Day: May 12, 2017

Dimension Therapeutics Discontinues DTX101, a Investigational Gene Therapy Product

Dimension Therapeutics announced it will discontinue the development of DTX101, an investigational AAVrh10-based gene therapy product in development for the treatment of moderate/severe-to-severe hemophilia B. The decision followed the review of the emerging DTX101 Phase 1/2 clinical study data, including the data as of the beginning of May 2017, and the observation that the data would not meet […]

Sangamo Therapeutics And Pfizer Announce Collaboration For Hemophilia A Gene Therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter. Click here to read the full press release from Sangamo Therapeutics.

uniQure Announces Successful Gene Therapy Delivery in Animal Model

uniQure recently presented data at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting in Washington, D.C. on successful repeated hepatic gene delivery with their AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). The results described successful sequential transduction with the two reporter transgenes and highlighted the progress made in readministration protocols […]

Kedrion Announces Return to Normal Supply Levels of Koāte

Kedrion Biopharma announced a return to normal supply levels of Koāte in the 1000 vial size and a series of large-scale improvements to their overall operations following a temporary period of limited availability of product. Click here to read the full contents of letter provided by Kedrion Biopharma regarding this matter.