BioCentury | Jun 27, 2020

Who’ll buy uniQure now?

UniQure’s out-licensing deal for its lead gene therapy program in hemophilia B may have disappointed investors, but that does not preclude the Dutch company from remaining an attractive takeout target. Investors immediately soured on Wednesday’s...
BioCentury | Jun 26, 2020
Product Development

Data Bytes: Comparing Factor VIII levels of hemophilia A gene therapies

Hemophilia A is expected to be the next market where gene therapies will create competition, but long-term durability remains a key unknown. While the BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) therapy valoctocogene roxaparvovec, or “valrox,” maintains Factor...
BioCentury | Jun 22, 2020

First-day pop gives SinocellTech massive market cap on Shanghai’s STAR

With a first-day stock move that nearly tripled its share price, SinocellTech became the highest-valued biotech traded on Shanghai’s STAR market since the launch of its biotech chapter last year. SinocellTech Ltd. (Shanghai:688520) climbed 185%...
BioCentury | Mar 17, 2020

Sigilon nears first clinical test of cell shielding tech with $80M series B

With its $80.3 million series B round, Sigilon is planning to bring the first of its shielded cell therapies into the clinic, while also advancing toward INDs on four other programs. Canada Pension Plan Investment...
BioCentury | Feb 21, 2020
Product Development

Feb. 20 Product Development Quick Takes: Priority Review puts BioMarin on track to score first hemophilia gene therapy approval in U.S.; plus Gilead-CDC, Genfit

BioMarin's hemophilia gene therapy gets Priority Review  BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) could become the first company to have a hemophilia gene therapy approved in the U.S. FDA accepted and granted Priority Review to a BLA...
BC Extra | Dec 24, 2019
Company News

Regulatory roundup: BioMarin poised to have first U.S., EU hemophilia gene therapy; plus Allergan, Eisai, FibroGen, ViiV and Vero

Monday's basket of regulatory news includes two landmark regulatory applications for BioMarin's hemophilia gene therapy in the U.S. and EU, and FDA approval of Allergan's migraine drug. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) submitted a BLA to...
BC Innovations | Dec 6, 2019
Targets & Mechanisms

Bispecifics and allogeneics steal the spotlight from autologous CAR Ts

Standing out at this year's ASH meeting is a pair of growing threats to first-generation CAR T cell therapies: bispecific antibodies and allogeneic cell therapies. Gone are the days when autologous CAR Ts were the...
BC Innovations | Oct 11, 2019
Targets & Mechanisms

CAR Ts can move well beyond cancer, but will need to go allogeneic first

Preclinical studies showing the utility of CAR T cells outside of cancer are ramping up, and developers have taken notice. But turning the modality into a general-purpose tool will require companies to break through safety...
BC Extra | Oct 10, 2019
Company News

bluebird tech to jump-start Novo in gene editing for hemophilia

A deal with bluebird bio is the latest move in Novo Nordisk’s push to remake its R&D, this time by partnering to develop a gene therapy for hemophilia A. The deal comes as sales for...
BC Extra | Sep 30, 2019
Company News

Sobi adding to U.S. presence, hematology pipeline via $915M Dova takeout

With the $915 million takeout of Dova Pharmaceuticals and a co-development deal with Sanofi in hemophilia, Sobi is continuing its push into the U.S. and strengthening its presence in hematology. Swedish Orphan Biovitrum AB (SSE:SOBI)...
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