Büşra Ece Günaydın: A New Approach to Factor VIII Gene Insertion for Hemophilia A
Büşra Ece Günaydın, Founders Associate at Onyx Biotech GmbH, shared a post on LinkedIn:
“This week there is a good news for people living with hemophilia A. The FDA approved Metagenomi’s application to start the first in human Phase 1/2 trial of MGX-001.
This a one time treatment designed to help the body make its own clotting factor VIII for the long term.
The trial is only beginning, and it is not know how well it works in people yet. But the approach is different from the gene therapies approved so far for hemophilia A.
Instead of delivering the gene so that it stays separate in the cell, MGX-001 is designed to insert the factor VIII gene at a specific safe location in the albumin gene, so the cell can read it with its own machinery.
Another interesting part about this treatment is how it is manufacturing.
The therapy has two parts: an AAV vector that carries the factor VIII gene, and a lipid nanoparticle that carries the mRNA for the gene-editing tool and its guide.
Two different modalities, each with its own manufacturing and purification process, and both need to meet the same quality bar.
It is a good reminder that as gene therapies get more sophisticated, the manufacturing side often grows in complexity along with them.”
Stay updated with Hemostasis Today.
-
Nov 13, 2026, 22:57Rolf Bekendam: Moderating a Conversation With Experts on Rare Bleeding Disorders
-
Nov 13, 2026, 16:17Shailendra Singh: Bombay Hospital Jaipur Inaugurates New Facility with Voluntary Blood Donation Camp
-
Nov 13, 2026, 16:10Minka Zivkovic: UMC Utrecht Announces Research Analyst Vacancy in Thrombosis and Haemostasis
-
Nov 13, 2026, 16:02Rounak Dubey: Elsevier’s Upcoming Handbook Explores Digitalization in Transfusion Medicine
-
Nov 13, 2026, 15:50Maha Othman: CanVECTOR 2026 Highlights Research on Immunothrombosis and Cancer-Associated Thrombosis
-
Nov 13, 2026, 15:48Cedric Hermans: Advances in Gene Therapy for Haemophilia A
-
Nov 13, 2026, 15:45Strengthens Global Collaboration in Thrombosis and Hemostasis for 2027 – ISTH
-
Nov 13, 2026, 15:42David McIntosh: The Global Blood Shortage Crisis and the Urgent Need for National Action
-
Nov 13, 2026, 15:41JTH Highlights Future Directions in Thromboprophylaxis for Hospitalized Children