PolyActiva, a clinical-stage biopharmaceutical company pioneering a novel drug delivery technology to improve outcomes for patients with ocular conditions, and RareSight, Inc., an ophthalmology-focused life sciences company committed to advancing therapies for inherited retinal diseases (IRDs), today announced a strategic collaboration to develop first-in-class therapies for rare pediatric retinal diseases that currently have no approved drug treatments..

PolyActiva是一家临床阶段的生物制药公司，率先开发一种新型药物递送技术以改善眼部疾病患者的治疗效果，RareSight, Inc.是一家专注于眼科的生命科学公司，致力于推进遗传性视网膜疾病（IRDs）的治疗方法。两家公司今日宣布达成战略合作，共同开发针对目前尚无获批药物治疗的罕见儿童视网膜疾病的首创疗法。

The research collaboration agreement, leveraging PolyActiva’s proprietary PREZIA™ platform, will offer first-of-its-kind, new chemical entity (NCE)-eligible pro-drug candidates to people affected by blinding eye conditions. Administered intravitreally, the approach will deliver sustained-release, zero-order treatment directly to the retina, requiring no patient administration—a key benefit for children and their caregivers..

这项研究合作协议利用了PolyActiva公司专有的PREZIA™平台，将为受致盲眼疾影响的人们提供首创的、符合新化学实体（NCE）资格的前药候选药物。通过玻璃体内注射给药，该方法可直接向视网膜提供持续释放、零级动力学治疗，且无需患者自行给药——这对儿童及其护理者来说是一个重要的优势。

“This collaboration represents an important step in PolyActiva’s growth as we expand our proprietary platform into new therapeutic areas,” said Jerry St. Peter, CEO and Board Director of PolyActiva. “Partnering with RareSight allows us to apply our PREZIA technology beyond glaucoma to address the urgent unmet needs of children living with inherited retinal disorders.

“这次合作代表了PolyActiva成长过程中的重要一步，因为我们正在将我们的专有平台扩展到新的治疗领域，”PolyActiva首席执行官兼董事会董事杰里·圣彼得表示。“与RareSight的合作使我们能够将PREZIA技术应用到青光眼以外的领域，满足患有遗传性视网膜疾病的儿童迫切未满足的需求。”

This effort reflects our broader vision to build a diversified ophthalmic pipeline that delivers durable, targeted, and potentially life-changing treatments for people who have long been overlooked.”.

这一努力反映了我们更广泛的愿景，即建立一个多元化的眼科产品线，为长期以来被忽视的人们提供持久、有针对性且可能改变生活的治疗方案。

“At RareSight, we are dedicated to preventing sight loss caused by rare pediatric eye diseases,” said Carmen Caricchio, CEO and Founder of RareSight. “By combining RareSight’s therapeutic discovery and development expertise with PolyActiva’s novel drug delivery platform, this collaboration represents a major step toward transforming how inherited eye diseases are treated.

“在RareSight，我们致力于预防由罕见的儿童眼科疾病引起的视力丧失，”RareSight首席执行官兼创始人卡门·卡里基奥表示。“通过将RareSight的治疗发现和开发专业知识与PolyActiva的新型药物递送平台相结合，这次合作标志着我们在改变遗传性眼病治疗方法上迈出了重要一步。”

Together, we are advancing a new class of long-acting pharmacologic therapies to address early-onset vision loss, with the potential to change a child’s life and bring hope to generations to come.”.

“我们共同推进一类新型长效药物疗法，以应对早期视力丧失，有潜力改变一个孩子的人生，并为后代带来希望。”

IRDs are rare genetic disorders that cause progressive vision loss and blindness, with symptoms often beginning in childhood. IRDs collectively affect up to 6,800,000 people worldwide.1 There are no approved drug treatments for these severe and progressive genetic ophthalmic conditions.

IRDs是罕见的遗传性疾病，会导致视力逐渐丧失和失明，症状通常在儿童时期就开始出现。IRDs总共影响全球多达680万人。这些严重且逐渐发展的遗传性眼病尚无获批的药物治疗方案。

'Children with inherited retinal diseases face lifelong visual challenges with no approved drug therapies,' said Sandeep Grover, MD, Medical Retina, Inherited Retinal Disease Specialist, and Professor of Ophthalmology based in Jacksonville, Fla. 'As a pioneer of the earliest research in this field and caring for kids and their families for more than two decades, I am encouraged by novel therapeutic strategies that support our goal of helping patients maintain vision, function, and independence as long as possible.'.

“患有遗传性视网膜疾病的儿童面临终身的视觉挑战，目前尚无获批的药物疗法，”佛罗里达州杰克逊维尔的医学视网膜、遗传性视网膜疾病专家兼眼科教授桑迪普·格罗弗医学博士说道。“作为这一领域最早研究的先驱者，并在二十多年间照顾这些孩子及其家庭，我对我们支持患者尽可能长时间维持视力、功能和独立性的目标感到鼓舞，这些新颖的治疗策略为此提供了希望。”

The collaboration with RareSight expands PolyActiva’s clinical-stage ophthalmic portfolio, which includes PA5108, a new chemical entity and biodegradable ocular micro-implant designed for sustained intraocular pressure control for up to six months with repeat dosing using the PREZIA platform. PolyActiva’s Phase 2b U.S.

与RareSight的合作扩展了PolyActiva的临床阶段眼科产品组合，其中包括PA5108——一种新的化学实体和可生物降解的眼部微植入物，旨在通过PREZIA平台重复给药，实现长达六个月的眼内压持续控制。PolyActiva的2b期美国试验。

trial is currently underway in approximately 75 patients across 12 sites to evaluate PA5108’s safety, tolerability, and durability in controlling intraocular pressure over time. By broadening its proprietary pro-drug technology into new therapeutic domains, including pediatric rare retinal diseases, both companies have the potential to develop the first-ever drug treatments for underserved patient populations..

目前，大约75名患者在12个试验点参与了PA5108的安全性、耐受性以及随时间控制眼内压的持久性的评估试验。通过将其专有的前药技术扩展到新的治疗领域，包括儿科罕见视网膜疾病，两家公司都有可能为服务不足的患者群体开发出前所未有的药物治疗方案。

About PREZIA™

关于PREZIA™

PolyActiva’s proprietary PREZIA™ drug delivery platform underpins ophthalmic therapeutics in the company’s pipeline. Unlike traditional polymer matrix or nanoparticle-based systems that rely on passive diffusion, PREZIA uses covalent bonding to attach therapeutic agents to a polymer backbone. This approach enables precise, consistent, and fully customizable drug release over periods ranging from one week to over one year.

PolyActiva公司专有的PREZIA™药物递送平台是其研发管线中眼科治疗药物的基础。与依赖被动扩散的传统聚合物基质或纳米颗粒系统不同，PREZIA采用共价键将治疗剂连接到聚合物主链上。这种方法能够在一周到一年以上的时间内实现精确、一致且完全可定制的药物释放。

The platform’s biodegradable design eliminates residual buildup and supports repeat dosing. PREZIA-based therapies can be formulated as rod-shaped implants or injectable gels and are compatible with both single-agent and combination therapies for a broad range of ocular conditions. The PREZIA technology is unique in that it allows for new patent protection over existing drugs when delivered with this platform..

该平台的可生物降解设计消除了残留堆积，并支持重复给药。基于PREZIA的疗法可以制成棒状植入物或可注射凝胶，兼容单药和联合疗法，适用于广泛的眼部疾病。PREZIA技术的独特之处在于，当通过该平台递送时，它能够为现有药物提供新的专利保护。

About PolyActiva

关于PolyActiva

PolyActiva is a clinical-stage biopharmaceutical company developing novel drug delivery solutions to improve outcomes for patients with ocular conditions. The company’s lead asset, PA5108, is a biodegradable ocular implant that delivers sustained latanoprost therapy to reduce intraocular pressure in glaucoma and ocular hypertension.

PolyActiva是一家处于临床阶段的生物制药公司，致力于开发新型药物递送解决方案，以改善眼部疾病患者的治疗效果。该公司的主要产品PA5108是一种可生物降解的眼部植入物，能够持续释放拉坦前列素疗法，用于降低青光眼和眼部高血压患者的眼内压。

For more information, visit polyactiva.com..

欲了解更多信息，请访问 polyactiva.com。

About RareSight, Inc.

关于RareSight公司

RareSight, Inc. is an ophthalmology-focused life sciences company committed to advancing therapies for inherited retinal diseases (IRDs), a group of rare genetic disorders that cause progressive vision loss and blindness. The company combines expertise in pharmacology, clinical development and commercialization to discover innovative treatments for underserved patient populations where no approved pharmacologic options exist.

RareSight, Inc. 是一家专注于眼科的生命科学公司，致力于推进遗传性视网膜疾病 (IRD) 的治疗方法，这是一组导致进行性视力丧失和失明的罕见遗传性疾病。该公司结合药理学、临床开发和商业化的专业知识，为尚未有获批药物治疗选择的服务不足的患者群体发现创新疗法。

For more information, please visit raresight.com..

有关更多信息，请访问raresight.com。

References:

参考文献：

Durham TA, Duncan JL, Ayala AR, Birch DG, Cheetham JK, Ferris FL 3rd, Hoyng CB, Pennesi ME, Sahel JA; Foundation Fighting Blindness Consortium Investigator Group. Tackling the Challenges of Product Development Through a Collaborative Rare Disease Network: The Foundation Fighting Blindness Consortium.

杜伦TA，邓肯JL，阿亚拉AR，伯奇DG，切瑟姆JK，费里斯FL 3rd，霍因CB，潘内西ME，萨赫尔JA；抗盲基金会联盟研究员组。通过协作的罕见病网络应对产品开发的挑战：抗盲基金会联盟。

Transl Vis Sci Technol. 2021 Apr 1;10(4):23. doi: 10.1167/tvst.10.4.23. PMID: 34004001; PMCID: PMC8083110..

Transl Vis Sci Technol. 2021年4月1日；10(4):23. doi: 10.1167/tvst.10.4.23. PMID: 34004001; PMCID: PMC8083110.

Contacts

联系人

PolyActiva

聚多能

Jenna Kane

珍娜·凯恩

Health+Commerce

健康+商业

jennakane@healthandcommerce.com

詹娜·凯恩@健康与商业网

RareSight

稀有景象

Carmen Caricchio, CEO and Founder

卡门·卡里基奥，首席执行官兼创始人

carmen@raresight.com

carmen@raresight.com

Source: businesswire.com

来源：businesswire.com