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Breakthrough in Hemophilia Gene Therapy

January 31, 2018

The following is an excerpt from an article in Medical News Bulletin. Read the full article here. Researchers at Spark Therapeutics and Pfizer tested the safety and efficacy of a new hemophilia gene therapy vector for the treatment of hemophilia B. The body’s ability to stop bleeding and repair damaged blood vessels depends heavily upon […]

uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study

October 19, 2017

Note: The followed is excerpted from a press release from uniQure. Read uniQure’s full press release HERE.  uniQure announced today plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their product candidate called AMT-061. AMT-061 is identical to AMT-060 in every way apart from a change from […]

Spark Therapeutics Releases Preliminary Data for SPK-8011

August 9, 2017

Note: The following is an edited version of a press release from Spark Therapeutics. Read the press release from Spark Therapeutics in it’s entirety here. The company has disclosed preliminary initial data from the Phase 1/2 dose-escalation clinical trial for SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the Spark200 capsid and containing a codon-optimized human factor […]

Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent & Sustained Response at ISTH 2017

July 11, 2017

Note: The following is an edited version of a press release from Spark Therapeutics. Read the press release from Spark Therapeutics in it’s entirety here. Spark Therapeutics recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of SPK-9001 for hemophilia B, as of the June 5, 2017 data cut off, had their AIR reduced approximately […]

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

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. Read the press release from Shire in it’s entirety here. Shire has announced 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 […]

Dimension Therapeutics Discontinues DTX101, a Investigational Gene Therapy Product

May 12, 2017

Dimension Therapeutics announced it will 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 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 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 Annual Meeting in Washington, D.C. on successful repeated hepatic gene delivery with their 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 […]

uniQure Receives EU PRIME Designation For Hemophilia B Product

May 2, 2017

 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 in patients with severe […]

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. Read the press release from Spark Therapeutics in it’s entirety here. Spark Therapeutics has announced that its Phase 1/2 trial for the investigation of gene therapy for hemophilia A (factor VIII deficiency) has begun. This trial will initially be enrolling participants at the Children’s Hospital […]

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’s entirety here. UniQure announced that AMT-060,  its proprietary, investigational gene therapy in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).  This designation is based on results from the […]

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’s entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure’s proprietary, investigational gene therapy 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 […]

Spark Therapeutics Shares Updated Phase I/II Clinical Trial Data

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in it’s entirety here.. Spark Therapeutics and Pfizer announced updated results from the Phase 1/2 study of SPK-9001, an investigational recombinant gene transfer product in hemophilia B, at the 58th American Society of Hematology (ASH) Annual Meeting, […]

Sangamo BioSciences Receives Orphan Drug Designation From The FDA For SB-FIX

September 9, 2016

The following is excerpted from a press release from PRNewswire. Read the press release in it’s entirety here. Sangamo BioSciences, Inc. announced earlier this week that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-FIX, the company’s zinc finger nuclease (ZFN)-mediated genome editing product candidate for the treatment of hemophilia B. Sangamo […]

BioMarin Releases Results of Phase 1/2 Study of Gene Therapy Treatment

July 28, 2016

Note: The below is an edited version of a press release issued by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here. BioMarin Pharmaceutical announced today positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. Nine patients with severe hemophilia […]

Sangamo BioSciences Announces New Gene Therapy Clinical Development Program

July 28, 2016

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in its entirety here. Sangamo BioSciences, Inc. announced the presentation of preclinical data that supports the clinical development of its new proprietary gene therapy for the treatment of hemophilia A. This new therapeutic comprises an adeno-associated […]

uniQure Presents Updated Clinical Data in Patients with Severe Hemophilia B

July 28, 2016

Note: The below is an edited version of an article originally published on Globe Newswire. The original release can be read in its entirety here. uniQure N.V. yesterday announced updated clinical data from its ongoing Phase I/II trial of AMT-060, its proprietary, investigational gene therapy, in patients with severe hemophilia B.  The updated data show that […]

FDA Gives Orphan Drug Designation for Hemophilia A Gene Therapy

March 1, 2016

Note: The below is an edited version of a press release by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here. BioMarin Pharmaceutical Inc. announced on Tuesday, March 1, that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by […]

New Gene Therapy Treatment Proves Effective In Dogs

February 2, 2016

Note: The following is an edited form of a press release from the University of North Carolina School of Medicine. The original release can be read here. A single injection. That’s all someone with a factor VII deficiency would need for a life-long cure, thanks to a new gene therapy treatment developed in a collaboration of […]

Infusing Love: A Long Lasting Opportunity

October 22, 2015

In our family, we are open to participating in research, thinking, “if it can help our kids or help a future family or patient then we are all for it.” So when the opportunity arose to be enrolled in a trail to test long lasting factor, we jumped on it. We did our homework and […]

Five New Developments in Hemophilia

April 7, 2015

This post first appeared on Vector, a blog of Boston Children’s Hospital an was written by Ellis Neufeld, MD, PhD, a hematologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.   ________________________________________________ From new longer-acting drugs to promising gene therapy trials, much is changing in the treatment of hemophilia, the inherited bleeding disorder in which the blood does not clot. […]

New Gene Therapy For Hemophilia Shows Potential As Safe Treatment

March 12, 2015

Research showed that bleeding events were drastically decreased in animals with Hemophilia B. Using a viral vector to swap out faulty genes proved safe and could be used for the more common Hemophilia A. _______________________________ A multi-year, ongoing study suggests that a new kind of gene therapy for Hemophilia B could be safe and effective […]

Biogen Idec Forms Partnership to Focus on Gene Therapy for Hemophilia

January 30, 2015

Biogen Idec, Fondazione Telethon, and Ospedale San Raffaele have entered into a worldwide collaboration to jointly develop gene therapies for the treatment of both hemophilia A and B. The agreement will combine San Raffaele – Telethon Institute for Gene Therapy’s (TIGET) extensive expertise in creating new gene therapy strategies and developing them from the bench to bedside […]

Pfizer Establishes Gene Therapy Partnership

December 8, 2014

Pfizer Inc. announced on Monday, December 8, 2014  two strategic decisions to expand the company’s rare disease research and development activities through the establishment of a gene therapy platform to investigate potential treatments for patients. First is an agreement with Spark Therapeutics to develop SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential […]

Promising Gene Therapy Results in Treating Hemophilia B

November 20, 2014

On November 19, St. Jude Children’s Research Hospital announced the results of their ongoing gene therapy partnership with University College London and the Royal Free Hospital. The report indicates success in the trial, which used a modified adeno associated virus (AAV) 8 to deliver genetic material for making Factor IX in Hemophilia B patients. In […]

Stanford University: New Form of Gene Therapy Could Cure Hemophilia in Mice

October 30, 2014

[glossary_exclude] A technique developed by Stanford researchers could provide a safer, longer-lasting method of replacing faulty, disease-causing genes with working copies. The ability to pop a working copy of a faulty gene into a patient’s genome is a tantalizing goal for many clinicians treating genetic diseases. Now, researchers at the Stanford University School of Medicine […]

Bayer to Develop Hemophilia Gene Therapy Treatment

June 23, 2014

Dimension Therapeutics, a Cambridge biotechnology startup, has struck a deal worth up to $252 million with the pharmaceutical giant Bayer HealthCare to develop a gene therapy to treat hemophilia, a rare disease that prevents blood from clotting. The goal of the prospective treatment, which is not yet in clinical trials, is to repair a faulty […]


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