Quellen (Gentherapie) - Hämophilietherapie

(1) Gentherapie bei Hämophilie: Eine ISTH-Bildungsinitiative
(2) Interessengemeinschaft Hämophiler e.V. (IGH)
(3) PM Spark Therapeutics (2. Dezember 2018): Spark Therapeutics Announces Updated Data on SPK-8011 from Ongoing Phase 1/2 Dose-escalation Clinical Trial in Hemophilia A at 60th American Society of Hematology (ASH) Annual Meeting and Exposition
(4) PM Roche (25. Februar 2019): Roche und Spark Therapeutics schliessen verbindliche Übernahmevereinbarung
(5) PM bluebird bio (9. Oktober 2019): bluebird bio and Novo Nordisk enter into research agreement to develop in vivo genome editing candidates for haemophilia and other severe genetic diseases
(6) PM CRISPR (21. Oktober 2019): CRISPR Therapeutics and Bayer Announce an Update on Casebia Therapeutics
(7) PM Sangamo (7. Dezember 2019): Sangamo and Pfizer Announce Updated Phase 1/2 Results Showing Sustained Increased Factor VIII Activity Through 44 Weeks Following SB-525 Gene Therapy Treatment
(8) PM BioMarin (23. Dezember 2019): BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A
(9) NEJM (2. Januar 2020): Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
(10) WSJ (16. Januar 2020): BioMarin Explores Pricing Experimental Gene Therapy at $2 Million to $3 Million
(11) PM BioMarin (20. Februar 2020): BioMarin’s Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA with Review Action Date of August 21, 2020
(12) PM BioMarin (31. Mai 2020): BioMarin Provides Highlights of 4 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
(13) PM Spark Therapeutics (2. Juni 2020): Study Demonstrating Role of IdeS in Enabling of Gene Therapy in the Presence of Neutralizing Anti-AAV Antibodies Published in Nature Medicine
(14) PM BioMarin (17. Juni 2020): BioMarin Provides Additional Data from Recent 4 Year Update of Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in Late-Breaking Oral Presentation at World Federation of Hemophilia Virtual Summit
(15) PM Pfizer (18. Juni 2020): Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Factor VIII Activity Levels and No Bleeding Events or Factor Usage in 3e13 vg/kg Cohort Following giroctocogene fitelparvovec (SB-525) Gene Therapy
(16) PM uniQure (24. Juni 2020): uniQure Announces License Agreement with CSL Behring to Commercialize Hemophilia B Gene Therapy
(17) PM Spark Therapeutics (12. Juli 2020): Spark Therapeutics Announces Updated Data on SPK-8011 from Phase 1/2 Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress
(18) PM BioMarin (19. August 2020): BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
(19) PM Pfizer (7. Oktober 2020): PFIZER AND SANGAMO DOSE FIRST PARTICIPANT IN PHASE 3 STUDY EVALUATING HEMOPHILIA A GENE THERAPY TREATMENT
(20) PM Bayer (26. Oktober 2020): Bayer erwirbt Asklepios BioPharmaceutical und baut Innovationsbasis im Bereich Zell- und Gentherapien aus
(21) PM WFH, EHC und NHF (6. November 2020): Globaldosing hold in fitusiran trialsinitiated by Sanofi Genzyme to investigate new adverseevents
(22) Letter BioMarin (4. November 2020): Withdrawal of Roctavian, valoctocogene roxaparvovec, 2 x 1013 vector genomes/ml, solution for infusion, for Marketing Authorisation Application, EMEA/H/C/0004749
(23) News EMA (13. November 2020): Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 9-12 November 2020 (s.: Withdrawals of applications)
(24) PM uniQure (19. November 2020): Positive Top-Line Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B
(25) PM Pfizer (23. November 2020): Pfizer Doses First Participant in Phase 3 Study Evaluating anti-TFPI Investigational Therapy, Marstacimab, for People With Severe Hemophilia A and B With or Without Inhibitors
(26) PM Pfizer (7. Dezember 2020): Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Factor VIII Activity Levels in 3×10¹³ VG/KG Cohort Through One Year Following Hemophilia A Gene Therapy
(27) PM uniQure (21. Dezember 2020): uniQure Announces Clinical Update on Hemophilia B Gene Therapy Program
(28) PM uniQure (29. März 2021): uniQure Announces Findings from Reported Case of Hepatocellular Carcinoma (HCC) in Hemophilia B Gene Therapy Program
(29) PM uniQure (26. April 2021): uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program
(30) PM uniQure (9. Dezember 2021): uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B
(31) PM CSL Behring (16. Dezember 2021): EMA gewährt beschleunigte Beurteilung der Hämophilie-B-Gentherapie von CSL Behring
(32) PM BioMarin (17. März 2022): BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A
(33) PM CSL Behring (28. März 2022): European Medicines Agency Commences Review of Novel Gene Therapy Candidate Etranacogene Dezaparvovec for People with Hemophilia B