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BioMarin’s Investigational Gene Therapy for Hemophilia A Maintains Average Factor VIII Levels within Normal Range for over One Year

July 11, 2017

Note: The following is an edited version of a press release from BioMarin.聽Read the press release from BioMarin聽in it鈥檚 entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The updated results were聽presented […]

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

July 11, 2017

Note: The following is an edited version of a press release from uniQure.聽Read the press release from uniQure聽in it鈥檚 entirety here. uniQure N.V.聽has聽announced updated results聽from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy聽in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose […]

Bioverativ Files Complaints Against CSL Behring’s Idelvion

July 10, 2017

Note: This story has been sourced from the website, www.thepharmaletter.com聽and The Australian Business Review. On Friday, July 7, 2017, Bioverativ filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon […]

Alnylam & Sanofi Genzyme Report Positive Results from Ongoing Phase 2 Study with Investigational RNAi Therapeutic Fitusiran in Patients with Hemophilia A and B With or Without Inhibitors

July 10, 2017

Note: The following is an edited version of a press release from Alnylam.聽Read the press release from Alnylam聽in it鈥檚 entirety聽here. Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme,聽announced today new positive results from the ongoing Phase 2 open-label extension study with fitusiran in patients with hemophilia A and B, with or without inhibitors.聽These results were presented today […]

Shire Gets Injunction Against Roche Over Emicizumab

July 10, 2017

Note: The following is an excerpt from a Reuters news article. Read the article in full here. Pharmaceutical group Shire said on Sunday it had obtained a preliminary injunction in a Hamburg court against rival Roche聽over its hemophilia drug emicizumab, alleging incomplete and misleading statements surrounding the treatment. Roche said in an emailed statement it […]

Genentech Announces Positive Phase III Results for Emicizumab at ISTH 2017

July 10, 2017

Note: The following is an edited version of a press release from Genentech.聽Read the press release from Genentech聽in it鈥檚 entirety聽here. Genentech, a member of the Roche Group, announced today that data from HAVEN 1, a Phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with hemophilia A with inhibitors, were published […]

Shire Submits Investigational New Drug Application to FDA for Gene Therapy candidate SHP654 for treatment of Hemophilia A

July 7, 2017

Note: The following is an edited version of a press release from Shire.聽Read the press release from Shire聽in it鈥檚 entirety聽here. Shire has announced聽the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII (FVIII) gene therapy for the […]

New Biopharmaceutical Company, Sigilon Therapeutics, Launches

July 7, 2017

Note: The following is an edited version of a press release from Sigilon聽Therapeutics.聽Read the press release from Sigilon聽Therapeutics聽in it鈥檚 entirety聽here. Flagship Pioneering, a fully-integrated life science innovation聽enterprise, recently聽announced the launch of Sigilon Therapeutics Inc. Sigilon Therapeutics is a聽biopharmaceutical company that discovers and develops category-defining biocompatible encapsulated聽cell therapies. Sigilon Therapeutics鈥 discovery platform combines cell engineering and […]

Genentech Announces Results in Emicizumab Phase III Studies (HAVEN 1 And HAVEN 2) in Hemophilia A With Inhibitors

June 26, 2017

Genentech, a member of the Roche Group announced today positive data from the primary analysis of the Phase III HAVEN 1 study in adults and adolescents and interim analysis of the Phase III HAVEN 2 study in children evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) for the treatment of hemophilia A with inhibitors to factor VIII. […]

FDA Accepts Bioverativ’s Application for Investigational Hemophilia A Drug

June 12, 2017

Bioverativ announced on June 12, 2017 that the U.S. Food and Drug Administration (FDA) has accepted the company鈥檚 Investigational New Drug (IND) application for BIVV001 (also known as rFVIIIFc-VWF-XTEN), a novel, investigational factor VIII therapy designed to potentially extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. […]

FDA Approves New Novo Nordisk Treatment for Patients with Hemophilia B

June 1, 2017

  Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application for REBINYN庐 (Coagulation Factor IX (Recombinant), GlycoPEGylated) for the treatment of adults and children with hemophilia B.聽Novo Nordisk expects to launch REBINYN庐 in the U.S. in the first half of 2018. For more info on REBINYN, […]

Dimension Therapeutics Discontinues DTX101, a Investigational Gene Therapy Product

May 12, 2017

Dimension Therapeutics聽announced it will聽discontinue the development of DTX101, an investigational AAVrh10-based gene therapy product in development for the treatment of moderate/severe-to-severe hemophilia B. The decision followed the review of the emerging DTX101 Phase 1/2 clinical study data, including the data as of the beginning of聽May 2017, and the observation that the data would not meet […]

Sangamo Therapeutics And Pfizer Announce Collaboration For Hemophilia A Gene Therapy

May 12, 2017

Sangamo Therapeutics, Inc. and Pfizer Inc. recently聽announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter. Click here to read the full press release from Sangamo Therapeutics.

uniQure Announces Successful Gene Therapy Delivery in Animal Model

May 12, 2017

uniQure recently presented data at the American Society of Gene and Cell Therapy (ASGCT) 20th聽Annual Meeting in Washington, D.C.聽on successful repeated hepatic gene delivery with their聽AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). The results described successful sequential transduction with the two reporter transgenes and highlighted the progress made in readministration protocols […]

Kedrion Announces Return to Normal Supply Levels of Ko膩te

May 12, 2017

Kedrion Biopharma announced a return to normal supply levels of Ko膩te in the 1000 vial size and a series of large-scale improvements to their聽overall聽operations following a temporary period of limited availability of product. Click here to read the full contents of聽letter provided by Kedrion Biopharma regarding this matter.  

uniQure Receives EU PRIME Designation For Hemophilia B Product

May 2, 2017

聽uniQure recently received the designation of a “priority medicine” from the European Union for a potential gene therapy treatment for Hemophilia B. From the press release: uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy聽in patients with severe […]

Pharmaceutical Companies Recognize World Hemophilia Day

April 17, 2017

Today, on World Hemophilia Day, many pharmaceutical companies joined in the celebration by recognizing and supporting the hemophilia community. Linked below are statements from a wide group of companies on today’s celebration of World Hemophilia Day: Grifols Press Release Bioverativ Press Release Novo Nordisk on supporting women with bleeding disorders Shire Press Release CSL Behring […]

Novo Nordisk Recognizes World Hemophilia Day

April 17, 2017

Drug manufacturer, Novo Nordisk recognized World Hemophilia Day 2017 by celebrating the theme of the year; Hear Their Voices, by showing support for women with bleeding disorders. To learn more, read Novo Nordisk’s press release in it’s entirety.聽here.

Shire Recognizes World Hemophilia Day

April 17, 2017

Clotting factor manufacturer, Shire, is celebrating World Hemophilia Day in part by hosting an awareness rally in Chicago, Illinois. The rally is aimed mainly to educate the public on the dramatic estimate of a hemophilia bleeding episode occurring worldwide every 3-15 seconds. See more on their website. Read a full statement from聽Shire recognizing World Hemophilia […]

Grifols Donates 140 Million Units of Factor on World Hemophilia Day

April 17, 2017

As part of聽World Hemophilia Day celebrations, Grifols has announced a large donation of clotting factor to the World Federation of Hemophilia (WFH) Humanitarian Aid Program.聽This announcement is a continuation of the company鈥檚 three-year commitment from 2014, bringing the total humanitarian aid commitment to more than 200M IU of Factor VIII over eight years Read the […]

Catalyst Biosciences Completes New Hemophilia B Drug Toxicology Study

April 14, 2017

On April 11, Catalyst Biosciences, a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, reached a key milestone towards starting human trials with聽completion of the CB 2679d/ISU304 toxicology studies. Their press release can be read in full聽here.

Aptevo’s Ixinity Found To Be Safe In Children Under 12 With Hemophilia B

April 14, 2017

Aptevo’s Ixinity, previously approved for use in people over the age of 12, just received approval for use in children under 12, per their ongoing phase 3 trial. Read the full press release here.

Spark Therapeutics Presents Updated Preliminary Data from Hemophilia B Phase 1/2 Trial

April 14, 2017

Spark Therapeutics issued a press release on updated preliminary data from 10 infused participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for hemophilia B. The data is聽being presented at Hemostastis &聽Thrombosis Research Society (HTRS) this week. The press release can be read in full here.

Spark Therapeutics Announces Phase 1/2 Gene Therapy Trial for Hemophilia A

February 23, 2017

Note: The following is an edited version of a press release from Spark Therapeutics.聽Read the press release from Spark Therapeutics聽in it鈥檚 entirety聽here. Spark Therapeutics has announced that its Phase 1/2 trial for the investigation of gene therapy for hemophilia A (factor聽VIII deficiency) has begun. This trial will initially be enrolling participants at the Children鈥檚 Hospital […]

Bioverativ Officially Spins-Off of Biogen鈥檚 Hemophilia Business

February 1, 2017

Note: The following is an edited version of a press release from Bioverativ.聽Read the press release from聽Bioverativ聽in it’s entirety聽here. Today, February 1, 2017, Bioverativ聽launched as an independent, global biotechnology company focused on the discovery, development, and commercialization of innovative therapies for hemophilia and other rare blood disorders. Bioverativ is formed by a spin-off of Biogen鈥檚 […]

UniQure Receives FDA Breakthrough Designation of AMT-060 Investigational Gene Therapy

January 30, 2017

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety聽here. UniQure announced that AMT-060, 聽its proprietary, investigational gene therapy聽in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).聽 This designation is based on results from the […]

Shire’s Adynovate Receives Two New FDA Approvals for Treatment of Hemophilia A

December 29, 2016

Note: The following is edited from a press release from Shire. Read the full press release in it鈥檚 entirety聽here. Shire plc recently announced that the U.S. Food and Drug Administration (FDA) has approved Adynovate, an extended circulating half-life recombinant Factor VIII treatment for hemophilia A, in pediatric patients under 12 years of age. The FDA […]

Genentech Meets Primary Endpoint for Phase III Emicizumab Study

December 22, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Genentech. Read the full press release in it鈥檚 entirety聽here. Genentech, a member of the Roche Group, announced that the primary endpoint has been met for the Phase III HAVEN 1 study evaluating emicizumab prophylaxis in people 12 years of age or older with hemophilia […]

UniQure Announces New Data in Phase I/II Clinical Trial

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting聽in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure鈥檚 proprietary, investigational gene therapy聽for patients with severe […]

Sangamo Biosciences Announces Plans to File for New Investigational Drug

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Sangamo Biosciences. Read the full press release in its entirety here. Sangamo Biosciences presented data during the recent American Society of Hematology (ASH) Annual Meeting regarding preclinical and manufacturing data of SB-525, its gene therapy program for hemophilia A. “We have developed an improved […]


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