Sanofi

Sanofi and partner Swedish Orphan Biovitrum shared positive topline results from their ongoing Phase III trial on a candidate drug for patients diagnosed with severe hemophilia A. 

Three companies – Intellia Therapeutics, Editas Medicine and uniQure – are each anticipating a bright 2022 as the promises of gene therapy continue to be realized.

BioMarin

BioMarin updated several of the company’s gene therapy programs on February 17 following a September 2021 update, when the drug manufacturer reported the U.S. Food and Drug Administration had placed a clinical hold on the BMN 307 Phearless Phase I/II trial.

BioMarin Pharmaceutical shared positive results from the company’s ongoing Phase III GENEr8-1 trial on the viability of valoctocogene roxaparvovec as a treatment for patients diagnosed with severe hemophilia A.

Genentech’s Hemlibra (emicizumab-kxwh) showed no new safety signals and continued to deliver consistent bleeding control in the final analysis of a long-term, late-stage hemophilia A study.

After touting positive Phase III results at the American Society of Hematology’s annual meeting, the U.S. FDA placed a clinical hold on uniQure”s hemophilia B treatment AMT-061 due to a patient developing liver cancer. 

Sanofi Hits the Brakes on Its Hemophilia Program Published: Nov. 6, 2020 By Mark Terry BioSpace   Three hemophilia organizations, the World Federation of Hemophilia, the European Haemophilia Consortium and […]

The U.S. Food and Drug Administration’s approval-review schedule for August includes the Biologics License Application for Bristol Myers Squibb and Juno Therapeutics’ lisocabtagene maraleucel (liso-cel) for relapsed or refractory large B-cell lymphoma after at least two previous therapies. 

London-based Freeline announced the closing of a $120 million extended Series C financing to advance the company’s gene therapy programs, and is considering additional capital fundraising options during 2020.

CSL Behring bolstered the company’s growing gene therapy portfolio with the acquisition of global rights to uniQure’s gene therapy program for hemophilia B that has a price tag of up to $2 billion.