全球基因治疗估计为2021年的亿美元市场。该gene therapy market预计在偏热市场研究报告“基因治疗竞争分析和市场预测 - [2016-2021]中，预计在2016 - 2016年2016 - 2016年期间推出了潜在疗法的潜在疗法。betway苹果下载
市场上有许多基因治疗。欧洲联盟已批准了两种基因疗法，直到现在是来自Uniqure的Glybera在2012年期间治疗脂蛋白脂肪酶缺乏症。此外，2016年，欧洲联盟批准了儿童严重综合免疫缺陷的Strimvelis。虽然FDA直到现在已经批准了三个基因疗法，即尤西巴尔为成年患者，某些类型的大型B细胞淋巴瘤患者没有回应或者在至少两种治疗后复发。2017年12月19日FDA批准了Luxturna（voretigene neparvovec-rzyl），一种新的基因治疗，治疗儿童和成年患者的遗传形式的视力丧失，可能导致失明。此外，大约12-14个新的基因疗法处于开发途径，用于各种常见条件和罕见的条件，预计将在未来2至3年内达到FDA批准阶段。
什么是基因治疗？
基因治疗is a novel method, which has the potential to treat, cure and even prevent disease by means of altering the gene expression, which in turn alters the protein in the body. Most of the diseases are caused by damaged; dysfunctional genes which when rectified can restore the normal function of the gene and give us the “long life”. Genetic disorders cause around 4000 diseases, which theoretically can be corrected by gene therapy, thus bringing the ray of hope especially for the terminally ill patients.
它是如何完成的？基因治疗的遗传ABC。
Genes are made of DNA, which carry information to produce the correct protein, which is quintessential for normal functioning of the human body. However, genetic mutations cause the DNA sequences to change leading to faulty protein sequences or non at all. To correct the faulty gene sequence, a carrier vector in the form of virus is used to deliver the modified gene. It is interesting to note here that the virus is modified as it might cause a disease on its own. The delivery of modified gene containing virus is done through injection (intravenous, subcutaneous, intraperitoneal or intramuscular). Once inside the virus integrates with the cell and produces the protein for the gene encoded, thereby eliminating the problem at the genetic level. Therefore, theoretically, gene therapy could be the proverbial “magic bullet”.
The milestones reached by Gene Therapy so far.
There are number of gene therapies for different disease segments, with cancer leading the chart with 693, followed by rare diseases with 501, besides several neurological, cardiovascular, metabolic diseases, infectious diseases, blood and clotting, musculoskeletal and others.
Opportunities and Challenges associated with Gene Therapy.
基因治疗can address the unmet clinical needs for the patients suffering from life threatening diseases, who for now have no other options except for supportive care and the prescribed treatment includes serious side effects besides being costly. However, given the fact that gene therapy is challenging to develop, it could prove difficult for small biotech companies owing to associated set-backs in the various development stages and the unforeseen side-effects of the gene therapy. To make the gene therapy available to the patients, financial viability studies are to be conducted by the manufacturers. Perhaps, new model of pricing and payment could be adopted to address the concerns.
Given the small population segment, which suffers from genetic disorders, it might affect the generation of robust clinical evidence. Also, the use of viral vectors to transfer the therapeutic gene into the target area could raise long-term safety issues.
The biggest challenge to be addressed for gene therapy is regarding the entire regime of pricing and payment as the affordability of gene therapy is one of the primary concern. To address this challenge, their needs to be collaboration for effective policy development in order to create a stable pricing, financing, and health insurance structure for gene therapy.