NHLBI Grant Supports Scientific Research to Enhance Hemophilia A Gene Therapies
NHLBI Grant Supports Scientific Research to Enhance Hemophilia A Gene Therapies
The new grant will fund basic and translational studies to improve gene therapies for hemophilia A.
Navigating Clinical Trials
Clinical trials are key to advancing safe and effective treatments for bleeding disorders. They are an important step in the FDA drug-approval process and provide researchers with invaluable data about a drug’s effectiveness and side effects.
The Basics of Innovative Therapies: What You Need to Know
We continue to see significant research and innovation in the treatment of Hemophilia. It is becoming more important than ever to stay informed on all the advancements and innovation – what they are, how they work in the body and how safe they are. Understanding all the options including currently approved treatments, the pros and cons and the promise and limitations of each type of therapy will enable community members to have informative conversations with their providers and to ultimately make the best decision on treatment. This presentation is designed to provide a basic overview
Gene Therapy Basics for Hemophilia: What You Need to Know
Recently there has been an explosion of innovative research in the treatment of Hemophilia utilizing gene therapy technology. This workshop will help you develop a basic understanding of the complexities of gene therapy including terminology, how gene therapy works, and identifying the potential, limitations, risks and unknowns associated with gene therapy. We also provide a brief introduction to other novel therapies and an overview of what treatments are currently approved.
New Medscape Activity Seeks to Prepare Clinicians for Gene Therapy
New Medscape Activity Seeks to Prepare Clinicians for Gene Therapy
This program is intended for an international audience of hematologists/oncologists, pediatricians, hematology/oncology nurses, nurse practitioners, and physician assistants.
BioMarin Provides Update Including Next Steps for Investigational Gene Therapy
BioMarin Provides Update Including Next Steps for Investigational Gene Therapy
Valoctocogene roxaparvovec gene therapy is being investigated for safety and efficacy in adults with severe hemophilia A via multiple clinical trials.
Sigilon Announces a Priority Shift in Light of Clinical Trial Setbacks
Sigilon Announces a Priority Shift in Light of Clinical Trial Setbacks
The phase 1/2 trial of SIG-001 for hemophilia A has been on an FDA clinical hold since July 2021.
uniQure and CSL Behring Announce Trial Updates for Hemophilia B Gene Therapy
uniQure and CSL Behring Announce Trial Updates for Hemophilia B Gene Therapy
The HOPE-B trial is investigating etranacogene dezaparvovec in patients with severe and moderately severe hemophilia B.
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
The FDA placed a clinical hold on the company's investigational therapy in July of 2021.
Gene Therapy: A Candid Conversation
The realization that gene therapy could be just around the corner is both daunting and exciting. Join us for a balanced, patient-centric discussion on gene therapy from multiple perspectives: a provider, a patient in a gene therapy clinical trial, a patient on a newer therapy, and a patient who is satisfied with conventional factor replacement therapy.
Gene Therapy: Getting Up to Speed
Advancements in gene therapy are happening at a fast clip. Having a solid understanding of this technology is important to patients, caregivers and families. Join us for an in-depth discussion of gene therapy including historical aspects, key terms, various approaches to the technology, and how it may revolutionize hemophilia treatment.
What Is Gene Therapy?
This introduction to gene therapy explains how genes work, and how gene therapy can be used to create new therapies for hemophilia.
Spark Announces Multiyear Data Update for Investigational Gene Therapy
Spark Announces Multiyear Data Update for Investigational Gene Therapy
This data was drawn from a phase 1/2 clinical trial of SPK-8011, Spark's investigational gene therapy for hemophilia A.
FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
Sangamo responds to a clinical hold.
Vector Biology and Hemophilia Gene Therapy the Focus of Review
Vector Biology and Hemophilia Gene Therapy the Focus of Review
This review provides a comprehensive overview of the scientific underpinnings of several hemophilia gene therapy platforms, with particular focus on the delivery of genetic payloads at the heart of these technologies.
Spark Provides Trial Updates for Investigational Gene Therapy SPK-8011
Spark Provides Trial Updates for Investigational Gene Therapy SPK-8011
SPK-8011 is Spark Therapeutics' investigational gene therapy candidate for hemophilia A.
Review Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy
Review Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy
In this review, the authors discuss some of the primary considerations relevant to investigational gene therapies that employ adeno-associated viral
New Article Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy
New Article Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy
In this review, the authors discuss some of the primary considerations relevant to investigational hemophilia gene therapies that employ adeno-associated viral (AAV) vectors.
BioMarin Presents Positive Safety and Efficacy Data for their Hemophilia A Gene Therapy
BioMarin Presents Positive Safety and Efficacy Data for their Hemophilia A Gene Therapy
These data were presented by BioMarin at the American Society of Gene & Cell Therapy Virtual Meeting, which was held May 11-14, 2021.
Gene Therapy Trial Data Suggests Efficacy in the Presence of Antibodies
Gene Therapy Trial Data Suggests Efficacy in the Presence of Antibodies
The data were summarized here is from a phase III clinical trial of etranacogene dezaparvovec, an investigational hemophilai B gene therapy candidate.
New Inhibitor Study Using MLOF Research Repository Data Published
New Inhibitor Study Using MLOF Research Repository Data Published
The study represents a significant step toward better understanding of inhibitors and improving clinical care.
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
The commercialization and licensing agreement between the two companies went into effect on May 6th.
April 2021 Webinar Webinar Lineup
April 2021 Webinar Webinar Lineup
NHF's Wednesday Webinars for April 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.
April Webinars
All webinars are from 2:00pm-3:00pm ET.
uniQure Announces Findings from HCC Case Investigation
uniQure Announces Findings from HCC Case Investigation
The focus of the HOPE-B trial is etranacogene dezaparvovec, uniQure's investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B.
Update to Hemlibra® (emicizumab-kxwh) USPI
Update to Hemlibra® (emicizumab-kxwh) USPI
In Genentech’s ongoing efforts to transparently communicate with the hemophilia community, we are sharing two updates to the Hemlibra label that were requested by the FDA.
Spark Announces Preliminary Data from Investigational Gene Therapy Trial
Spark Announces Preliminary Data from Investigational Gene Therapy Trial
The first part of this phase 1/2 trial is designed to evaluate the safety and efficacy of SPK-8016 in adult males with clinically severe hemophilia A and no measurable inhibitor against FVIII.
Octapharma Announces Final Results of Nuwiq® Study
Octapharma Announces Final Results of Nuwiq® Study
Nuwiq® is indicated for on-demand treatment and control of bleeding episodes and routine prophylaxis to reduce the frequency of bleeding episodes and perioperative management of bleeding.
Advancements in Treatment for Hemophilia: What You Need to Know
These are exciting times in hemophilia research, with many new treatment options on the horizon. Come learn about the latest results of various phases of clinical trials on gene therapy and other new treatment options to see what the future holds for the treatment of hemophilia.
BioMarin Announces Positive Phase 3 Study Data for Valoctocogene Roxaparvovec
BioMarin Announces Positive Phase 3 Study Data for Valoctocogene Roxaparvovec
The phase 3 trial currently includes 134 participants with hemophilia A, all of whom have received a single dose of the gene therapy.
Medscape Activity Designed to Prepare Clinicians for Arrival of Gene Therapy
Medscape Activity Designed to Prepare Clinicians for Arrival of Gene Therapy
The goal of this activity is to prepare healthcare providers for real-world considerations with regard to successfully incorporating gene therapy into clinical practice.
Public Health Pre Con: Future Therapies and Surveillance for Blood Disorders
NOT AVAILABLE FOR CREDIT
Management of Patients on Novel Therapies
*ANCC
Moderators:
Penny Kumpf, RN-BC, BSN
Von Willebrand Outreach Nurse, Hemophilia of Georgia
Kelly Tickle, MSN, APN, PPCNP-BC<
Pediatric Nurse Practitioner, Children’s Healthcare of Atlanta
Speakers:
Lydia Johnson, RN, BSN
Hemostasis Clinical Nurse Coordinator
SSM Cardinal Glennon Children’s Medical Center
Saint Louis Univ. Dept of Pediatrics
Walking a New Pathway: Coagulation & Measures of Hemostasis
*ANCC
Moderator:
Kerry Hansen, RN
Nurse Clinician
University of Minnesota Health, Center for Bleeding and Clotting Disorders
Speaker:
Jim Munn, MS, BS, BSN, RN-BC
Program Nurse Coordinator
Hemophilia and Coagulation Disorders Center, University of Michigan
By the end of this session, the participant should be better able to:
Forging a New Path – Gene Therapy Readiness and HTC Implementation
*CME/ACPE
Moderator/Speaker:
Steven W. Pipe, MD, Faculty Chair
Professor of Pediatrics and Pathology
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases,
Pediatric Medical Director, Hemophilia and Coagulation Disorders Program,
Director, Special Coagulation Laboratory,
University of Michigan
Ann Arbor, Michigan
Speakers:
The Next Wave: Clinical Updates on New and Emerging Treatment Options in Hemophilia (1)
*CME/ACPE
Moderator:
Stacy E. Croteau, MD, MMS
Assistant Professor of Pediatrics, Harvard Medical School Director, Hemophilia/VWD Program, Boston Children's Hospital Medical Director, Boston Hemophilia Center
Dana-Farber/Boston Children's Cancer Center & Blood Disorders Center
Boston, MA
Speakers:
The Next Wave: Clinical Updates on New and Emerging Treatment Options in Hemophilia (2)
*CME/ACPE
Moderator:
Christopher E. Walsh, MD, PhD
Associate Professor of Medicine, Division of Hematology and Medical Oncology
Icahn School of Medicine at Mount Sinai
Director, Hemophilia Treatment Center, Mount Sinai Hospital
New York, NY
Speakers:
Resource Guide
Downloadable resources about gene therapy and innovative therapies for hemophilia and other bleeding disorders.
Glossary
A glossary of terms that are commonly used when discussing gene therapy for hemophilia.
Frequently Asked Questions
Answers to the most common questions asked about gene therapy for hemophilia.
Future Therapies
Researchers are investigating new and innovative ways to prevent bleeds in people with bleeding disorders.
Gene and Innovative Therapies Education
We are committed to ensuring people with bleeding disorders understand how gene and innovative therapies work.
BioMarin Announces Three Year Updates on Investigational Gene Therapy
BioMarin Announces Three Year Updates on Investigational Gene Therapy
On December 23rd, the BioMarin submitted a Biologics License Application for valoctocogene roxaparvovec to the U.S. Food and Drug Administration.
Shire Submits IND Application for Hemophilia A Gene Therapy Candidate
Shire Submits IND Application for Hemophilia A Gene Therapy Candidate
The therapy aims to prompt the sustained production of FVIII.