GeneVentiv 獲得 250 萬美元 SBIR 撥款用於開發血友病基因療法

GeneVentiv Therapeutics 已獲得 250 萬美元撥款，用於對其治療 A 型和 B 型血友病的一次性輸注基因療法（使用或不使用凝血因子抑制劑）進行臨床前測試。繼在兩種血友病小鼠模型中取得了良好的結果後，該療法 GENV-HEM 將在一項大型研究中用血友病犬模型進行測試。資金也將用於評估基因療法在體內的分佈和相關的毒理學。這些研究預計將支持未來該療法的研究性新藥應用。

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

GeneVentiv 獲得 250 萬美元 SBIR 撥款，用於推進血友病的一次性輸注基因療法

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 是一家領先的生物技術公司，已獲得美國國立衛生研究院下屬的國家心肺血液研究所提供的 250 萬美元小型企業創新研究 (SBIR) 資助。這項資助標誌著 GeneVentiv 的變革性基因療法 GENV-HEM 開發的一個重要里程碑，該療法用於治療 A 型和 B 型血友病（無論是否使用凝血因子抑制劑）。

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 是一種一次性輸注基因療法，在臨床前小鼠模型中顯示出治療血友病的卓越前景。 SBIR 資助將使 GeneVentiv 能夠在使用血友病犬模型的更大規模研究中進一步評估該療法的療效和安全性。犬類模型提供了更可靠的人類生理表徵，為在更接近患者的環境中的治療效果提供了有價值的見解。

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.

該補助金還將支持關鍵研究，以評估 GENV-HEM 在體內的生物分佈和毒理學。這些全面的研究對於建立該療法的安全性和準備研究性新藥 (IND) 申請至關重要，這將使 GENV-HEM 進入人體臨床試驗。

"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 首席科學官 Paris Margaritis 表示：“這筆 SBIR 資助是對 GENV-HEM 變革潛力的充分認可。” “我們的小鼠研究結果非常令人鼓舞，這筆資助將使我們能夠在更大的動物模型中驗證這些結果，並進行 IND 提交所需的嚴格安全評估。”

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.

血友病是一種罕見的遺傳性疾病，其特徵是過度和長期出血，全世界約有五千分之一的男性患有血友病。此病症是由凝血因子缺乏或功能障礙引起的，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.

目前血友病的治療選擇包括因子替代療法，其中涉及施用合成或血漿衍生的凝血因子。然而，這種方法可能會受到中和抗體（抑制劑）的限制，中和抗體（抑制劑）會阻止凝血因子正常發揮作用。

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 採用尖端的基因治療方法，旨在克服這些挑戰。此療法利用無害的腺相關病毒（AAV）作為載體來傳遞編碼活化凝血因子V（FVa）的基因。 FVa 與另一種凝血蛋白 X 因子 (FX) 相互作用，引發一系列事件，導致獨立於 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 執行長 Damon Race 表示：“GENV-HEM 有潛力為血友病的治療帶來革命性的變化，為有或沒有抑制劑的患者提供單次輸注、終生解決方案。” “美國國立衛生研究院的這筆資助是將這種改變生活的療法帶給患者的關鍵一步。”

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.

鑑於其巨大的治療潛力，GENV-HEM 已被美國食品和藥物管理局 (FDA) 授予治療 A 型和 B 型血友病的孤兒藥資格。符合嚴格的良好生產規範(GMP) 標準和良好實驗室規範(GLP) 的製造和測試要求。

"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 補充道：“這筆贈款的綜合支持以及我們與 ReciBioPharm 的製造合作夥伴關係顯著減輕了投資者的財務負擔，同時最大限度地提高了關鍵 IND 支持研究的資源。” 「這筆資助使我們距離提交 GENV-HEM 的 IND 又近了一步，為人體臨床試驗鋪平了道路，並最終為血友病患者提供了一種變革性的治療選擇。”

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 資助證明了 GeneVentiv 血友病基因治療方法的開創性。憑藉令人鼓舞的臨床前結果、穩健的犬類研究以及正在進行的全面安全評估，GENV-HEM 有望對全世界血友病患者的生活產生深遠影響。