日本药企安斯泰来（Astellas）日前宣布，与全美国最优秀的医学院——哈佛大学医学院（HMS）达成研究合作，双方已签订一项为期3年的合作协议，探索视网膜色素变性（retinitis pigmentosa，RP）的病理机制，确定新的治疗靶标，并进一步发现、开发和商业化有前途的候选药物。合作的初衷，是开发基因疗法，为全球RP患者群体提供一种新的治疗选择。此次合作，将采用无害的腺相关病毒（AAV）载体，将矫正基因递送至RP患者体内。
此次与哈佛大学的合作，也标志着安斯泰来首次宣布进军基因治疗领域。长期以来，严重的安全问题和药物递送能力方面的困境，制约着该领域的研发。不过，经过多年的耕耘，新一代学者和研究人员认为他们已经敲定了合适的病毒载体，可以安全可预测地纠正靶组织中的基因，这些突破性的进展，为眼科基因疗法的临床成功，提供了新的希望。
目前，也有其他一些财大气粗的制药公司涉足基因治疗领域。上个月，生物技术巨头百健艾迪（Biogen Idec）宣布计划，建立一个内部基因治疗部门，独立于其现有的反义RNA疗法、生物制剂、和传统小分子。在此之前，另一家生物技术巨头——新基（Celgene）也联手蓝鸟生物（Bluebrid bio）开发基因疗法，用于肿瘤学。拜耳（Bayer）已与Dimension治疗公司签署了一笔2.52亿美元的协议，开发A型血友病的基因疗法。葛兰素史克（GSK）和百特（Baxter）也在基因治疗领域有在研的实验性药物。
视网膜色素变性（RP）是一种由基因突变导致的视网膜退行性疾病，特征是视力减退、夜盲和视野缩小及眼底视网膜色素沉着，视力会逐渐消失，并且可能会遗传给下一代，目前该病还没有有效的治疗办法。而视网膜色素变性（RP）的发病机理尚未完全了解。
此次合作研究，将由哈佛医学院遗传学和眼科学教授Constance L.Cepko博士领导，Cepko博士同时也是美国非营利性医学研究所——霍华德-休斯医学研究所（HHMI）的研究员，在眼科基因治疗领域，具有享誉全球的权威性。Cepko博士表示，这些治疗方法也可有效治疗其他类型的眼科疾病，如年龄相关性黄斑变性（AMD）和青光眼（glaucoma）。
英文原文：Astellas dives into gene therapy with a Harvard expert in tow
Japan's Astellas Pharma is the latest big drugmaker allured by the promise of gene therapy, mounting an R&D effort that could lead to a new treatment for an inherited eye disease.
The company has signed a deal with Harvard Medical School investigator and gene therapy expert Constance Cepko to collaborate on a gene therapy approach to retinitis pigmentosa (RP), a degenerative eye disease that can damage vision and lead to blindness. The plan is to investigate whether harmless adeno-associated virus (AAV) vectors can be used to deliver corrective genes to RP patients, Astellas said, in the process mapping out the mutations at the heart of the disease.
Under the three-year agreement, Astellas has agreed to pay for discovery, development and, if things go according to plan, marketing, striking a commercialization agreement with Harvard with the potential to expand into other avenues of gene therapy.
"We are hopeful that some of our candidates will preserve the color and daylight vision of RP patients, regardless of the disease gene in their family," Cepko said in a statement. "These treatments may also be effective in people that have other types of ocular disease, such as age-related macular degeneration or glaucoma."
Astellas' effort is part of its Innovation Management program, a 2013 initiative designed to reshape its R&D machine by gutting some internal operations and putting a new emphasis on partnering. As part of the move, the company shut down its OSI Pharmaceuticals and Perseid Therapeutics subsidiaries, all the while scaling back its U.S.-headquartered research institute to focus solely on CNS treatments.
The Harvard deal makes for Astellas' first announced foray into gene therapy, a space in midst of a resurgence of popularity. Serious safety issues and deliverability woes long hampered R&D in the field, but, after years of work, a new generation of academics and investigators believes it has hammered out the right viral vectors to safely and predictably get corrective genes to their target tissues, spurring renewed hope for widespread clinical success.
And now deep-pocketed drugmakers are getting involved.
Last month, Biogen Idec ($BIIB) made a splash in the field by unveiling plans to build an internal gene therapy division, one that will stand alongside its existing efforts in antisense therapies, biologics and traditional small molecules. Before that, Celgene ($CELG) teamed up with bluebird bio ($BLUE) on a gene therapy approach to oncology, and Bayer signed a $252 million deal with Fierce 15 honoree Dimension Therapeutics to get its hands on a hemophilia A treatment. GlaxoSmithKline ($GSK), Baxter ($BAX) and others are working on in-house treatments in the field, as well.
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