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Save One Life Inc. Welcomes Chris Bombardier as New Executive Director

January 16, 2019

The following is an excerpt from a press release from Save One Life Inc. Read the press release in its entirety here.聽 Save One Life Inc. announces a new executive director. Save One Life is an international nonprofit that assists people with hemophilia in developing countries. Christopher G. Bombardier, famous for being the first person […]

Plasma Protein Therapeutics Association Welcomes New President and CEO

January 16, 2019

  The following is an excerpt from the Plasma Protein Therapeutics Association. Read the full press release here. The Board of Directors of the Plasma Protein Therapeutics Association announced Amy Efantis will be its next President and Chief Executive Officer effective Jan. 16, 2019. Having served most recently as Vice President, Global Public Policy & […]

Genentech’s Hemlibra Provided Sustained Bleed Control in a Study of Children with Hemophilia A with Factor VIII Inhibitors

December 20, 2018

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra庐 prophylaxis in children younger than 12 years of age with hemophilia A with factor […]

Sangamo Announces Treatment Of First Patient In Clinical Trial Of In Vivo Genome Editing Therapy For Hemophilia B

December 20, 2018

The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Hemophilia B is a rare, genetic bleeding disorder caused by a lack of the factor IX (FIX) protein, which is necessary for normal blood clotting. Current hemophilia B treatments typically require frequent intravenous infusions of clotting […]

Genentech’s Hemlibra Provided Sustained Bleed Control in the Largest Pivotal Study in Children with Hemophilia A with Inhibitors

December 5, 2018

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group, announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra庐聽prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII […]

Bioverativ Data Presented at ASH Underscore Potential for Once Weekly Dosing with Sustained High Factor Levels in Hemophilia A

December 5, 2018

The following is an excerpt from a press release from Bioverativ. Read the press release in its entirety here. Bioverativ Inc.,聽a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented new data from the EXTEN-A Phase 1/2a trial of BIVV001 showing that a single 65 IU/kg dose of BIVV001 extended […]

uniQure Announces Long-Term Clinical Data from Ongoing Phases and Confirms Dose for Hemophilia B Study

December 5, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated results聽from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company鈥檚 next-generation gene therapy聽candidate […]

Update on uniQure’s Hemophilia B Gene Therapy Program

November 28, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure announces the initial topline data from our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. All patients achieved and sustained therapeutic […]

uniQure Highlights Pipeline Expansion and Advancements in Technology at Research and Development Day

November 28, 2018

The following is a press release from uniQure. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced the expansion of its research pipeline with novel AAV gene therapy approaches to treating Hemophilia A, Fabry disease and Spinocerebellar Ataxia Type 3 at the Company鈥檚 Research & Development Day held this […]

uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B

November 19, 2018

The following is an excerpt from a press release from uniQure. uniQure announced聽that they have treated the first patient in our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). 鈥淎s a one-time administered therapy, AMT-061 has the […]

Genentech to Present New Data at the American Society of Hematology 2018 Annual Meeting

November 7, 2018

  The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech has announced new data that will be presented at this year’s annual American Society of Hematology meeting held in San Diego from Dec. 1-4. Ten聽Genentech聽medicines will be featured in more than 70 abstracts, including […]

FDA Approves Genentech’s Hemlibra

October 4, 2018

The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration has approved Hemlibra庐(emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, […]

LA FDA APRUEBA HEMLIBRA聽DE GENENTECH

October 4, 2018

El siguiente es un comunicado de prensa proporcionado por Genentech.聽 LA FDA APRUEBA HEMLIBRA庐聽DE GENENTECH (EMICIZUMAB-KXWH) PARA LA HEMOFILIA A SIN INHIBIDORES DE FACTOR VIII 鈥 El primer medicamento que reduce considerablemente la hemorragia tratada en comparaci贸n con la previa profilaxis de factor VIII basada en una comparaci贸n intrapaciente 鈥 鈥 脷nico medicamento que puede […]

Genentech Announces the Launch of New Safety Website

September 3, 2018

Genentech has announced a new web portal for patients and caregivers intended to provide timely and accurate information on targeted serious adverse events of interest for HEMLIBRA. Visit the safety website:聽http://www.emipatientinfo.com/. Earlier this year, Genentech launched a similar site specifically for health care providers. In announcing the launch, Genentech stated 鈥淭his website is meant to […]

Phase III Results for Genentech’s Hemlibra Published in New England Journal of Medicine

August 31, 2018

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here.聽 Genentech, a member of the Roche Group, announced that pivotal data from the Phase III HAVEN 3 study, which evaluated HEMLIBRA庐聽prophylaxis administered every week or every two weeks in adults and adolescents aged 12 years or […]

Bayer Receives FDA Approval for Jivi, New Hemophilia A Treatment With Step-Wise Prophylaxis Dosing Regimen

August 31, 2018

The following is an excerpt from a press release from Bayer. Read the press release in its entirety here.聽 Bayer announced that the U.S. Food and Drug Administration has approved Jivi庐 for the routine prophylactic treatment of hemophilia A in previously-treated adults and adolescents 12 years of age or older. The initial recommended prophylactic regimen […]

uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B

August 24, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.聽 uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy […]

U.S. District Court in Delaware rules against Shire in preliminary injunction

August 8, 2018

On Aug. 7, 2018, the U.S. District Court in Delaware ruled against Shire in its request for a preliminary injunction against Roche AG from creating, transporting and marketing its recently-approved drug Hemlibra in the U.S., which received FDA approval to treat people with hemophilia A with inhibitors in 2017. This ruling means there are no […]

Spark Therapeutics investigational trail moves into Phase 3

August 7, 2018

The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here. Investigational SPK-8011 moving to Phase 3 As of the July 13 data cutoff, 12 participants in the Phase 1/2 trial have received a single administration of investigational SPK-8011, including two at a dose of 5×1011 […]

Catalyst Biosciences releases update on trial of Factor IX treatment of severe hemophilia B

June 19, 2018

The following is a summary of a press release from Catalyst Biosciences by Dr. Robert Sidonio, a trusted medical advisor. Read the full press release here. On June 18, Catalyst Biosciences provided an update of the progress from its Phase 陆 trial investigating the use of FIX variant product, called CB 2679d/ISU304, which is a […]

FDA GRANTS PRIORITY REVIEW TO GENENTECH鈥橲 HEMLIBRA FOR PEOPLE WITH HEMOPHILIA A WITHOUT FACTOR VIII INHIBITORS

June 5, 2018

The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced the U.S. Food and Drug Administration has accepted the company鈥檚 supplemental Biologics License Application and granted Priority Review for HEMLIBRA 聽for adults and children with hemophilia A without factor VIII […]

BioMarin Presented an Update on the Phase 1/2 Study of Valoctocogene Roxaparvovec at WFH’s 2018 World Congress in Scotland

May 22, 2018

The following is an excerpt from a press release from BioMarin. Read the entire press release here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The updated results were presented during […]

Novo Nordisk Announced New Data at WFH’s 2018 World Congress in Scotland

May 22, 2018

The following is an excerpt from a press release from Novo Nordisk. Read the full press release here. Adults with haemophilia B who received a single dose Refixia庐 (nonacog beta pegol; N9-GP) achieved greater total factor IX exposure than those treated with rFIXFc (recombinant factor IX-Fc fusion protein). The head-to-head paradigm7 trial also observed a […]

uniQure Presents New Data Demonstrating Clinical Benefits at American Society of Gene and Cell Therapy Annual Meeting in Chicago

May 22, 2018

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented data showing successful liver transduction with the AAV5 vector in both non-human primates and humans with pre-existing anti-AAV5 neutralizing antibodies (NABs). […]

Bioverativ Presents Preliminary Phase 1/2a Data at WFH’s 2018 World Congress in Scotland

May 22, 2018

The following is a press release from Bioverativ. Read the full press release here. Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented initial clinical data for BIVV001, a novel and investigational von Willebrand factor (VWF)-independent factor VIII therapy for people with hemophilia A. Preliminary safety and […]

Spark Therapeutics and Pfizer Announced Data at WFH’s 2018 World Congress in Scotland

May 22, 2018

The following is an excerpt from a press release from Spark Therapeutics. Read the full press release here. Spark Therapeutics, a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer, announced, with a cumulative follow-up of more than 18 patient years of observation, all 15 participants in the ongoing […]

Genentech Announced Full Results of Phase III Haven 3 Study at WFH’s 2018 World Congress in Scotland

May 21, 2018

The following is an excerpt from the press release from Genentech. Read the full press release here. Genentech, a member of the Roche Group, announced the full results from the Phase III HAVEN 3 study evaluating HEMLIBRA庐 (emicizumab-kxwh) prophylaxis administered every week or every two weeks in people with hemophilia A without factor VIII inhibitors […]

Genentech Releases Statement on Hemlibra Patient

April 24, 2018

The following is an excerpt from a statement from Genentech. Read the entire statement 聽HERE. _________________________________________________________ Genentech has recently learned that a patient in the Phase III HAVEN 2 clinical trial developed a neutralizing anti-drug聽antibody. For聽this patient, the anti-drug antibody resulted in reduced efficacy of HEMLIBRA.

Bioverativ Highlights the Impact of Humanitarian Aid in Hemophilia

April 17, 2018

The following is an excerpt from a press release from Bioverativ. Read the entire press release here.聽 More than 15,000 people with hemophilia in 40 developing countries have already been treated following Bioverativ and Sobi鈥檚 unprecedented donation of factor therapy to the WFH Humanitarian Aid Program Bioverativ Inc., a Sanofi company, joins the global hemophilia […]

FDA Grants Breakthrough Therapy Designation for Genentech’s Hemlibra

April 17, 2018

The following is an excerpt from a press release from Genentech. Read the full press release here.聽 Genentech, a member of the Roche Group, announced today the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to HEMLIBRA聽for people with hemophilia A without factor VIII inhibitors. Breakthrough Therapy Designation is designed to accelerate the […]


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