Haemophilia

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codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn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codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn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codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Centessa Pharmaceuticals Reports Business Highlights and Financial Results for the Fourth Quarter and Full-Year 2023

Retrieved on: 
Thursday, March 28, 2024
Narcolepsy, Toxicity, Administration, Development, ASH, ABR, Haemophilia, Part, APC, Tumor microenvironment, Observation, Volunteering, Trial of the century, CD47, Investment, Society, Bleeding, CNTA, ATM, Safety, OX2R, Sleep, Person, Pharmaceutical industry

“This is an exciting and pivotal time for Centessa,” said Saurabh Saha MD PhD, Chief Executive Officer of Centessa.

Key Points: 
  • “This is an exciting and pivotal time for Centessa,” said Saurabh Saha MD PhD, Chief Executive Officer of Centessa.
  • Cash, Cash Equivalents and Short-term Investments: $256.5 million as of December 31, 2023, which includes approximately $6.2 million in net proceeds through ATM sales in the fourth quarter ended December 31, 2023.
  • General & Administrative Expenses: $12.3 million for the fourth quarter ended December 31, 2023, compared to $13.8 million the fourth quarter ended December 31, 2022, and $53.7 million for the full-year 2023 compared to $55.2 million for the full-year 2022.
  • Net Loss Attributable to Ordinary Shareholders: $36.8 million for the fourth quarter ended December 31, 2023, compared to $43.2 million for the fourth quarter ended December 31, 2022, and $151.1 million for the full-year 2023 compared to $216.2 million for the full-year 2022.

Metagenomi Reports Business Updates and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 27, 2024
Research, Dicarboxylic acid, AAV, Enzyme, Development, Gene editing, CAST, Cardiovascular disease, Haemophilia, Vivisection, Patient, Primate, RNA, DNA, Wave 1, Sale, Metagenomics, G&A, IPO, Ultra, Doctor of Philosophy, Protein, MBA, Genome, SMART, Factor VIII, CRISPR, Amyloidosis, MGX, AGT, Liver, LNP, Ionis, PAM, Vaccine

EMERYVILLE, Calif., March 27, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived gene editing toolbox, today reported financial results for the full year ended December 31, 2023, and additional business updates.

Key Points: 
  • The aggregate gross proceeds to Metagenomi from the offering were approximately $93.75 million, before deducting underwriting discounts and commissions and offering expenses.
  • In addition, cash used to fund our operations was $91.4 million for the year ended December 31, 2023.
  • R&D Expenses: Research and development (R&D) expenses were $94.4 million for the full year ended December 31, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $28.8 million for the full year ended December 31, 2023.

Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter Treatment of haemophilia [...]

Retrieved on: 
Tuesday, April 9, 2024
Community, Civil service commission, Diagnosis, Prevalence, Disease, Patient, Committee, Haemophilia, EMA, IRIS, Human, Agency, European, COMP, Bleeding, Factor VIII, Telephone numbers in Hungary, European Commission, MDC, Marketing, Blood, Liver, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter Treatment of haemophilia A, 13/04/2022 Positive

Key Points: 


Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter Treatment of haemophilia A, 13/04/2022 Positive

Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

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

Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 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

Registration Opens for the National Association of Specialty Pharmacy 2024 Annual Meeting & Expo

Retrieved on: 
Tuesday, March 12, 2024
Convention center, Pharmacist, Annual, Organ transplantation, Rheumatoid arthritis, CE, Woman, Multiple sclerosis, Haemophilia, Pharmacy, Patient, Cancer, Abstract, Love, Specialty pharmacy, National Association, CSP, Cell, Data management, Nursing, Education, NASP, Consultant, Cystic fibrosis, Accreditation, Hospital

Washington, DC, March 12, 2024 (GLOBE NEWSWIRE) -- Today, the National Association of Specialty Pharmacy (NASP), the only industry association in the U.S. whose membership includes all specialty pharmacy stakeholders, opened registration for the NASP 2024 Annual Meeting & Expo, to be held October 6 - 9, 2024, at the Gaylord Opryland Resort & Convention Center in Nashville, TN.

Key Points: 
  • Washington, DC, March 12, 2024 (GLOBE NEWSWIRE) -- Today, the National Association of Specialty Pharmacy (NASP), the only industry association in the U.S. whose membership includes all specialty pharmacy stakeholders, opened registration for the NASP 2024 Annual Meeting & Expo, to be held October 6 - 9, 2024, at the Gaylord Opryland Resort & Convention Center in Nashville, TN.
  • “The NASP 2024 Annual Meeting & Expo offers everything that attendees have come to love and expect, including top-of-class educational content and networking events.
  • Workshops include Accreditation, Cell & Gene Therapy, Certified Specialty Pharmacist (CSP) Exam Prep Course, Hospital/Health System Specialty Pharmacy, Specialty Pharmacy Law, and Technology.
  • The Annual Meeting & Expo also features a showcase for specialty pharmacy clinical research with its abstract and poster program.

Hemophilia a (Factor Viii Deficiency) Global Clinical Trials Review 2024: Trial Status, Trial Phase, Sponsor Type and End Point Status - ResearchAndMarkets.com

Retrieved on: 
Thursday, April 4, 2024
Research, Clinical Trials, Cardiology, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Trial, Haemophilia, Clinical trial, Haemophilia A, Pharmaceutical industry

The "Hemophilia a (Factor Viii Deficiency) - Global Clinical Trials Review, 2024" clinical trials has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Hemophilia a (Factor Viii Deficiency) - Global Clinical Trials Review, 2024" clinical trials has been added to ResearchAndMarkets.com's offering.
  • The report enhances the decision making capabilities and helps to create an effective counter strategies to gain competitive advantage.
  • This report provides top line data relating to the clinical trials on Hemophilia A (Factor VIII Deficiency).
  • The report offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status and sponsor type.

United States In-Vitro Diagnostics Market Analysis 2023-2030: Technological Advancements Drive IVD Adoption, Enhancing Patient Outcomes Fueling Demand - ResearchAndMarkets.com

Retrieved on: 
Friday, March 22, 2024
Biotechnology, Baby, Maternity, Health, General Health, Health Technology, Medical Devices, Research, Hospitals, Genetics, Consumer, Science, Automation, PCR, Cardiovascular disease, Hematology, Haemophilia, Diabetes, Ageing, American Cancer Society, Tissue, Cancer, IVD, Leukemia, Workflow, Anemia, Blood, Medical device

The market is poised for growth with the development of automatic IVD structures that provide improved efficiency, accuracy, and throughput.

Key Points: 
  • The market is poised for growth with the development of automatic IVD structures that provide improved efficiency, accuracy, and throughput.
  • This reflects a commitment to enhancing patient care through innovative technology and positioning the United States at the forefront of the in-vitro diagnostics market.
  • Technological advancements are set to propel the United States in the in vitro diagnostics (IVD) market.
  • Instruments are predicted to be among the leading products in the United States in vitro diagnostics (IVD) market.

BioTek reMEDys Promotes Awareness and Education for Bleeding Disorders Awareness Month

Retrieved on: 
Wednesday, March 20, 2024
Research, Infectious Diseases, Public Policy, Government, Healthcare Reform, Biotechnology, Health, Pharmaceutical, General Health, Science, Diagnosis, Coagulopathy, Haemophilia, Caregiver, Education, Patient, Von Willebrand disease, Pharmacist, Blood, Physician, BioTek

In recognition of March being Bleeding Disorders Awareness Month , BioTek reMEDys , a multi-site national integrated infusion therapy provider focused on providing support to people with rare diseases, is publicly calling for awareness of safe medication use and treatments for patients with bleeding disorders and conditions.

Key Points: 
  • In recognition of March being Bleeding Disorders Awareness Month , BioTek reMEDys , a multi-site national integrated infusion therapy provider focused on providing support to people with rare diseases, is publicly calling for awareness of safe medication use and treatments for patients with bleeding disorders and conditions.
  • “Bleeding Disorders Awareness Month is an opportunity and a reminder to advocate for education as a priority throughout the healthcare continuum as we work towards recognizing the challenging journey patients often have on the way to a correct diagnosis and treatment."
  • Each March, the community calls attention to inheritable blood and bleeding disorders during Bleeding Disorders Awareness Month.
  • To learn more about hemophilia, von Willebrand disease, rare factor disorders and other bleeding disorders, visit hemophilia.org .