FFB WRITTEN ARTICLES » RETINITIS PIGMENTOSA
Retinal Gene Therapy Developer AGTC Receives $37.5 Million in Venture Capital
Nov. 19, 2012 – Applied Genetic Technologies Corporation (AGTC), a leading developer of gene therapies, has received $37.5 million in Series B (second round) venture capital funding to advance potential gene therapies for the retinal degenerative diseases achromatopsia and X-linked retinoschisis (XLRS) toward human studies. The investment will also enable AGTC to continue its Phase II clinical trial for alpha-1 antitrypsin deficiency, a condition which causes serious lung and liver dysfunction.
The Foundation Fighting Blindness is currently funding translational research in support of AGTC’s emerging gene therapies for achromatopsia and XLRS. Dr. Jeff Chulay is leading the preclinical effort for XLRS at Oregon Health & Science University, and Dr. William Hauswirth is conducting achromatopsia research at the University of Florida. By demonstrating safety and efficacy in the laboratory, AGTC will be well positioned to move the potential treatments into clinical trials.
“We are delighted by this major boost in funding for these two important gene therapy projects,” says Dr. Stephen Rose, chief research officer of the Foundation Fighting Blindness. “The investment provides us critical momentum in moving these potentially sight-saving treatments into human studies and out to the people who need them. We have the opportunity to save vision for tens of thousands of people.”
AGTC’s gene therapies work by delivering copies of healthy genes to the retina to replace the defective copies that are causing vision loss. A human-engineered virus known as an adeno-associated virus, or AAV, is used to deliver copies of the corrective genes to the patients’ retinal cells. Researchers believe that a single ocular injection of the treatment will halt vision loss for several years or perhaps a lifetime.
Often referred to as day blindness, achromatopsia is caused by genetic defects that affect cones, the cells in the retina that provide central and daytime vision, as well as the ability to perceive colors and detail. Approximately 22,000 people in the United and Europe, and tens of thousands more around the world, are affected by achromatopsia.
Affecting males, XLRS is a genetic condition causing progressive and severe loss of central vision due to splitting of the layers of the retina. XLRS affects tens of thousands of people all over the world. Approximately 35,000 have the condition in the United States and Europe.
The second round of venture capital investing in AGTC’s gene therapies is being led by Alta Partners. Additional investors include: SROne, Osage University Partners, InterWest, Intersouth and MedImmune Ventures.
“It is critical that our research attracts outside investment to move treatments into clinical trials. These human studies cost tens and sometimes hundreds of millions of dollars,” says William Schmidt, chief executive officer of the Foundation. “It is imperative that we provide compelling evidence for safety and effectiveness in the lab to build investor confidence in their potential for saving vision in people.”
视网膜基因疗法开发AGTC获得3750万美元风险投资中的
2012年11月19号-基因疗法的领先开发商，应用基因技术公司（AGTC），已收到3750万美元的B系列（第二轮）风险投资的资金，以促进潜在的基因治疗的视网膜变性疾病色盲和X-连锁视网膜（XLR的）对人类的研究。的投资也将启用AGTC，以继续其第二阶段II临床试验为α-1抗胰蛋白酶缺乏症，会导致严重的肺和肝功能障碍的条件。
该基金会战斗失明是目前资金转化研究的支持AGTC的新兴基因疗法为色盲和XLR的。杰夫Chulay博士领导的临床前努力为XLR的俄勒冈卫生科学大学，并在佛罗里达大学的威廉·豪斯维特博士进行色盲研究。通过展示在实验室中的安全性和有效性，AGTC将进入临床试验准备就绪，将潜在的治疗方法。“我们很高兴这主要提升在这两个重要的基因治疗项目提供资金，斯蒂芬·罗斯博士说：”首席研究基金会战斗失明的官员。“投资把这些潜在的挽救视力的治疗方法，为人类研究的人谁需要他们为我们提供了重要的势头。我们有机会挽救视力为数万成千上万的人。“ AGTC的基因疗法的工作，提供健康的基因副本的视网膜，造成视力减退，以取代有缺陷的副本。甲人体工程的病毒被称为腺伴随病毒或AAV，用于提供患者的视网膜细胞的纠正基因的副本。研究人员认为，一个单一的眼部注射治疗数年或阻止视力减退可能是一辈子的。通常被称为昼盲，色盲是由遗传缺陷，影响视锥细胞，细胞在视网膜的中央和白天的视野，以及能够感知的颜色和细节。约22,000名员工，在美国和欧洲，和世界各地的数千数万，受影响男性色盲。，XLR的是一种遗传性疾病，导致进步，严重的导致中心视力丧失的视网膜层分裂。XLR的影响数十万世界各地的人。大约有35,000具备的条件，在美国和欧洲。AGTC的基因疗法的第二轮风险资本投资正在由阿尔塔合作伙伴。其他投资者包括：SROne，奥沙大学的合作伙伴，主语，Intersouth和MedImmune公司的风险投资公司。“这是至关重要的，我们的研究吸引外来投资将进入临床试验阶段的治疗。这些人的研究耗资数，有时数百数百万美元，“威廉·施密特说，基金会的行政总裁。“这是我们必须提供令人信服的证据的安全性和有效性在实验室中建立投资者的信心，在他们的潜能，为节省人们的视觉。” |
