News

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…

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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…

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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…

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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…

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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…

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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…

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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…

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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…

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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…

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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….

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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…

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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…

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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…

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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…

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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…

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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…

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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…

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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…

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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…

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FDA Recommends Additional Data for BioMarin’s Investigational Gene Therapy

Gene Therapy, Industry News & Research, Living with a Bleeding Disorder

Biomarin announced that they received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) on August 18th for their…

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CSL Acquires Hemophilia B Gene Therapy Candidate from uniQure

Gene Therapy, Industry News & Research, Living with a Bleeding Disorder

CSL Behring recently announced that it has agreed to acquire exclusive global license rights from uniQure, to commercialize etranacogene dezaparvovec (AMT-061), an…

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Two Gene Therapy Medscape Activities Recertified

Gene Therapy, Industry News & Research, Living with a Bleeding Disorder

The National Hemophilia Foundation (NHF) is pleased to announce that two educational activities in the “Clinical Advances in Gene Therapy for Hemophilia”…

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Casebia Presents Dual Approach to Gene Therapy

Gene Therapy, Industry News & Research, Living with a Bleeding Disorder

Cambridge, Massachusetts-based Casebia Therapeutics recently presented new research on a novel therapy being developed for patients with hemophilia A. Casebia Therapeutics is…

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