02:37 , Sep 14, 2018 |  BC Innovations  |  Product R&D

Academia’s manufacturing problem

With backups at academic manufacturing centers beginning to hamper translation of early stage gene therapies, pushing those who can afford it to CMOs, at least one non-profit has stepped in with a model to deliver...
18:37 , Aug 17, 2018 |  BC Week In Review  |  Company News

Express Scripts wants blood disorder gene therapies exclusively

Express Scripts Holding Co. (NASDAQ:ESRX) said it is in discussions with gene therapy companies to become the exclusive distributor for blood disorder treatments, which could enable the PBM to test a payment-over-time reimbursement model. The PBM...
22:50 , Aug 15, 2018 |  BC Extra  |  Company News

Express Scripts wants blood disorder gene therapies exclusively

Express Scripts Holding Co. (NASDAQ:ESRX) said it is in discussions with gene therapy companies to become the exclusive distributor for blood disorder treatments, which could enable the PBM to test a payment-over-time reimbursement model. The PBM...
19:51 , Aug 10, 2018 |  BioCentury  |  Finance

Sound story

Preclinical data supporting Akouos Inc.’s hearing loss program convinced 5AM Ventures to re-up for a $50 million series A round, and those funds plus additions to the Akouos team have the company poised to take...
22:00 , Aug 9, 2018 |  BC Extra  |  Politics & Policy

China releases list of overseas drugs eligible for Priority Review

China’s Drug Evaluation Center posted a list of 48 drugs already approved in the U.S., EU or Japan that could be eligible for Priority Review in China. Last December, China's State Drug Administration released guidance to...
00:47 , Jul 27, 2018 |  BC Innovations  |  Product R&D

Syncona’s sister act

With the final pieces of its gene therapy portfolio in place, Syncona Investment Management Ltd. thinks its five “sister companies” are poised to capitalize on recent regulatory and technological advances in gene therapy, and push...
23:07 , Jul 20, 2018 |  BioCentury  |  Regulation

FDA’s positive reinforcement

Companies are still sifting through a half dozen gene therapy guidance documents issued by FDA, but so far it appears the recommendations support the paths taken by some of the most advanced clinical candidates for...
13:30 , Jul 11, 2018 |  BC Extra  |  Politics & Policy

FDA releases gene therapy guidance documents

FDA Wednesday released six draft guidance documents on gene therapy: three advising companies on how to use the technology to treat certain diseases, two about manufacturing and one on the design of long-term follow-up studies. The...
03:04 , Jun 30, 2018 |  BioCentury  |  Finance

Eyes on launches

The annual summer slowdown isn’t restricted to just the stock market this year, as the clinical catalyst calendar also appears to be on holiday in 3Q18. Buysiders are therefore turning their attention to a spate of...
15:32 , Jun 8, 2018 |  BC Week In Review  |  Company News

Axovant reboots with gene therapy deal

Months after a clinical failure in Alzheimer's disease destroyed most of its value, Axovant Sciences Ltd. (NASDAQ:AXON) is seeking a path forward by obtaining rights to a gene therapy for Parkinson's disease. Oxford BioMedica plc (LSE:OXB)...