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June 19, 2020June 19, 2020

Efficacy of Gene Therapy SB-525 Now at 14 Months in Alta Hemophilia A Trial

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Up to 14 months after a one-time infusion of SB-525, an investigational gene therapy, durable increases in the activity of clotting factor VIII (FVIII) are continuing in all five ... Read more

June 17, 2020June 17, 2020

DalcA Shows Sustained Efficacy in Hemophilia B in Trial

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Under-the-skin, preventive treatment with Dalcinonacog alfa (DalcA) led to sustained and protective levels of factor IX (FIX) in people with severe hemophilia B, final data from a Phase 2b trial ... Read more

June 15, 2020June 15, 2020

Roughly 30,000 Males With Hemophilia in the US, Study Estimates

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Approximately 30,000 males have hemophilia in the U.S., according to estimates from a recent study, which also found that patients with hemophilia are generally young and that the disease ... Read more

June 12, 2020June 12, 2020

Catalyst Completes Phase 1 Trial of MarzAA, Is on Track to Start Phase 3

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Despite the COVID-19 pandemic, Catalyst Biosciences has successfully completed a Phase 1 trial of marzeptacog alfa activated (MarzAA), with results supporting the treatment as an on-demand option for bleeding ... Read more

June 10, 2020June 10, 2020

Sernova Testing AgeX’s UniverCyte Technology to Deliver FVIII to Hemophilia A Patients

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Sernova will use AgeX Therapeutics’ UniverCyte gene technology to create a cell-based therapy that delivers therapeutic levels of factor VIII (FVIII) — the blood clotting protein deficient in people with ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

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The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis ... Read more

June 8, 2020June 8, 2020

Regeneron, Intellia Expand Deal to Develop CRISPR/Cas9-Based Treatments

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Regeneron Pharmaceuticals and Intellia Therapeutics are expanding their collaboration to develop hemophilia A and B treatments using the CRISPR/Cas9 gene editing technology. The two companies signed a six-year agreement ... Read more

June 5, 2020June 5, 2020

Gene, Cell Therapy Combo Shows Promise for Treating Hemophilia A, Study Reports

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A new combination of gene and cell-based therapy durably delivered therapeutic levels of factor VIII (FVIII) — the blood clotting protein missing or defective in people with hemophilia A ... Read more

June 3, 2020June 3, 2020

BioMarin’s Gene Therapy for Hemophilia A Continues to Prevent Bleeds After Four Years, Trial Shows

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BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec continues to safely and effectively prevent bleeding episodes and the need for prophylactic clotting factor VIII in adults with severe hemophilia A, four-year data from ... Read more

June 1, 2020June 1, 2020

RNA Gene Therapy Seen to Effectively Treat Hemophilia A Mouse Model

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A new type of gene therapy that uses RNA instead of DNA led to rapid and prolonged production of therapeutic levels of factor VIII (FVIII) in a mouse model ... Read more

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