Hemophilia Insights

China Approves First Hemophilia B Gene Therapy

April 16, 2025 • Estimated Reading Time: 1 minute

Latest Research

Current gene therapies for hemophilia B aim to deliver a functional gene to replace or supplement the defective one, often using viral vectors like adeno-associated viruses (AAVs). These treatments have shown potential in reducing bleeding episodes and minimizing the need for frequent factor replacement infusions.

However, challenges remain, including the need for more predictable and durable treatments that can be redosed if necessary. Gene therapies have shown promise, particularly with advancements like the use of factor IX-Padua, which has significantly improved outcomes in clinical trials (Liu & A, 2025).

Clinical Trials

This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A.

Study for Turoctocog Alfa Treatment Regimen in Iraqi Haemophilia A Patients

View the full list of clinical trials →

Community News

Hemophilia Federation of AmericaApr 08, 2025

On this day 35 years ago, Ryan White lost his battle with HIV/AIDS at the age of 18. During the 1980s, Ryan bravely became a national spokesperson and symbol of hope for the hemophilia and HIV/AIDS community after being expelled from middle school due to community fears the virus would spread to other children. Today, his mother Jeanne continues to work tirelessly to spread Ryan's message and advocate for all individuals who are suffering. We remember Ryan everyday, but especially today. ❤️ 🩸

World Federation of HemophiliaApr 11, 2025

This week, the WFH took part in the WHO side event on Advancing Health Equity: Universal Access to Health Products, held during the 58th Session of the Commission on Population and Development at the United Nations.

During this policy roundtable, the WFH delivered a statement emphasizing its submissions for the 2025 update of the WHO Essential Medicines List (EML). The statement highlighted the need to better leverage the EML to improve equitable access to treatment at the country level—through more effective centralized procurement mechanisms and greater involvement of patient organizations and clinicians in the process. Watch the recording of the meeting here: https://bit.ly/3G2VkqR#AdvanceHealthEquity# BleedingDisorders

The Haemophilia Society UKApr 08, 2025

Last weekend, we hosted the latest of our Newly-Diagnosed Families events. This is always a highlight of our events calendar as we bring together a group of families for a weekend of information, insights and workshops to answer questions and help families begin to get their heads round a recent bleeding disorder diagnosis.

It gives them an opportunity to ask any burning questions and meet some of the THS team, including our amazing youth ambassadors.

It was, as ever, a fantastic event and such a pleasure to meet all of those who attended. We'll be sharing details of the next one very soon.