00:12 , Jun 7, 2019 |  BC Extra  |  Company News

June 6 Company Quick Takes: Take two for Celgene's ozanimod; plus Protalix, ABPI, Axovant and Mylan

Celgene resubmits NDA for ozanimod   FDA and EMA accepted submissions from Celgene Corp. (NASDAQ:CELG) for ozanimod to treat multiple sclerosis. The PDUFA date for the selective S1PR1 and S1PR5 modulator is March 25, 2020,...
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...
19:17 , Dec 21, 2018 |  BC Week In Review  |  Clinical News

Benitec's BB-401 misses Stage 1 response threshold in Phase II for head and neck cancer

Benitec Biopharma Ltd. (ASX:BLT; NASDAQ:BNTC) said an initial analysis of the 12-patient Stage 1 cohort in a Phase II trial to treat recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) showed...
18:36 , Dec 14, 2018 |  BC Week In Review  |  Clinical News

Axovant discontinues development of nelotanserin after Phase II readout

Axovant Sciences Ltd. (NASDAQ:AXON) discontinued development of nelotanserin after the product missed the primary endpoint in a Phase II trial to treat REM sleep behavior disorder in patients with Lewy body dementia. The double-blind trial...
19:34 , Jul 13, 2018 |  BC Week In Review  |  Company News

Axovant gets rights to Benitec's gene therapy BB-301

Axovant Sciences Ltd. (NASDAQ:AXON) broadened its gene therapy pipeline by obtaining exclusive, worldwide rights from Benitec Biopharma Ltd. (ASX:BLT; NASDAQ:BNTC) to BB-301. Axovant plans to start a placebo-controlled clinical trial next year of the gene...
04:00 , Jul 9, 2018 |  BC Extra  |  Company News

Axovant deepens gene therapy pipeline with Benitec deal

Axovant Sciences Ltd. (NASDAQ:AXON) broadened its gene therapy pipeline by obtaining exclusive, worldwide rights from Benitec Biopharma Ltd. (ASX:BLT; NASDAQ:BNTC) to BB-301. Axovant plans to start a placebo-controlled clinical trial next year of the gene...
22:21 , Oct 12, 2017 |  BC Innovations  |  Targets & Mechanisms

Mining the negatives

By investigating why its discontinued siRNA candidate against hepatitis B caused only weak responses in certain patients, Arrowhead Pharmaceuticals Inc. has upended a dogma about the virus’ genetics that has been clouding drug design and...
23:22 , Oct 11, 2017 |  BC Extra  |  Company News

Management tracks: Aerpio, Scancell, TCR2

Ophthalmic company Aerpio Pharmaceuticals Inc. (OTCQB:ARPO) named Stephen Hoffman CEO, effective Dec. 1, 2017. He succeeds Aerpio founder Joseph Gardner, who will remain its president. Hoffman was a senior advisor at PDL BioPharma Inc. (NASDAQ:PDLI),...
16:00 , Apr 25, 2017 |  BC Innovations  |  Distillery Therapeutics

Musculoskeletal

INDICATION: Muscular dystrophy Mouse studies suggest combining knockdown of mutant PABPN1 with expression of normal PABPN1 could help treat oculopharyngeal muscular dystrophy (OPMD). In a mouse model of OPMD, intramuscular injection of shRNA targeting mutant...
18:32 , Mar 8, 2017 |  BC Innovations  |  Distillery Techniques

Drug delivery; drug platforms

TECHNOLOGY: Viral vectors; gene therapy A Cas9 ortholog derived from Campylobacter jejuni could enable the use of AAV vectors to deliver CRISPR-based gene therapies. The 984-residue C. jejuni ortholog is smaller than the Cas9 enzymes...