- August 20, 2019
Topical Gene Therapy Shows Promise for DEB
A novel topical gene therapy, developed by Krystal Biotech, shows promise for treating wounds of patients with dystrophic epidermolysis bullosa (DEB). The therapy KB103 targets the type VII collagen gene (COL7A1), missing in patients with DEB, to improve wound closure and skin cohesion.
- August 20, 2019
Old made new: Alkahest plasma fraction research entering another phase II
Chief Commercial and Strategy Officer Elizabeth Jeffords told BioWorld that the endpoints in Alkahest Inc.’s just-begun phase II trial with human plasma fraction GRF-6021 will not only test how well patients bounce back from hip or knee arthroplasty but “could absolutely support the work in cognitive and neurodegenerative indications as well,” where data rolled out earlier this month.
- August 15, 2019
In conversation with Samumed CEO Osman Kibar: Drugging Wnt, restoring youth and unconventional capital
- August 14, 2019
Straight A’s for MarzAA
Last month at the 2019 Congress of the International Society on Thrombosis and Haemostasis (ISTH), Catalyst Biosciences Inc. presented Phase 2 data regarding its subcutaneous (SQ) Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA) for prophylaxis in patients with hemophilia A or B with inhibitors. The study met its primary endpoint of significantly reducing the annualized bleed rate, as well as all secondary endpoints of safety, tolerability and lack of anti-drug antibody or inhibitor formation.
- August 12, 2019
ProQR’s retinitis pigmentosa candidate gets investigational NDA clearance
The FDA has cleared an investigational new drug application for QR-1123, an investigational oligonucleotide treatment candidate for vision loss with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene, according to a press release from ProQR Therapeutics.
- August 8, 2019
Inside the Arena Makeover | Kevin Lind, CFO, Arena Pharmaceuticals
Up until about three years ago Kevin Lind would likely have been identified as yet another gifted private equity executive – capable of issuing business remedies from the tip of his tongue. At least in the minds of his CFO peers, who are accustomed to listening to PE pundits routinely hand down such remedies for ailing businesses. For Lind the CFO office at Arena Pharmaceuticals is a game changer, where he no longer hands down remedies but serves as a finance leader – an individual tasked with summoning others forward and building trust across an organization even as he or she sometimes completes necessary layoffs.
- August 7, 2019
Initial Reports Positive for Plasma Treatment of Alzheimer Disease
Officials are optimistic about the initial results of a phase 2 clinical trial for a new plasma-based treatment for mild-to-moderate Alzheimer disease (AD). California-based Alkahest announced that GRF6019, a proprietary plasma fraction comprised of about 400 proteins, was both safe and well-tolerated with no cognitive decline as a treatment for AD.
