A5075639715- Hemophilia Treatment and Research
- Virus-based gene therapy research
- CAR-T cell therapy research
- CRISPR and Genetic Engineering
- Acute Ischemic Stroke Management
- Platelet Disorders and Treatments
- Blood Coagulation and Thrombosis Mechanisms
- Cerebrovascular and Carotid Artery Diseases
- Stroke Rehabilitation and Recovery
- Cancer-related gene regulation
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Central Venous Catheters and Hemodialysis
- Neurogenetic and Muscular Disorders Research
- Blood properties and coagulation
- Viral Infectious Diseases and Gene Expression in Insects
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Sexual function and dysfunction studies
- RNA modifications and cancer
- Venous Thromboembolism Diagnosis and Management
- Adolescent and Pediatric Healthcare
- Uterine Myomas and Treatments
- Cerebral Venous Sinus Thrombosis
Oregon Health & Science University
2016-2024
National Institutes of Health
2006
National Institute of Neurological Disorders and Stroke
2003-2005
Walter Reed National Military Medical Center
2003
Inova Fairfax Hospital
2003
Naval Medical Research Command
2003
Emory University
2003
Washington Adventist Hospital
2003
Adventist HealthCare
2003
Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for aims establish sustained activity, thereby protecting against bleeding without burdensome replacement.
Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including gene cassette containing the factor IX (FIX) Padua variant under control of liver-specific promoter. A phase 2b study was conducted to confirm that single dose 2 × 1013 genome copies per kilogram etranacogene will result in FIX activity ≥5% 6 weeks after dosing. Secondary end points included at other time points, bleed frequency, replacement, and safety. administered as IV infusion 3 adults with severe moderately...
Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing codon-optimized Padua variant human factor IX (FIX) transgene with liver-specific promoter. Here, we report 3-year outcomes from phase 2b, open-label, single-dose, single-arm, multicenter trial conducted among adults severe or moderately hemophilia B (FIX ≤2%). All participants (n = 3) received single intravenous dose (2 × 1013 gene copies per kg) and will be followed up for years....
Background The National Hemophilia Foundation (NHF) conducted extensive all-stakeholder inherited bleeding disorder (BD) community consultations to inform a blueprint for future research. Sustaining and expanding the specialized comprehensive Treatment Center care model, better serve all people with BDs (PWIBD), increasing equitable access optimal health emerged as top priorities.Research Design Methods NHF, American Thrombosis Hemostasis Network (ATHN), convened multidisciplinary expert...
To increase the proportion of ischemic stroke patients treated with thrombolytic therapy, establishment primary centers in community hospitals has been advocated. We evaluated use therapy before and after institution a center hospital.The availability an on-call emergency response team was only significant additional resource required for this hospital. All eligible were intravenous tissue plasminogen activator (tPA). The number cerebrovascular disease, tPA, times to treatment, patient...
Etranacogene dezaparvovec (CSL222, formerly AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing the highly active factor IX (FIX) Padua variant controlled by liver-specific promoter. This Phase 2b, open-label, single-dose, single-arm, multi-center trial evaluated efficacy and safety of etranacogene dezaparvovec. All participants (n=3) were adults, had severe or moderately hemophilia B (FIX ≤2%), AAV5 neutralizing antibodies. Participants received single...
Rare, chronic diseases such as hemophilia and other congenital coagulation disorders require coordinated delivery of services for optimal outcomes. Hemophilia Treatment Centers (HTCs) are specialized, multidisciplinary health-care centers providing team-based care to meet the physical, psychosocial, emotional needs people with (PWH) may serve a model rare disorders. Health-care purchasers, well general medical community, not appreciate breadth quality provided by HTCs. They exemplify...
Previous studies identified a high frequency of silent ischemic lesion recurrence on magnetic resonance imaging (MRI) after an index stroke.To investigate whether MRI predicts subsequent clinical events.Retrospective cohort study.General community hospital. Patients We recruited 120 patients who experienced acute stroke (IS) and underwent initial within 24 hours onset day 5. Of those patients, 68 follow-up up to 90 days onset.Early was defined as new asymptomatic lesions 5-day MRI, late 30-...
To address the need for a patient-reported outcome that can measure clinically and personally meaningful change in people with haemophilia (PwH) on prophylaxis, an approach based Goal Attainment Scaling (GAS) was developed: GAS-Hēm.To establish real-world feasibility of GAS-Hēm PwH.Patients aged 5-65 years were enroled from four North American centres 12-week study. The primary proportion participants who completed interviews at baseline, 6 12 weeks. scores obtained by subject-...
Scientific Research Question: We have previously reported steady state factor IX (FIX) activity levels of 3–13% following a Phase I/II trial AMT-060, an investigational gene therapy consisting adeno-associated virus serotype 5 (AAV5) vector containing codon-optimized wildtype human FIX under control liver-specific promoter. AMT-061 consists the same AAV5 and promoter as but with Padua variant (hFIX) resulting from single nucleotide change arginine to leucine at R338L. This has been enhance...
Introduction Etranacogene dezaparvovec (formerly AMT-061) is an investigational gene therapy for haemophilia B comprising adeno-associated virus serotype 5 (AAV5) vector and a codon-optimised factor IX (FIX) Padua R338L transgene with liver-specific promoter. The Phase 3 HOPE-B trial (NCT03569891) aims to assess the efficacy safety of etranacogene dezaparvovec.