News

July 17, 2020July 17, 2020

#ISTH2020 – FLT180a Gene Therapy Shows Promise for Hemophilia B Patients in Phase 1/2 Trial

News

A single dose of the investigational gene therapy FLT180a can lead to clinically meaningful, sustained increases in the activity of factor IX (FIX), effectively preventing bleeds and the need for ... Read more

July 15, 2020July 15, 2020

#ISTH2020 – Hemlibra Continues to Show Favorable Safety Profile, Early Phase 3b Trial Data Show

News

When given as preventive treatment to avoid spontaneous bleeds in people with hemophilia A with inhibitors, Hemlibra (emicizumab) continues to show a favorable safety profile that is consistent with ... Read more

July 13, 2020July 13, 2020

#ISTH2020 – Gene Therapy SPK-8011 Lowers Bleeding Rate in Hemophilia A for up to 3 Years, Trial Shows

News

A single dose of the investigational gene therapy SPK-8011 is safe and leads to durable production of factor VIII (FVIII) for up to three years, effectively lowering the frequency ... Read more

July 10, 2020July 10, 2020

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

News

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 ... Read more

July 9, 2020July 9, 2020

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

News, syndicated

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 ... Read more

July 8, 2020July 8, 2020

Early Data Support Catalyst Biosciences’ Potential Hemophilia B Gene Therapy

News

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, ... Read more

July 6, 2020July 6, 2020

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

News

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 ... Read more

July 1, 2020July 1, 2020

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

News

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 ... Read more

June 29, 2020June 29, 2020

Sobi, Sanofi Again Donate Clotting Factors to WFH Aid Program

News

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. ... Read more

June 26, 2020June 26, 2020

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

News

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, ... Read more

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