17:48 , May 28, 2019 |  BC Innovations  |  Distillery Techniques

Raising lentiviral surface levels of CD47 to improve therapeutic index

TECHNIQUES CATEGORY: Drug delivery; drug platforms TECHNOLOGY: Viral vectors; gene therapy A method for increasing the surface density of CD47 on lentiviral vectors could enhance the safety and duration of Factor IX-based hemophilia B gene...
23:01 , May 22, 2019 |  BC Extra  |  Preclinical News

SR-Tiget-led team boosts ‘don’t eat me’ signals on viral vectors to improve efficacy

Boosting surface levels of a "don't eat me" signal on lentiviral vectors could enable administration of lower gene therapy doses and prevent immune responses by shielding the vectors from phagocytosis. HIV-derived lentiviral gene therapy vectors...
20:44 , Apr 18, 2019 |  BC Extra  |  Clinical News

FDA draft guidance suggests comparing bispecifics to monospecific antibodies

Demonstrated efficacy of a bispecific antibody against monospecific products targeting the different antigens could be a requirement for gaining FDA approval, according to draft guidance released Thursday. The guidance describes non-clinical and clinical recommendations for...
17:46 , Mar 20, 2019 |  BC Extra  |  Company News

Pfizer gains option to buy liver gene therapy company Vivet

Pfizer's latest gene therapy deal gives it a 15% stake in Vivet and comes with an exclusive option to buy the rare liver disease company after the French company advances its Wilson disease candidate through...
01:10 , Mar 2, 2019 |  BioCentury  |  Product Development

Roche broadens its BD scope

Roche’s proposed acquisition of Spark Therapeutics Inc. is the pharma’s first major deal since James Sabry took over partnering, and could be a glimpse of what’s to come as the pharma broadens its horizons on...
20:03 , Mar 1, 2019 |  BC Week In Review  |  Company News

Spark gives Roche beachhead in gene therapy

Roche (SIX:ROG; OTCQX:RHHBY) will acquire Spark Therapeutics Inc. (NASDAQ:ONCE) for $4.8 billion in a deal that marks the Swiss pharma's first major move into gene therapy. The deal announced early Feb. 25 was the first...
07:11 , Feb 25, 2019 |  BC Extra  |  Company News

Spark gives Roche beachhead in gene therapy

Roche (SIX:ROG; OTCQX:RHHBY) will acquire Spark Therapeutics Inc. (NASDAQ:ONCE) for $4.8 billion in a deal that marks the Swiss pharma's first major move into gene therapy. The deal announced early Monday was the first major...
23:27 , Feb 13, 2019 |  BC Week In Review  |  Clinical News

uniQure gene therapy leads to sustained Factor IX activity in Phase IIb study

uniQure N.V. (NASDAQ:QURE) said Feb. 8 new data showed sustained Factor IX activity in a Phase IIb study of gene therapy AMT-061 to treat hemophilia B. Separately, the company said Feb. 4 it had dosed...
18:36 , Feb 8, 2019 |  BC Extra  |  Clinical News

uniQure gets bump on latest hemophilia readout

uniQure N.V. (NASDAQ:QURE) added $3.31 (10%) to $36.49 Friday after new data showed sustained Factor IX activity in a Phase IIb study of gene therapy AMT-061 to treat hemophilia B. Separately, the company said Monday...
03:50 , Dec 1, 2018 |  BioCentury  |  Product Development

Cell therapy momentum at ASH

This year’s American Society of Hematology (ASH) meeting will showcase a surge in clinical CAR T cell presentations. But gene therapies are not following the same trajectory, despite both modalities being fresh off their first...