Athletes and Scientists Partner to Support Rare Disease Research

Published on :

Sponsored by the not-for-profit Uplifting Athletes, the Young Investigators Draft provides $20,000 grants to 10 beginning-career stage research scientists in the rare disease community. The award is an unrestricted grant that will support the vital investigation of potential therapies and cures, including gene therapies, for rare disease, said Rob Long, executive director of Uplifting Athletes.

CSL Announces Positive Top-Line Phase 3 Results for Garadacimab as Preventive Treatment in Patients with Hereditary Angioedema (HAE)

CSL Behring Logo
Published on :

Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY), today announced positive top-line Phase 3 results for garadacimab (CSL312), the company’s investigational first-in-class monoclonal antibody inhibiting Factor XIIa being developed as a long-term preventive treatment for patients with hereditary angioedema (HAE).  The study met its primary and secondary efficacy objectives and also demonstrated favorable safety and tolerability. CSL aims to begin filing with global health authorities next year for full approval.

Final Analysis of Pivotal HOPE-B Study Demonstrates Durable and Sustained Therapeutic Effect of Etranacogene Dezaparvovec Gene Therapy in Hemophilia B – Data Presented at EAHAD 2022

CSL Behring Logo
Published on :

Global biotherapeutics leader CSL Behring today announced positive long-term results from the Phase 3 HOPE-B clinical trial evaluating etranacogene dezaparvovec (EtranaDez), an investigational adeno-associated virus five (AAV5)-based gene therapy for people living with hemophilia B, a life-threatening bleeding disorder. Following a single infusion of etranacogene dezaparvovec, participants experienced a stable and durable increase in mean Factor IX (FIX) activity and hemostatic protection at 18 months. The final data, from the largest gene therapy study in hemophilia B, were presented as part of the latest clinical trial results session at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting.

10 Philadelphia Biotech Companies Targeting Rare Diseases

Published on :

Philadelphia, the birthplace of cell and gene therapy, is home to a number of companies focused on developing multiple treatment options for rare diseases, those that affect less than 200,000 people in the United States.