Factor VIII

hC Bioscience Announces Lead Program in Hemophilia and Reports Positive Preclinical Data on Novel Protein Editing Approach Using Anticodon Engineered tRNA

Retrieved on: 
Tuesday, April 23, 2024
Biotechnology, General Health, Pharmaceutical, Health, Haemophilia, World government, Mouse, DSM-IV codes, IND, Liver, Patient, Small RNA, Factor VIII, PTC

hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today announced preclinical data supporting its lead program in severe hemophilia A at the World Federation of Hemophilia 2024 World Congress in Madrid, Spain.

Key Points: 
  • hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today announced preclinical data supporting its lead program in severe hemophilia A at the World Federation of Hemophilia 2024 World Congress in Madrid, Spain.
  • Jose Lora, Ph.D., Chief Science Officer of hC Bioscience, unveiled the company’s first development candidate, HCB-101, an anticodon engineered tRNA designed to suppress nonsense mutations.
  • HCB-101 is delivered as a lipid nanoparticle to target the liver, the organ where Factor VIII is produced.
  • “We’re excited to develop tRNA-based protein editing as a potential new breakthrough treatment option for patients with severe hemophilia A.

Avenacy Announces Launch of Desmopressin Acetate for Injection in the U.S. Market

Retrieved on: 
Monday, April 15, 2024
Hospitals, Pharmaceutical, Health, Cardiology, FDA, Administration, Boxed warning, Respiratory arrest, Injury, Bleeding, Risk, Factor VIII, Food, Hemarthrosis, Warning, Hyponatremia, Death, Polydipsia, Seizure, Contraindication, Patient, Diabetes insipidus, Central diabetes insipidus, Haemophilia, Coma, Pharmaceutical industry

Avenacy, a specialty pharmaceutical company focused on supplying critical injectable medications, today announced it has launched Desmopressin Acetate for Injection in the United States as a therapeutic equivalent generic for DDAVP® for Injection (Desmopressin Acetate) approved by the U.S. Food and Drug Administration.

Key Points: 
  • Avenacy, a specialty pharmaceutical company focused on supplying critical injectable medications, today announced it has launched Desmopressin Acetate for Injection in the United States as a therapeutic equivalent generic for DDAVP® for Injection (Desmopressin Acetate) approved by the U.S. Food and Drug Administration.
  • Desmopressin Acetate for Injection is multi-indicated for patients with central diabetes insipidus, hemophilia A, and von Willebrand’s disease (Type I).
  • View the full release here: https://www.businesswire.com/news/home/20240415847399/en/
    Avenacy’s Desmopressin Acetate for Injection is available in two presentations: 4 mcg per 1 mL single-dose vials, and 40 mcg per 10 mL multiple-dose vials.
  • Avenacy will begin shipping Desmopressin Acetate for Injection to wholesale partners this week.

Metagenomi Reports Business Updates and Full Year 2023 Financial Results

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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

Octapharma to present clinical and scientific data that advances our understanding and treatment of bleeding disorders at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), which takes place in Frankfurt, Ge

Retrieved on: 
Wednesday, January 17, 2024
Factor VIII, Senior, Octapharma, Patient, Research, Nosebleed, SLAM, VWD, Trial of the century, Pharmaceutical industry

The study shows that wilate® prophylaxis is highly effective at reducing bleeding rates in children and adults with all types of VWD and across examined bleeding sites.

Key Points: 
  • The study shows that wilate® prophylaxis is highly effective at reducing bleeding rates in children and adults with all types of VWD and across examined bleeding sites.
  • The results provide compelling evidence for the use of regular prophylaxis in people with VWD.
  • "The new data from the WIL-31 study provide strong evidence for the use of wilate® prophylaxis in people with all types of VWD.
  • In such instances, indirect treatment comparisons can be used to compare the effects of different treatments.

Sernova Receives Orphan Drug and Rare Pediatric Disease Designations for its Hemophilia A Program from FDA

Retrieved on: 
Monday, November 27, 2023
Food, FDA, Cell, ODD, Haemophilia, Haemophilia A, TSX, Patient, Factor VIII, PSH, Pharmaceutical industry

LONDON, Ontario and WINDHAM COUNTY, Conn., Nov. 27, 2023 (GLOBE NEWSWIRE) -- Sernova Corp. (TSX:SVA) (OTCQB:SEOVF) (FSE/XETRA:PSH), a clinical-stage company and leader in cell therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the company’s Hemophilia A program.

Key Points: 
  • LONDON, Ontario and WINDHAM COUNTY, Conn., Nov. 27, 2023 (GLOBE NEWSWIRE) -- Sernova Corp. (TSX:SVA) (OTCQB:SEOVF) (FSE/XETRA:PSH), a clinical-stage company and leader in cell therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the company’s Hemophilia A program.
  • The FDA grants orphan designation, also referred to as orphan status, to therapies intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S.
  • This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.
  • Separately, rare pediatric disease designations are granted for rare diseases that primarily affect children under 18 years old with recipients of this designation being awarded a priority review voucher, upon approval.

FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B

Retrieved on: 
Monday, December 11, 2023
Biotechnology, FDA, Pharmaceutical, Health, Medicine, Safety, NYSE, Senior, PFE, Food, TFPI, Haemophilia, BLA, European Medicines Agency, European Commission, ASH, MAA, EMA, Civil service commission, Patient, Research and development, Research, Internal medicine, FIX, Rusyns, Exposition, Factor IX, Hemostasis, Development, Factor VIII, PDUFA, Society, Inc., Infection, Pharmaceutical industry, Pfizer

The European marketing authorization application (MAA) for marstacimab also passed validation and is currently under review by the European Medicines Agency (EMA).

Key Points: 
  • The European marketing authorization application (MAA) for marstacimab also passed validation and is currently under review by the European Medicines Agency (EMA).
  • If approved in the U.S. and EU, marstacimab is expected to become the first once-weekly subcutaneous treatment for people living with hemophilia B and the first treatment administered as a flat dose for people living with hemophilia A or B.
  • For appropriate patients living with hemophilia A and B, the goal of this treatment is to prevent potentially life-threatening bleeds with a once-weekly, subcutaneous flat-dose administration.
  • The submissions for marstacimab are based on efficacy and safety data from the Phase 3 BASIS trial ( NCT03938792 ).

Marstacimab Phase 3 Data Presented at ASH 2023 Demonstrate Significant Bleed Reduction in Hemophilia A and B

Retrieved on: 
Sunday, December 10, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Cardiology, Safety, NYSE, Senior, PFE, Haemophilia, ASH, Risk, Research and development, Research, Internal medicine, FIX, ABR, Rusyns, Factor IX, Bleeding, Development, ATP, Factor VIII, Society, Inc., Infection, Pharmaceutical industry, Pfizer

The results from the BASIS trial demonstrated a statistically significant and clinically meaningful effect on annualized bleeding rate (ABR).

Key Points: 
  • The results from the BASIS trial demonstrated a statistically significant and clinically meaningful effect on annualized bleeding rate (ABR).
  • The findings were presented today at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego.
  • In addition to the BASIS study, fidanacogene elaparvovec and giroctocogene fitelparvovec are investigational gene therapy treatments being studied for the treatment of adults living with hemophilia B and hemophilia A, respectively.
  • Updated data from both gene therapy programs, including an oral presentation of four-year results from Pfizer’s Phase 1/2 study of giroctocogene fitelparvovec in adults living with severe hemophilia A, will be presented at the ASH meeting.

Octapharma AG: Indirect comparison data indicate that personalised prophylaxis with Nuwiq® may lead to fewer patients with haemophilia A having bleeds than with other recombinant factor VIII products

Retrieved on: 
Tuesday, October 31, 2023
Haemophilia, Factor VIII, European Journal, PROPEL, PROTECT, Exercise, Georgetown University Medical Center, VIII, Georgetown Lombardi Comprehensive Cancer Center, Efmoroctocog alfa, Patient, Pharmaceutical industry, Dentistry

The analysis used data from prospective clinical trials of personalised prophylaxis in adults with severe haemophilia A.

Key Points: 
  • The analysis used data from prospective clinical trials of personalised prophylaxis in adults with severe haemophilia A.
    Dr Kessler commented, "This MAIC exercise provides important comparative data on the efficacy and consumption of various recombinant FVIII products in adults with haemophilia A on personalised prophylaxis.
  • The results can be considered by clinicians and patients when treatment decisions are being made."
  • MAICs are a way of comparing the efficacy of different medical interventions or products in the absence of head-to-head randomised trials3.
  • In addition, a short video summarising the results is available in the online version of the article.