23:34 , Jul 8, 2019 |  BC Extra  |  Clinical News

July 8 Clinical Quick Takes: Hemophilia data from Sangamo-Pfizer, Novo; plus GamaMabs, Xarelto and more

Sustained Factor VIII levels for Sangamo, Pfizer hemophilia A gene therapy  Sangamo Therapeutics Inc. (NASDAQ:SGMO) and partner Pfizer Inc. (NYSE:PFE) presented data at the International Society on Thrombosis and Haemostasis (ISTH) meeting in Melbourne showing...
22:59 , Jul 8, 2019 |  BC Extra  |  Company News

July 8 Company Quick Takes: Merck, Skyhawk in discovery deal; plus MorphoSys, BioMarin, Roche-Spark and Hanmi

Skyhawk, Merck in neurology, cancer deal  Skyhawk Therapeutics Inc. (Waltham, Mass.) and Merck & Co. Inc. (NYSE:MRK) parterned to discover, develop and commercialize small molecules capable of modulating RNA splicing discovered using Skyhawk’s SkySTAR platform...
21:07 , Jun 13, 2019 |  BC Innovations  |  Product Development

Why BioMarin took gene therapy manufacturing into its own hands

As gene therapy companies fight for space at contract manufacturers, BioMarin has leveraged its expertise in biologics to design its own capabilities for manufacturing gene therapies and gain control of the process -- a move...
00:05 , May 29, 2019 |  BC Extra  |  Clinical News

Longevity of hemophilia gene therapy in question as BioMarin seeks accelerated approval

As BioMarin reported interim Phase III data Tuesday that sets the company up for regulatory submissions, long-term Phase I/II data cast doubt on the durability of gene therapy valoctocogene roxaparvovec to treat severe hemophilia A....
20:22 , May 1, 2019 |  BC Extra  |  Preclinical News

May 1 Preclinical Quick Takes: MD Anderson, Sana, Sigilon

MD Anderson findings could make radiation possible for pancreatic cancer  Researchers at University of Texas MD Anderson Cancer Center reported findings suggesting that HIF-PH inhibition could help prevent radiation-induced GI injury, the main factor that...
22:52 , Apr 26, 2019 |  BC Extra  |  Company News

European regulatory roundup: CHMP opinions and a Portola approval

EMA's CHMP recommended a basket of approvals, including Dovato dolutegravir/lamivudine, Libtayo cemiplimab and two orphan therapies. Also Friday, the European Commission granted conditional approval to Ondexxya andexanet alfa from Portola Pharmaceuticals Inc. (NASDAQ:PTLA) to reverse...
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FDA draft guidance suggests comparing bispecifics to monospecific antibodies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...
19:34 , Apr 4, 2019 |  BC Extra  |  Financial News

Follow-on roundup: Sangamo, Tricida, DBV

Sangamo and Tricida each priced follow-ons soon after their share prices spiked on recent clinical readouts. DBV also raised a follow-on ahead of a planned BLA resubmission. Sangamo Therapeutics Inc. (NASDAQ:SGMO) raised $126.5 million on...
20:23 , Apr 2, 2019 |  BC Extra  |  Clinical News

Sangamo gains on Phase I/II readout for hemophilia A gene therapy

Interim Phase I/II data for Sangamo’s hemophilia A gene therapy Tuesday helped shares of the company complete a round-trip from its 31% tumble on a February readout for its mucopolysaccharidosis therapies. Sangamo Therapeutics Inc. (NASDAQ:SGMO)...
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...