21:26 , Jul 18, 2019 |  BC Innovations  |  Emerging Company Profile

DNA Script: enzymatic DNA synthesis

DNA Script believes its enzymatic DNA synthesis technology will speed R&D by producing DNA more quickly than chemical methods and putting the process into researchers' hands. Synthetic DNA production, a cyclical process that adds one...
02:22 , Apr 5, 2019 |  BC Innovations  |  Tools & Techniques

Second shot for shRNA

Having long been demoted to tool compound status, shRNA is gaining renewed attention as a therapeutic modality by offering a simpler path to creating next-generation cell therapies than gene editing. Celyad has jumped in, using...
00:21 , Aug 10, 2018 |  BC Week In Review  |  Clinical News

RegenxBio discloses second serious AE for hypercholesterolemia gene therapy

RegenxBio Inc. (NASDAQ:RGNX) said RGX-501 led to a second treatment-related serious adverse event in a Phase I/II trial to treat homozygous familial hypercholesterolemia (HoFH). The open-label, international trial has enrolled six patients to receive a...
04:56 , Jan 19, 2018 |  BC Week In Review  |  Clinical News

Ultragenyx reports interim Phase I/II data for DTX301 in OTC deficiency

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) reported interim data from three evaluable patients in the first cohort of a Phase I/II trial evaluating a single peripheral IV infusion of 2x10 12 GC/kg DTX301 to treat ornithine transcarbamylase...
21:20 , Feb 3, 2017 |  BC Week In Review  |  Clinical News

DTX101: Preliminary Ph I/II data

Preliminary data from 6 patients with moderate/severe to severe hemophilia B in an open-label, international Phase I/II trial showed that single doses of 1.6x10 12 and 5x10 12 GC/kg DTX101 led to peak Factor IX...
01:21 , Feb 1, 2017 |  BC Extra  |  Clinical News

Dimension falls on liver toxicity concerns

Dimension Therapeutics Inc. (NASDAQ:DMTX) fell $2.30 (54%) to $1.95 after it said it would wait for advice before advancing gene therapy candidate DTX101 to the third cohort of a Phase I/II trial to treat hemophilia...
07:00 , Sep 5, 2016 |  BC Week In Review  |  Clinical News

InCVAX vaccine: Phase II/III started

Immunophotonics began an open-label, South American Phase II/III trial to compare inCVAX with or without a regulatory T cell inhibitor vs. standard of care in about 246 patients. Patients will receive up to 4 treatments...
08:00 , Jan 11, 2016 |  BC Week In Review  |  Clinical News

AMT-060: Phase I/II data

Top-line data from 2 adult hemophilia B patients in the low-dose cohort of an open-label, European Phase I/II trial showed that a single IV infusion of 5x10 12 gc/kg AMT-060 led to confirmed Factor IX...
07:00 , Sep 8, 2014 |  BC Week In Review  |  Financial News

GC-Rise completes venture financing

GC-Rise Pharmaceutical Co. Ltd. , Beijing, China   Business: Genitourinary, Autoimmune, Infectious   Date completed: 2014-09-02   Type: Venture financing   Raised: Not disclosed   Investor: OrbiMed Advisors   Note: GC-Rise secured $15 million in...
08:00 , Feb 13, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Cancer

This week in therapeutics Indication Target/marker/pathway Summary Licensing status Publication and contact information Cancer Cancer RNA polymerase I (Pol I) In vitro studies identified a small molecule that causes the degradation of the RPA194 catalytic...