Hemophilia News Today

July 10, 2020July 10, 2020

Freeline Raises $120M for Pivotal Trial of FLT180a, a Gene Therapy Candidate for Hemophilia B

Freeline has raised $120 million to support the launch of a pivotal clinical trial of FLT180a, the company’s lead gene therapy candidate for the treatment of hemophilia B. FLT180a uses ... Read more

July 9, 2020July 10, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

Hemophilia News Today

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in ... Read more

July 8, 2020July 8, 2020

Early Data Support Catalyst Biosciences’ Potential Hemophilia B Gene Therapy

Hemophilia News Today

CB 2679d-GT, an investigational gene therapy for hemophilia B being developed by Catalyst Biosciences, can increase factor IX (FIX) levels and significantly reduce bleeding, early studies show. The data, “Combination ... Read more

July 6, 2020July 7, 2020

Idelvion Approved in Europe for 21-day Prophylactic Dosing in Adults With Hemophilia B

Hemophilia News Today

The European Medicines Agency (EMA) has approved 21-day dosing intervals for CSL Behring‘s Idelvion (rIX‐FP) as a prophylactic treatment for adults with hemophilia B. Idelvion — which is also approved ... Read more

July 1, 2020July 1, 2020

Access to Elocta for Hemophilia A Expanding in UK, Sobi Says

Hemophilia News Today

People with hemophilia A in the U.K. will soon have greater access to Elocta (efmoroctocog alfa) under a two-year agreement between Sobi and the National Health Service (NHS), the company announced. ... Read more

June 29, 2020June 29, 2020

Sobi, Sanofi Again Donate Clotting Factors to WFH Aid Program

Hemophilia News Today

Sobi and Sanofi announced an additional donation of up to 500 million international units (IUs) of clotting factor therapy in support of the World Federation of Hemophilia‘s Humanitarian Aid Program. The ... Read more

June 26, 2020June 26, 2020

CSL Behring Acquires Rights to Market AMT-061, Gene Therapy for Hemophilia B

Hemophilia News Today

CSL Behring has acquired exclusive global rights to commercialize AMT-061, a potential gene therapy for hemophilia B now in a pivotal clinical trial, from its developer uniQure. Under terms of the agreement, uniQure ... Read more

June 24, 2020June 24, 2020

Investigational Gene Therapy Results in Huge Drop in Bleeding Rates and Factor VIII Usage, Updated Data Show

Hemophilia News Today

Valoctocogene roxaparvovec, BioMarin Pharmaceutical‘s investigational gene therapy, results in a more than 90% drop in annual bleeding rates and the use of clotting factor VIII (FVIII) in men with severe ... Read more

June 22, 2020June 22, 2020

Fitusiran Continues to Prevent Bleeds in Hemophilia A or B, Trial Shows

Hemophilia News Today

As a long-term preventive treatment, fitusiran leads to sustained reductions in bleed frequency in people with moderate-to-severe hemophilia A or B regardless of inhibitor status, early results from an extension study ... Read more

June 19, 2020June 19, 2020

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

Hemophilia News Today

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 severe ... Read more

June 17, 2020June 18, 2020

DalcA Shows Sustained Efficacy in Hemophilia B in Trial

Hemophilia News Today

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 show. ... Read more

June 15, 2020June 15, 2020

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

Hemophilia News Today

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 is ... Read more

June 12, 2020June 12, 2020

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

COVID-19 News, Hemophilia News Today

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 episodes ... Read more

June 10, 2020June 11, 2020

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

Hemophilia News Today

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 hemophilia ... Read more

June 8, 2020June 8, 2020

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

Hemophilia News Today

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 in ... Read more