News
Learn More About Gene Therapy
Gene Therapy, Health and Well Being, Industry News & Research
A comprehensive document identifies 50 various publications about gene therapy, much of it accessible for parents and patients. The list includes: FAQs,…
Gene Therapy’s Slow Start
Gene Therapy, Health and Well Being, Industry News & Research
Multimillion-dollar gene therapies were long awaited and desired, but patients were unsure whether insurance would cover the costs. So far, very few patients are…
New Resource Compilation on Gene Therapy for Hemophilia
Gene Therapy, Industry News & Research
Learn about Gene Therapy for Hemophilia with NBDF’s new resource collection. Find answers, research, and support. The National Bleeding Disorders Foundation (NBDF) is…
BioMarin to Narrow Its Commercial Focus for Hemophilia A Gene Therapy
Gene Therapy, Industry News & Research
BioMarin recently announced that they will be changing the scope of their efforts to manufacture and market their gene therapy product ROCTAVIAN.™…
France Gets Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
by: Patricia Inácio, PhD | July 9, 2024 Hemgenix, the first approved hemophilia B gene therapy, was administered to two patients in France, marking the first time…
Defining Gene Therapy for Patients
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Gene therapy is a welcome treatment for patients, which has been anticipated for decades. But how do parents and patients discuss this…
Gene Therapy Candidates Face ‘Once in a Lifetime’ Choice
Gene Therapy, Health and Well Being, Living with a Bleeding Disorder
People with hemophilia considering gene therapy are wrestling with concerns including the cost of treatment and variations in results. You only get…
First Sickle Cell Patient to Receive Gene Therapy Featured in NYT
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Sickle cell disease is linked to an abnormality in the hemoglobin protein, which normally helps carry oxygen throughout the body via red…
FDA Approves Second Hemophilia B Gene Therapy Product
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
The U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (Pfizer, Inc.), a new hemophilia B gene therapy product. It is designed with…
Green Bay Resident Became First Wisconsinite to Receive Gene Therapy Treatment for Hemophilia A
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
A Green Bay resident became the first Wisconsinite to receive a gene therapy treatment for hemophilia A since it was approved by…
More Gene Therapy Trials from New Players
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
ReciBioPharm and GeneVentiv Therapeutics are developing GENV-HEM, a gene therapy for hemophilia patients, including those with inhibitors. ReciBioPharm will help GeneVentiv take GENV-HEM from preclinical development to Phase 1/2…
Does Your HTC Administer Gene Therapy?
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Not every HTC administers gene therapy. CSL Behring has updated their website so that patients can locate HTC’s that have its gene…
Answers to Common Questions Asked about Gene Therapy for Hemophilia
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Did you know? Gene therapy is the use of genetic material to treat or prevent disease. Research into gene therapy for hemophilia…
Countries Across the Globe Join the Gene Therapy Race
Gene Therapy, Health and Well Being, Living with a Bleeding Disorder
Canada Considers Funding Patient Gene Therapy; India Conducts First Human Clinical Trials for Hemophilia A An excellent article explains how Canada’s government…
Gene Therapy Struggles in Market
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Although it was eagerly anticipated, commercial sales of gene therapy are disappointing. BioMarin’s hemophilia treatment Roctavian had only one US patient last year….
Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial
Gene Therapy, Industry News & Research
Roctavian™ was approved in the summer of 2023 for the treatment of adults with severe hemophilia. The first hemophilia A gene therapy…
First Patient Dosed with Hemophilia Gene Therapy Candidate ASC618
Gene Therapy, Industry News & Research
The first patient has been dosed in ASC Therapeutics clinical trial of ASC618, the company’s investigational AAV8 vector-based gene therapy for patients…
Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Two new documents have been issued by the NBDF’s Medical and Scientific Advisory Council (MASAC), which periodically writes recommendations and advisories on…
Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations
Gene Therapy, Industry News & Research
Two new documents have been issued by the NBDF’s Medical and Scientific Advisory Council (MASAC), which periodically writes recommendations and advisories on…
Statement: HFA Responds to Latest Gene Therapy Announcement
Gene Therapy, Industry News & Research
Yesterday, the U.S. Food and Drug Administration approved Roctavian (valoctocogene roxaparvovec), the first gene therapy for hemophilia A. This new treatment option, for the treatment of…
Statement: HFA Responds to Latest Gene Therapy Announcement
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Yesterday, the U.S. Food and Drug Administration approved Roctavian (valoctocogene roxaparvovec), the first gene therapy for hemophilia A. This new treatment option, for the treatment of…
New Medscape Activity to Help Institutions Offer Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
NHF is pleased to announce that its collaboration with Medscape continues with a new accredited educational activity designed to help healthcare providers…
FDA Approves First Gene Therapy to Treat Adults with Hemophilia B
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
On November 22, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus (AAV) vector-based gene therapy. It is…
Unanswered Questions Remain in Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
A recent commentary published in the journal Haemophilia, posits that while clinical trials for hemophilia gene therapy approach conclusion, there remain significant unanswered…