GeneVentiv 获得 250 万美元 SBIR 拨款用于开发血友病基因疗法

GeneVentiv Therapeutics 已获得 250 万美元拨款，用于对其治疗 A 型和 B 型血友病的一次性输注基因疗法（使用或不使用凝血因子抑制剂）进行临床前测试。继在两种血友病小鼠模型中取得了良好的结果后，该疗法 GENV-HEM 将在一项大型研究中用血友病犬模型进行测试。资金还将用于评估基因疗法在体内的分布和相关的毒理学。这些研究预计将支持未来该疗法的研究性新药应用。

GeneVentiv Secures $2.5 Million SBIR Grant to Advance One-Time Infusion Gene Therapy for Hemophilia

GeneVentiv 获得 250 万美元 SBIR 拨款，用于推进血友病的一次性输注基因疗法

GeneVentiv Therapeutics, a pioneering biotechnology company, has received a prestigious $2.5 million Small Business Innovation Research (SBIR) grant from the National Heart, Lung, and Blood Institute, a division of the esteemed National Institutes of Health. This grant marks a significant milestone in the development of GeneVentiv's transformative gene therapy, GENV-HEM, for the treatment of hemophilia A and B, both with and without clotting factor inhibitors.

GeneVentiv Therapeutics 是一家领先的生物技术公司，已获得美国国立卫生研究院下属的国家心肺血液研究所提供的 250 万美元小型企业创新研究 (SBIR) 资助。这笔资助标志着 GeneVentiv 的变革性基因疗法 GENV-HEM 开发的一个重要里程碑，该疗法用于治疗 A 型和 B 型血友病（无论是否使用凝血因子抑制剂）。

GENV-HEM, a one-time infusion gene therapy, has demonstrated remarkable promise in treating hemophilia in preclinical mouse models. The SBIR grant will enable GeneVentiv to further evaluate the therapy's efficacy and safety in a larger study using a canine model of hemophilia. The canine model offers a more robust representation of human physiology, providing valuable insights into the therapy's performance in a setting closer to that of patients.

GENV-HEM 是一种一次性输注基因疗法，在临床前小鼠模型中显示出治疗血友病的卓越前景。 SBIR 资助将使 GeneVentiv 能够在使用血友病犬模型的更大规模研究中进一步评估该疗法的疗效和安全性。犬类模型提供了更可靠的人类生理学表征，为在更接近患者的环境中的治疗效果提供了有价值的见解。

The grant will also support critical studies to assess the biodistribution and toxicology of GENV-HEM in the body. These comprehensive investigations are essential for establishing the safety profile of the therapy and for preparing an Investigational New Drug (IND) application, which would allow GENV-HEM to enter human clinical trials.

该赠款还将支持关键研究，以评估 GENV-HEM 在体内的生物分布和毒理学。这些全面的研究对于建立该疗法的安全性和准备研究性新药 (IND) 申请至关重要，这将使 GENV-HEM 进入人体临床试验。

"This SBIR grant is a resounding endorsement of the transformative potential of GENV-HEM," said Paris Margaritis, Chief Scientific Officer of GeneVentiv. "The findings from our mouse studies have been tremendously encouraging, and this grant will enable us to validate these results in a larger animal model and conduct the rigorous safety assessments required for IND submission."

GeneVentiv 首席科学官 Paris Margaritis 表示：“这笔 SBIR 资助是对 GENV-HEM 变革潜力的充分认可。” “我们的小鼠研究结果非常令人鼓舞，这笔资助将使我们能够在更大的动物模型中验证这些结果，并进行 IND 提交所需的严格安全评估。”

Hemophilia, a rare genetic disorder characterized by excessive and prolonged bleeding, affects approximately 1 in 5,000 males worldwide. The condition arises from deficiencies or malfunctions of clotting factors, primarily factor VIII (FVIII) in hemophilia A and factor IX (FIX) in hemophilia B.

血友病是一种罕见的遗传性疾病，其特征是过度和长期出血，全世界大约有五千分之一的男性患有血友病。该病症是由凝血因子缺乏或功能障碍引起的，主要是血友病 A 中的凝血因子 VIII (FVIII) 和血友病 B 中的凝血因子 IX (FIX)。

Current treatment options for hemophilia include factor replacement therapy, which involves administering synthetic or plasma-derived clotting factors. However, this approach can be limited by the development of neutralizing antibodies (inhibitors) that prevent the clotting factors from functioning properly.

目前血友病的治疗选择包括因子替代疗法，其中涉及施用合成或血浆衍生的凝血因子。然而，这种方法可能会受到中和抗体（抑制剂）的限制，中和抗体（抑制剂）会阻止凝血因子正常发挥作用。

GENV-HEM employs a cutting-edge gene therapy approach that aims to overcome these challenges. The therapy utilizes a harmless adeno-associated virus (AAV) as a carrier to deliver a gene encoding activated clotting factor V (FVa). FVa interacts with another clotting protein, factor X (FX), initiating a cascade of events that lead to the formation of blood clots independently of FVIII or FIX.

GENV-HEM 采用尖端的基因治疗方法，旨在克服这些挑战。该疗法利用无害的腺相关病毒（AAV）作为载体来传递编码活化凝血因子V（FVa）的基因。 FVa 与另一种凝血蛋白 X 因子 (FX) 相互作用，引发一系列事件，导致独立于 FVIII 或 FIX 形成血栓。

"GENV-HEM has the potential to revolutionize the treatment of hemophilia by offering a single-infusion, lifetime solution for patients with and without inhibitors," said Damon Race, CEO of GeneVentiv. "This grant from the NIH is a pivotal step towards bringing this life-changing therapy to patients."

GeneVentiv 首席执行官 Damon Race 表示：“GENV-HEM 有潜力为血友病的治疗带来革命性的变化，为有或没有抑制剂的患者提供单次输注、终生解决方案。” “美国国立卫生研究院的这笔资助是将这种改变生活的疗法带给患者的关键一步。”

In recognition of its significant therapeutic potential, GENV-HEM has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of hemophilia A and B. GeneVentiv has also forged a strategic collaboration with ReciBioPharm to ensure that GENV-HEM meets the stringent Good Manufacturing Practice (GMP) standards and Good Laboratory Practice (GLP) requirements for manufacturing and testing.

鉴于其巨大的治疗潜力，GENV-HEM 已被美国食品和药物管理局 (FDA) 授予治疗 A 型和 B 型血友病的孤儿药资格。 GeneVentiv 还与 ReciBioPharm 建立了战略合作，以确保 GENV- HEM 符合严格的良好生产规范 (GMP) 标准和良好实验室规范 (GLP) 的制造和测试要求。

"The combined support of this grant and our manufacturing partnership with ReciBioPharm significantly reduces the financial burden on investors while maximizing resources for the critical IND-enabling studies," added Race. "This grant brings us one step closer to submitting the IND for GENV-HEM, paving the way for human clinical trials and ultimately providing a transformative treatment option for patients with hemophilia."

Race 补充道：“这笔赠款的综合支持以及我们与 ReciBioPharm 的制造合作伙伴关系显着减轻了投资者的财务负担，同时最大限度地提高了关键 IND 支持研究的资源。” “这笔资助使我们距离提交 GENV-HEM 的 IND 又近了一步，为人体临床试验铺平了道路，并最终为血友病患者提供了一种变革性的治疗选择。”

The SBIR grant from the NIH represents a testament to the groundbreaking nature of GeneVentiv's gene therapy approach for hemophilia. With promising preclinical results, a robust canine study, and comprehensive safety assessments underway, GENV-HEM is poised to make a profound impact on the lives of hemophilia patients worldwide.

NIH 的 SBIR 资助证明了 GeneVentiv 血友病基因治疗方法的开创性。凭借令人鼓舞的临床前结果、稳健的犬类研究以及正在进行的全面安全评估，GENV-HEM 有望对全世界血友病患者的生活产生深远影响。