Incentives for Starting Small Companies Focused on Rare and Neglected Diseases.
Incentives for Starting Small Companies Focused on Rare and Neglected Diseases.
复制标题
DOI：
10.1007/s11095-015-1841-9
复制
发表时间：
2016-04
影响因子：
3.7
通讯作者：
Wood J
中科院分区：
文献类型：
--
作者：
Ekins S;Wood J
传统上，生物技术或制药公司的初创企业被认为是以一位科学家、他们的技术平台或从另一家公司分拆出来的临床候选人为基础的。在我们之间，我们采取了不同的方法，在最初利用父母的视角与患有危及生命的罕见疾病的孩子一起组建了两家小型早期公司。Phoenix Nest（http：//www.phoenixnestbiotech.com/）是一家致力于治疗Sanfilippo综合征（一种毁灭性的神经退行性溶酶体贮积症）的公司。在短短3年多的时间里，我们与学术界和工业界的顶尖科学家建立了合作关系，并获得了多项NIH小企业赠款。第二家公司是Collaborations Pharmaceuticals Inc.。(http：//www.collaborationspharma.com/）的成立是为了解决其他7000种左右的罕见疾病以及被忽视的传染病。罕见儿科疾病优先审查计划可能是对那些致力于研究少数人群罕见疾病的公司最重要的激励措施。这也可能是最近收购具有晚期候选人的罕见病公司的部分原因。在创办我们公司的过程中吸取的教训是，罕见病父母或患者可以很容易地与科学家合作，并通过NIH赠款而不是风险投资或天使投资人资助研究。这个过程可能会很慢，所以耐心和毅力是关键。我们将鼓励其他制药科学家与罕见疾病的父母、患者或倡导者会面，并与他们合作，进一步研究他们的疾病，创造未来药物的来源。
Starting biotech or pharmaceutical companies is traditionally thought to be based around a scientist, their technology platform or a clinical candidate spun out from another company. Between us we have taken a different approach and formed two small early stage companies after initially leveraging the perspective of a parent with a child with a life-threatening rare disease. Phoenix Nest (http://www.phoenixnestbiotech.com/) was co-founded to work on treatments for Sanfilippo syndrome a devastating neurodegenerative lysosomal storage disorder. In the space of just over 3 years we have built up collaborations with leading scientists in academia and industry and been awarded multiple NIH small business grants. The second company, Collaborations Pharmaceuticals Inc. (http://www.collaborationspharma.com/) was founded to address some of the other 7000 or so rare diseases as well as neglected infectious diseases. The Rare Pediatric Disease Priority Review Voucher is likely the most important incentive for companies working on rare diseases with very small populations. This may also be partially responsible for the recent acquisitions of rare disease companies with late stage candidates. Lessons learned in the process of starting our companies are that rare disease parents or patients can readily partner with a scientist and fund research through NIH grants rather than venture capital or angel investors initially. This process may be slow so patience and perseverance is key. We would encourage other pharmaceutical scientists to meet rare disease parents, patients or advocates and work with them to further the science on their diseases and create a source of future drugs.