Haemophilia B

Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Brain, Disease, Coagulation, Patient, Committee, Knowledge, Internal, Regulation, Haemophilia, EMA, IRIS, Joint, Human, Wound healing, Clinical trial, European, Therapy, COMP, Bleeding, Spinal cord, Sponsor, European Commission, Immune system, Antibody, Safety, Factor IX, Blood, Marketing, Limited, Haemophilia B, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, Disease, Coagulation, Patient, Committee, Knowledge, Internal, Regulation, Haemophilia, EMA, IRIS, Joint, Human, Wound healing, European, COMP, Bleeding, Spinal cord, Antibody, Sponsor, European Commission, Immune system, Safety, Factor IX, Blood, Marketing, Haemophilia B, Medicine, Pharmaceutical industry

Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Key Points: 


Orphan designation: fidanacogene elaparvovec Treatment of haemophilia B, 19/11/2018 Withdrawn

Intellia Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Thursday, February 22, 2024
Research, Intellia Therapeutics, Regeneron Pharmaceuticals, Fatigue, Haemophilia, NK, CTA, Liver, Cystic fibrosis, ATM, A1AT, Amyloidosis, HAE, Therapy, European Medicines Agency, ATTR, Haemophilia B, DNA, European Commission, Hereditary angioedema, Recode, Conference, Cardiomyopathy, AATD, Civil service commission, Clinical trial, Facial muscles, CRISPR, Gene editing, Food, NTLA, KLKB1, Lung, NEJM, TTR, Patient, Medicine, Disease, Tissue, Pharmaceutical industry

Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.

Key Points: 
  • Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.
  • R&D Expenses: Research and development expenses increased by $9.0 million to $109.0 million during the fourth quarter of 2023, compared to $100.0 million during the fourth quarter of 2022.
  • G&A Expenses: General and administrative expenses increased by $5.4 million to $29.0 million during the fourth quarter of 2023, compared to $23.6 million during the fourth quarter of 2022.
  • Net Loss: The Company’s net loss was $132.2 million for the fourth quarter of 2023, compared to $113.4 million during the fourth quarter of 2022.

Hemophilia B Pipeline Insight Report 2024: Comprehensive Analysis of Emerging Therapeutics - ResearchAndMarkets.com

Retrieved on: 
Tuesday, March 5, 2024
Pharmaceutical, Health, Cardiology, Knowledge, Bleeding, Factor IX, Haemophilia B, Catalog, Genetic disorder, Research, Patient, Haemophilia, Pharmaceutical industry

The "Hemophilia B - Pipeline Insight, 2024" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Hemophilia B - Pipeline Insight, 2024" report has been added to ResearchAndMarkets.com's offering.
  • Significant advances in the treatment and management of Hemophilia B are forthcoming, according to a new comprehensive research publication now available on our website.
  • The global coverage within this report transcends geographical boundaries, providing a universal perspective on the research and development efforts aimed at combatting Hemophilia B.
  • The report elucidates the complexity of Hemophilia B diagnosis and underscores the importance of comprehensive blood coagulation tests.

Human medicines European public assessment report (EPAR): Refixia, nonacog beta pegol, Date of authorisation: 02/06/2017, Revision: 5, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Chills, Patient, Medical Subject Headings, Marketing, Lethargy, European Commission, Agency, INN, Allergy, Stinger, ATC, Antibody, Restless, Blood, Bleeding, International, PEG, European, Headache, Haemophilia B, Choroid plexus, Ageing, Nausea, Brain, Language, Profile, Swelling, Haemophilia, Flushing, Combustion, Select, Safety, Anatomy, Medicine, Vomiting, IIA, CHMP, Medical device

Human medicines European public assessment report (EPAR): Refixia, nonacog beta pegol, Date of authorisation: 02/06/2017, Revision: 5, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Refixia, nonacog beta pegol, Date of authorisation: 02/06/2017, Revision: 5, Status: Authorised

Novotech Releases In-Depth 2023 Global Clinical Trial Landscape Reports on Hemophilia and Primary Biliary Cholangitis

Retrieved on: 
Thursday, November 2, 2023
Female, Haemophilia B, United Kingdom, AAV, III, RNA, Cirrhosis, Ascending cholangitis, Health, Global Jurist of the Year Award, Research, PBC, Bile, FXR, Liver failure, NOX, Woman, Primary biliary cholangitis, Haemophilia A, Patient, Small RNA, Fine chemical, Pharmaceutical industry, Medical imaging, Haemophilia

BOSTON, Nov. 02, 2023 (GLOBE NEWSWIRE) -- Novotech, the leading Asia Pacific centred biotech CRO with global execution capabilities, today released the latest 2023 global clinical trial landscape reports on Primary Biliary Cholangitis (PBC) and Hemophilia .

Key Points: 
  • BOSTON, Nov. 02, 2023 (GLOBE NEWSWIRE) -- Novotech, the leading Asia Pacific centred biotech CRO with global execution capabilities, today released the latest 2023 global clinical trial landscape reports on Primary Biliary Cholangitis (PBC) and Hemophilia .
  • These reports provide up-to-date information on global clinical trial activity, shedding light on the regions that are witnessing the highest trial numbers and the reasons behind it.
  • As per the findings in the Primary Biliary Cholangitis – Global Clinical Trial Landscape report there have been approximately 140 active clinical trials focused on PBC since 2018.
  • In the Hemophilia Global Clinical Trial Landscape report, there has been a substantial increase in global clinical trials aimed at advancing hemophilia treatment since 2018.

Pathway to Cures Announces First Donor to Fund

Retrieved on: 
Monday, October 9, 2023
Philanthropy, Health, Other Philanthropy, Fund Raising, Foundation, Pharmaceutical, Biotechnology, Knowledge, Chairperson, Anemia, Haemophilia B, Sickle cell disease, MD, Haemophilia, Coagulopathy, Blood, Life, Quality of life, Pharmaceutical industry

Pathway to Cures (P2C), the venture philanthropy fund of the National Bleeding Disorders Foundation, focused on early-stage companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community, announces a transformational gift to their fund.

Key Points: 
  • Pathway to Cures (P2C), the venture philanthropy fund of the National Bleeding Disorders Foundation, focused on early-stage companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community, announces a transformational gift to their fund.
  • Kim and Scott Martin have made a founding donation to Pathway to Cures venture fund adding to the existing estate gift which launched the fund earlier this year.
  • “My wife and I feel privileged to launch the fundraising efforts for Pathway to Cures.
  • Dr. Valentino is the Chief Executive Officer at both Pathway to Cures and the National Bleeding Disorders Foundation (NBDF), “as a donor to the venture fund, Scott will also serve as a member of the Donor Advisory Committee for Pathway to Cures.

XOMA Reports Second Quarter 2023 Financial Results and Provides Update on its Royalty Monetization Strategy

Retrieved on: 
Tuesday, August 8, 2023
Glioma, Niemann–Pick disease, Face, NDA, Janssen, G&A, Arimoclomol, Haemophilia B, Acquisition, Patient, NPC, Research, Environment, Pharmaceutical industry, Video game, Chiesi Farmaceutici

EMERYVILLE, Calif., Aug. 08, 2023 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its second quarter 2023 financial results and highlighted recent operational achievements as XOMA accelerates its differentiated biotech royalty and milestone acquisition strategy.

Key Points: 
  • Completed two royalty acquisitions in the first half of 2023 adding one cash flow generating asset, one NDA-ready asset, and a Phase 2 asset
    EMERYVILLE, Calif., Aug. 08, 2023 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its second quarter 2023 financial results and highlighted recent operational achievements as XOMA accelerates its differentiated biotech royalty and milestone acquisition strategy.
  • Our team has been sourcing and reviewing more royalty and milestone acquisition opportunities than ever,” said Brad Sitko, Chief Investment Officer of XOMA.
  • Other income, net was $0.6 million for the second quarter of 2023, compared to other income, net of $0.1 million in the corresponding quarter of 2022.
  • Net loss for the second quarter of 2023 was $5.4 million, compared to net loss of $4.7 million for the second quarter of 2022.

uniQure announces achievement of $100 million milestone related to hemophilia B gene therapy

Retrieved on: 
Tuesday, June 20, 2023
EEA, Bleeding, CSL Behring, Factor IX, Haemophilia, IX, ASX, CSL, AAV, Sale, Haemophilia B, Patient, European Economic Area, Risk, Coagulopathy, Liver, Disease, Pharmaceutical industry, Medical device, EU

HEMGENIX® (etranacogene dezaparvovec) is a one-time administered gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.

Key Points: 
  • HEMGENIX® (etranacogene dezaparvovec) is a one-time administered gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.
  • “uniQure’s successful development of HEMGENIX® further validates our AAV platform built on the back of 25 years of scientific leadership and innovation in the field of gene therapy.
  • Under the agreement with CSL Behring, the milestone payment is due within 30 days after achievement of the milestone.
  • HEMGENIX® is the first approved gene therapy for hemophilia B in the United States, European Union (EU) and European Economic Area (EEA), and the UK.

Amarna Therapeutics announces appointment of Dr Henk Streefkerk as new CEO

Retrieved on: 
Wednesday, June 7, 2023
Streefkerk, Medicine, Heart failure, Clinical trial, CMO, Doctor of Philosophy, Haemophilia B, Actelion, Medical director, Novartis, MD, Patient, Haemophilia, Therapy, Henk, Pharmaceutical industry, Organon

LEIDEN, Netherlands, June 7, 2023 /PRNewswire/ -- Amarna Therapeutics , a privately-held biotechnology company developing transformative gene therapies in a range of rare and prevalent diseases, including Hemophilia B, today announces the appointment of Dr Henk Streefkerk as the Company's new Chief Executive Officer and Medical Director.

Key Points: 
  • LEIDEN, Netherlands, June 7, 2023 /PRNewswire/ -- Amarna Therapeutics , a privately-held biotechnology company developing transformative gene therapies in a range of rare and prevalent diseases, including Hemophilia B, today announces the appointment of Dr Henk Streefkerk as the Company's new Chief Executive Officer and Medical Director.
  • Dr Streefkerk has a distinguished career as CMO of several biotechs, including PIQUR Therapeutics, Cellprotera and Rivia, and brings over a decade of experience working in big pharma including Novartis, Actelion and Organon.
  • "We are very pleased to introduce Henk Streefkerk as our new CEO.
  • Henk will be instrumental in leading Amarna towards the first in human clinical trial," said Thomas Eldered, Chairman of Amarna's supervisory board.