种γδT细胞现成疗法ACE1831，用于非霍奇金淋巴瘤患者；以及针对表达EGFR的实体瘤的疗法ACE2016。D轮融资使Acepodia的总融资额达到2.59亿美元，其中包括2021年12月的1.09亿美元C轮融资。Carmot Therapeutics06首席执行官：Heather Turner融资轮次：E融资金额：1.5亿美元投资者：Deep Track Capital，5AM Ventures，Franklin Templeton，Frazier Life Sciences，Janus Henderson Investors，RA Capital Management，Millennium Management，TCGX，The Column Group，Venrock Healthcare Capital Partners和Willett Advisors。肥胖和糖尿病药物开发机构Carmot在D轮筹集到1.6亿美元，10个月后又在E轮融资中增加了1.5亿美元。该生物技术公司将利用超额认购和扩大筹款的现金，进行GLP-1/GIP受体调节剂CT-388的2期临床试验，用于患有和不患有2型糖尿病的肥胖成年人的体重管理。07ElevateBio首席执行官：David Hallal融资轮次：D融资金额：4.01亿美元投资者：AyurMaya Capital Management Fund，Woodline，Lee Family Office，Novo Nordisk，Matrix Capital Management，The Invus Group，Emerson Collective，SoftBank Vision Fund 2，Fidelity Management & Research Company，MPM Capital，F2 Ventures，Redmile Group，EcoR1 Capital，Samsara BioCapital，Citadel's Surveyor Capital，EDBI，Vertex Ventures，iTochu和一位未具名的机构投资者。 就在下文中的ReNAgade获得3亿美元融资仅仅一天后，ElevateBio就获得了4.01亿美元的融资，成为今年迄今为止最高的生物技术融资。ElevateBio在2021年获得5.25亿美元的C轮融资，之后进行了此次融资。从那时起，该公司已经推进了其技术平台，实现体内和离体疗法的开发。这家生物技术公司具有多个研发平台，包括诱导多能干细胞、RNA、细胞、蛋白质和载体工程技术，以及一个名为Life Edit的基因编辑平台。D轮融资所获得的4.01亿美元，还将用于ElevateBio的基因药物制造和工艺开发业务BaseCamp。ReNAgade Therapeutics08首席执行官：Amit Munshi融资轮次：A融资金额：3亿美元投资者：MPM BioImpact和F2 VenturesReNAgade 3亿美元的A轮融资，得益于RNA技术以及由前Moderna成员所组成的领导团队。他们的生物科学技术的差异化表现在一个融合了众多技术的平台，可以显著推动RNA疗法的扩展，尤其是通过增加基因编辑功能来实现这一愿景。ReNAgade生物技术的潜力，已经促成了与Orna Therapeutics的合作，两家公司已经吸引了默沙东成为他们的第一个客户。09Ray Therapeutics首席执行官：Paul Bresge融资轮次：A融资金额：1亿美元投资者：Novo Holdings，Deerfield Management，Norwest Venture Partners，Platanus，MRL Ventures Fund，Merck&Co. venture fund和4BIO Capital。总部位于加利福尼亚州的Ray Therapeutics已经完成了1亿美元的超额认购A轮融资，新资金将推进几个针对致盲疾病的项目。这家光遗传学公司使用基因疗法，将光敏蛋白递送到视网膜细胞，产生视觉信号，然后将其发送到大脑。该生物技术公司的光遗传学平台专为生物工程而设计，专注下一代基因型非依赖性疗法管线，用于多种视网膜疾病的视力恢复，如视网膜色素变性、其他遗传性视网膜营养不良和黄斑变性。Ray的主要候选药物RTx-015预计将很快进入临床试验。Boundless Bio10首席执行官：Zachary Hornby 融资轮次：C融资金额：1亿美元投资者：Leaps by Bayer，RA Capital Management，Sector Asset Management，Piper Heartland Healthcare Capital，Fidelity Management & Research Company，ARCH Venture Partners，Nextech Invest，Wellington Management，Vertex Ventures，Redmile Group，Citadel's Surveyor Capital，GT Healthcare Capital Partners，Alexandria Venture Investments，PFM Health Sciences，Logos Capital和City Hill Ventures。Boundless Bio是一家致力于癌基因扩增癌症的精准肿瘤学公司，于2019年完成4900万美元的A轮融资，随后于2021年4月进行了1.05亿美元的B轮融资。如今，Boundless Bio将利用其最新的1亿美元C轮融资来推动BBI-355的开发。BBI-355是一种检查点激酶1抑制剂，目前正在癌基因扩增癌症患者中进行1/2期研究，并支持其他试验和临床前研究。11Nido Biosciences首席执行官：Jeremy Springhorn, Ph.D.融资轮次：A/B融资金额：1.09亿美元投资者：5 AM Ventures，Abingworth，Bessemer Venture Partners，Eli Lilly，Osage University Partners，Bioluminescence Ventures和其他未具名投资者。总部位于马萨诸塞州沃特敦的生物技术公司Nido，已经带着1.09亿美元的种子轮、A轮和B轮融资 “露出尖尖角”，这些资金将用于开发神经系统疾病的新型精准药物。Nido的临床阶段候选药物包括NIDO-361的小分子产品，处于1期开发阶段，旨在治疗脊柱和延髓肌萎缩症。这是一种始于成年期的罕见神经肌肉疾病，也被称为肯尼迪病，会导致肌肉损失和运动神经元功能下降。Orbital Therapeutics12首席执行官：Giuseppe Ciaramella, Ph.D.  融资轮次：A融资金额：2.7亿美元投资者：ARCH Venture Partners，a16z，Newpath Partners，Abu Dhabi Growth Fund，Redmile Group，Exor N.V.，Invus，Moore Strategic Ventures，iGlobe Platinum Fund Group，Casdin Capital，Agent Capital，Alexandria Venture Investments，Rellim Capital Management，Heritage Medical Systems和其他未公开的投资者。Orbital 2.7亿美元的A轮融资令人惊讶，这得益于他们的RNA工具，以及对于下一代基因疗法的孜孜以求。这家年轻的生物技术公司拥有许多杰出的联合创始人，包括首席执行官Ciaramella，Beam Therapeutics现任总裁，以及Moderna传染病部门的前负责人。A轮融资获得的2.7亿美元将用于进一步地平台开发、研究开展、项目加速和公司建设。13Vedanta Biosciences首席执行官：Bernat Olle, Ph.D.融资轮次：E融资金额：1.065亿美元投资者：AXA IM Alts，AMR Action Fund，Bill & Melinda Gates Foundation，Skyviews Life Science，Reimagined Ventures，Fiscus Ventures，PEAK6，Atlantic Neptune，K2 HealthVentures，Korea Investment Partners，Korea Investment & Securities Asia Ltd.和Korea Investment & Securities US，PureTech Health，Revelation Partners，QUAD Investment Management，Seventure Partners，Hambro Perks和Pfizer Inc.Vedanta的1.065亿E轮融资将资助其微生物组的3期临床试验，以挑战Seres Therapeutics即将获批的针对艰难梭菌感染的SER-109疗法。Vedanta与其他微生物药物开发商的不同之处，在于它的治疗方法是通过可扩展的发酵过程，从克隆细胞库中培养细菌而制成的，这种策略被认为更合理并且可控。1.065亿美元使Vedanta能够在今年第三季度启动VE303的关键试验，同时将其第二个项目VE202推进到溃疡性结肠炎的2期研究。Abdera Therapeutics14首席执行官：Lori Lyons-Williams  融资轮次：A，B融资金额：1.42亿美元投资者：Versant Ventures，venBio Partners，Amplitude Ventures，adMare BioInnovations，Viking Global Investors，Qiming Venture Partners USA，RTW Investments，AbCellera和Northview Ventures。Abdera在获得了1.42亿美元的A轮和B轮融资后，将推动一系列放射性药物的开发。这家专注于癌症的公司的首要目标是开发一种独特的抗体，来运输比现有抗体溶解得更快的放射制剂，从而降低对于背景组织的毒性。除此之外，Abdera的主要管线产品针对δ样配体3（DLL3），这是小细胞肺癌和其他实体瘤上常见的靶标，由于表达水平低而难以治疗。Abdera计划在2024年向FDA递交DLL3靶向疗法的1期试验申请。15TORL BioTherapeutics首席执行官：David Licata  融资轮次：B融资金额：1.58亿美元投资者：Goldman Sachs Asset Management, UC, Office of the Chief Investment Officer, Bristol Myers Squibb, Deep Track Capital, Vertex Ventures, Moore Strategic Ventures, Cowen Healthcare Investments, Perceptive Xontogeny Venture Fund, Alexandria Venture Investments, OCV Partners.TORL BioTherapeutics 1.58亿美元的B轮融资，将用于推进他们的抗体药物偶联物（ADC）和单克隆抗体（mAb）的产品线。这家生物技术公司是围绕与加州大学洛杉矶分校斯拉蒙研究实验室（Slamon Research Lab）的合作而建立的，拥有两个主要项目。这两个项目目前都处于独立的1期临床试验中，一个项目针对Claudin 6，另一个针对Claudin 18.2。Flare Therapeutics16首席执行官：Amit Rakhit， M.D.融资轮次：B融资金额：1.23亿美元投资者：GordonMD Global Investments，Pfizer Ventures，Boxer Capital，Casdin Capital，Eventide Asset Management，Invus Financial Advisors，Nextech Invest，Third Rock Ventures，Agent Capital，Eli Lilly and Company，Memorial Sloan Kettering Cancer Center，Novartis，Pavilion Capital和ShangBay Capital。Flare Therapeutics的1.23亿美元B轮融资的投资者中，包括辉瑞、礼来和诺华这样的大型制药公司。这家总部位于马萨诸塞州剑桥市的生物技术公司于2021年以8200 万美元的A轮融资推出，其目标是转录因子。1.23亿美元这笔新资金，将用于启动该公司的首个临床试验FX-909。FX-909是一种针对晚期尿路上皮癌患者PPARG转录因子的小分子抑制剂。17Rapport Therapeutics首席执行官：Abraham Ceesay融资轮次：A  融资金额：1亿美元投资者：Third Rock Ventures、ARCH Venture Partners和Johnson&Johnson Innovation-JJDCRapport Therapeutics由Third Rock和强生的投资部门JJDC成立。该生物技术公司的平台，利用基因组学、蛋白质科学和脑成像技术，来识别用于精准神经医学的RAPs（受体相关蛋白）。Rapport目前有一个临床候选药物，为癫痫性精神障碍的1期研究。Noema Pharma18首席执行官：Luigi Costa融资轮次：B融资金额：1.12亿美元投资者：Forbion，Jeito Capital，UPMC Enterprises，Sofinnova Partners，Polaris Partners，Gilde Healthcare，Invus。Noema的成立得益于罗氏临床阶段的神经系统疾病药物，他们在B轮融资获得的1.12亿美元，有助于其在该领域继续深挖。Noema最主要的资产是basimglurant（以前称为NOE-101），这是一种mGluR5抑制剂，用于治疗三叉神经痛的严重疼痛，以及结节性硬化症复合体的癫痫发作，目前处于2期试验阶段。同样处于第2阶段的是 gemlapodect（以前称为NOE-105），这是一种PDE10a抑制剂，正在评估用于治疗图雷特综合征（亦称妥瑞症，Tourette's syndrome）和儿童期发病的语言流畅性障碍或口吃。还有一项NOE-115，这是一种三重再摄取抑制剂，Noema已准备好将其推进至非典型抑郁症和暴食症的中期试验。19Bicara Therapeutics首席执行官：Claire Mazumdar, Ph.D.融资轮次：B融资金额：1.08亿美元投资者：Red Tree Venture Capital，RA Capital Management，F-Prime Capital，Eight Roads Ventures，Invus，Piper Heartland Healthcare Capital，Premji Invest，Omega Funds，Bioqube Ventures，Acorn Bioventures和Janus Henderson InvestorsBicara Therapeutics已经完成了超额认购的B轮融资，以推动其项目BCA101和针对实体瘤癌症的候选产品。BCA101是一种双效双功能抗体，目前正处在头颈癌患者的1/1b期试验。他们的第二个精确肿瘤调节剂项目BCA356准备进入临床阶段。Chroma Medicine 20首席执行官：Catherine Stehman-Breen, M.D.融资轮次：B融资金额：1.35亿美元投资者：Google Ventures，ARCH Venture Partners，DCVC Bio，Mubadala Capital，Sixth Street，Alexandria Venture Investments，Atlas Venture，Casdin Capital，Cormorant Asset Management，Janus Henderson Investors，Newpath Partners，Omega Funds，Osage University Partners，Sofinnova Partners，T Rowe Price和Wellington ManagementChroma Medicine总部位于马萨诸塞州剑桥，这家生物技术公司获得了1.35亿美元来建立其平台。该平台的核心是单剂量表观遗传编辑，在不改变遗传密码本身的情况下调节基因表达。21CARGO Therapeutics首席执行官：Gina Chapman融资轮次：A融资金额：2亿美元投资者：Third Rock Ventures，RTW，Perceptive Xontogeny Venture Fund，Nextech，Janus Henderson Investors，Ally Bridge Group，Wellington Management，T. Rowe Price，Cormorant Asset Management和Piper Heartland。CARGO Therapeutics获得了2亿美元的超额认购，计划推进下一代CAR-T细胞疗法。这家新兴生物技术公司已经在临床上拥有2期就绪项目CRG-022，在超过50%的复发/难治性大B细胞淋巴瘤患者中显示出持久的反应。Paratus Sciences22首席执行官：Amir Nashat融资轮次：A融资金额：1亿美元投资者：Polaris Partners，Arch Venture Partners，ClavystBio，EcoR1 Capital，Leaps by Bayer和Alexandria Venture Investments。Paratus已融资1亿美元，并得到了大型制药风险投资部门Leaps by Bayer的支持。Paratus希望通过研究蝙蝠进化的独特特征，以治疗炎症、病毒感染等疾病。23Abivax首席执行官：Hartmut Ehrlich， M.D.融资轮次：不适用金额：1.30亿欧元投资者：CGX，Invus，Deep Track Capital，Sofinnova Partners，Venrock Healthcare Capital Partners，Great Point Partners，Deerfield Management Company，Commodore Capital，Samsara BioCapital，Boxer Capital。法国生物技术公司Abivax已经完成了一轮超额认购融资，筹集了1.3亿欧元，用于患有慢性炎症性疾病的患者的免疫疗法。Abivax的领先项目是obefazimod，针对溃疡性结肠炎患者，目前处于3期临床阶段。Hemab Therapeutics24首席执行官：Benny Sorensen, M.D., Ph.D.融资轮次：B融资金额：1.35亿美元投资者：Access Biotechnology，Deep Track Capital，Avoro Ventures，Invus，Rock Springs Capital，Maj Invest Equity，Novo Holdings，RA Capital Management和HealthCap。Hemab Therapeutics总部位于丹麦和美国，这家生物技术公司获得了超额认购的B轮资金，以加快单克隆和双特异性抗体的开发，这些抗体可以为格兰兹曼血栓衰弱症和血管性血友病提供第一个“功能性治疗”。25Aera Therapeutics首席执行官：Akin Akinc，Ph.D.融资轮次：A，B融资金额：1.93亿美元投资者：ARCH Venture Partners， GV，Lux Capital。Aera生物技术公司的交付平台，是由世界著名的合成生物学和功能基因组学专家张锋博士的实验室分离出来的，基于内源性人类蛋白质，旨在将基因药物的范围扩展到不同的组织和应用。君圣泰26首席执行官：刘利平博士融资轮次：C/C+融资金额：1.07亿美元投资单位：广东中医药大健康基金，越秀产业基金，昱烽晟泰基金。总部位于中国深圳的君圣泰获得了1.07亿美元的巨额融资，该公司正在寻求针对代谢和消化系统的新型多功能疗法。君圣泰将把这笔资金用于多个全球开发项目，包括中期和后期临床试验。君圣泰的主要产品是HTD1801，用于复杂的代谢和消化系统疾病。目前正处于针对2型糖尿病、非酒精性脂肪性肝炎和原发性硬化性胆管炎的2期试验中。该公司在2020年底筹集了6000万美元的B+轮融资。27Metagenomi首席执行官：Brian C. Thomas融资轮次：B 扩展融资金额：1亿美元投资者：Novo Holdings A/S，Catalio Capital Management，SymBiosis和其他未具名投资者。1亿美元的延期融资使Metagenomi的B轮融资总额达到2.75亿美元，之前Metagenomi与Moderna和Ionis Pharmaceuticals达成了合作协议。这家基因编辑公司计划利用这笔新资金，通过临床前开发和临床概念验证研究，来推进其领先的治疗项目。参考资料：1. Masson, G. et al. Fierce Biotech Fundraising Tracker '23: Septerna snags $150M; Crossbow takes aim with $80M. FiercePharma. 11. 07. 2023.封面图来源：123rf精彩推荐「神药」的神奇「副作用」！DS-8201恢复视力，PD-1让白发变黑！更有伟哥、二甲双胍、阿司匹林、青蒿素，欢迎补充！百济神州PD-1有望欧盟上市，在美获批还会远吗？谁的PD-1能率先出海？据研究，这能提升20%新药开发成功率和50%患者招募率，以患者为中心的临床试验获益明显小核酸巨头Alnylam之《长安三万里》——创始CEO讲述19年征程背后的神奇故事（终篇）点击阅读原文，与药时代一起快乐学习！

For some degenerative vision disorders, the therapies available don’t restore sight, they mainly slow its decline. To realize this benefit, a patient must receive an eye injection as frequently as once a month. The scientists at Tenpoint Therapeutics are trying to improve on the efficacy and the patient experience with cell therapies that restore vision with a single treatment. The research of London-based Tenpoint is still preclinical, and the startup isn’t offering any timelines for reaching human testing. But the new company emerged from stealth this past week backed by $70 million in financing and two technology platforms, each taking different approaches to getting the eye to repair itself. “We think the next step in the evolution of therapy is to replace the cells that are missing in order to restore vision,” CEO Eddy Anglade said. “We want to get to the place to restore sight.” In vision diseases such as age-related macular degeneration and a more advanced form of the disorder called geographic atrophy, the treatment options are therapies that block proteins associated with disease progression. Using an analogy that might be lost on young digital natives, Anglade compared Tenpoint’s approach to a camera and film. If the film is defective, the camera can’t take pictures. The solution is to replace the film. Tenpoint aims to do the same, replacing defective cells with functioning ones. Those cells can be engineered ex vivo in a lab, and then delivered to the eye. But the startup is also researching in vivo therapies that create those cells inside the eye. For the ex vivo approach, Tenpoint works with stem cells, which have the capacity to become almost any cell type in the body. In a lab, those cells are directed to become a cell type of interest. That cell type could be a retinal pigmented epithelial cell or a photoreceptor, depending on the disease. The startup isn’t naming the cells types or disease targets for its research just yet. The in vivo approach will use transcription factors to coax cells already in the eye to become other cell types. Anglade said this change will happen in a controlled fashion, but it will create a new population of cells to replace the ones lost to disease or genetic problems. Gene therapies for vision disorders, such as Spark Therapeutics’ Luxturna, reach their destination in the eye carried aboard an engineered virus. Anglade said Tenpoint is assessing both viral and non-viral approaches, adding that the delivery method will depend on the disease, the cell type, and that cell type’s location in the eye. Tenpoint represents an assembly of cell therapy technologies from various places. The startup’s scientific founders hail from Moorfields Eye Hospital in London; University College London Institute of Ophthalmology; Institut de la Vision in Paris; and the University of Washington. Tenpoint’s cell therapy toolbox also includes ex vivo technology acquired from SparingVision, a Paris-based company developing gene therapies for inherited eye disorders. Some of Tenpoint’s technologies have achieved preclinical proof of concept. But Anglade said more preclinical proof of concept work still needs to be done. Furthermore, the startup needs to be able to show it can manufacture these cells at scale. Tenpoint’s launch comes as other biotechs emerge with financing and clinical trial plans for vison-restoring therapies. In May, San Francisco-based Ray Therapeutics raised $100 million for clinical development of a gene therapy for the inherited disorder retinitis pigmentosa. Beacon Therapeutics launched last month with a $70 million Series A financing. Like Tenpoint, this London-based startup is also developing both ex vivo and in vivo cell therapies intended to restore vision. Anglade’s experience includes serving as the chief medical officer of Ocata Therapeutics, a biotech that was developing cell therapies for the eye until it was acquired by Astellas Pharma in 2015 in a $379 million deal. Anglade stayed on in executive roles at Astellas. Most recently, he was president and clinical lead, retina, at Janssen Research and Development. F-Prime Capital and Sofinnova Partners are Tenpoint’s founding investors. New investor British Patient Capital joined both founding investors to lead the startup’s Series A financing. All of Tenpoint’s seed investors participated in the latest round, including Qiming Venture Partners USA, Eight Roads, and the UCL Technology Fund. Regarding the startup’s name, Anglade said Tenpoint refers to 10 microseconds—the time it takes for an image to hit the retina and be processed by the brain. “The origin of the name is in the spirit of restoring that level of vision to the patient,” Anglade said.