Haemophilia

Sangamo Therapeutics Reports Recent Business Highlights and First Quarter 2024 Financial Results

Retrieved on: 
torsdag, maj 9, 2024
Biotechnology, Neurology, Health, Genetics, Pharmaceutical, STAC, FDA, CTA, Haemophilia, Enzyme replacement therapy, Kite, Investment, Nav1.7, Fabry disease, Poster, Disease, ERT, Sigma-Aldrich, STAAR, Health care, MAA, Partnership, Sangamo Therapeutics, MINT, AAV, Ligand Pharmaceuticals, IND, Tauopathy, Novartis, DNA, Patient, Pivotal, DSM-IV codes, EMA, Prion, BBB, Genome, BLA, MHRA, Manuscript, BioRxiv, Capsid, Modularity, Biogen, Therapy, Food and Drug Administration, European Medicines Agency, Gene, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.
  • “We are pleased with the progress being made in business development discussions across our portfolio, including our Fabry disease program.
  • An IND submission is expected in the fourth quarter of 2024.
  • Revenues for the first quarter ended March 31, 2024 were $0.5 million, compared to $158.0 million for the same period in 2023.

hC Bioscience Presents Preclinical Data at ASGCT Showing Strong Potential in Hemophilia A Using tRNA-based Protein Editing Platform

Retrieved on: 
onsdag, maj 8, 2024
Oncology, Health, Genetics, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Haemophilia, ACE, Cancer, DSM-IV codes, Bleeding, Mouse, Cell, Duchenne, IND, Patient, Gene, Society, Biology, Duchenne muscular dystrophy, Small RNA, PTC, Vaccine

hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today presented preclinical data supporting its lead program in severe hemophilia A at the American Society of Gene and Cell Therapy Annual Meeting in Baltimore, Md.

Key Points: 
  • hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today presented preclinical data supporting its lead program in severe hemophilia A at the American Society of Gene and Cell Therapy Annual Meeting in Baltimore, Md.
  • The data also show that HCB-101 can be delivered successfully via a lipid nanoparticle to target cells in the livers of mice.
  • This tRNA-based protein editing approach has the potential for application in about 20 percent of severe hemophilia A cases and could be extended across a broad spectrum of other diseases caused by nonsense mutations.
  • “We see hemophilia A as the first of many diseases that could be addressed using our tRNA-based protein editing technology, which has broad potential to functionally undo the effects of any disease-causing nonsense mutation.

Octapharma USA: FDA Grants Orphan Drug Exclusivity to wilate®, the First VWF Concentrate for Prophylaxis in All Types of VWD

Retrieved on: 
måndag, april 29, 2024
FDA, Haemophilia, Patient, Bleeding, News, Hospital, Food, Therapy, Von Willebrand disease, Disease, Caregiver, Thrombosis, Health, Coagulopathy, The O'Reilly Factor, VWD, Inherit, Quality of life, VWF, Pharmaceutical industry

Patients with severe VWD are recommended to utilize long-term prophylaxis with VWF concentrate.

Key Points: 
  • Patients with severe VWD are recommended to utilize long-term prophylaxis with VWF concentrate.
  • The FDA Orphan Drug Designation program provides orphan status to biologics and drugs for rare diseases.
  • "The FDA orphan exclusivity is exciting news for Octapharma and patients who have endured excessive bleeding episodes," said Octapharma USA President Flemming Nielsen.
  • The Factor My Way patient support program is designed by Octapharma USA for those living with VWD and hemophilia A.

Labcorp Receives FDA Approval for First Companion Diagnostic for Use with Pfizer's Newly Approved Gene Therapy to Treat Patients with Hemophilia B

Retrieved on: 
måndag, april 29, 2024
FDA, FIX, Lance, Food, Bleeding, Disease, Patient safety, Factor IX, Patient, Physician, CDX, Haemophilia, Pharmaceutical industry

BURLINGTON, N.C., April 29, 2024 /PRNewswire/ -- Labcorp (NYSE: LH), a global leader of innovative and comprehensive laboratory services, today announced the U.S. Food and Drug Administration (FDA) has approved its nAbCyte™ Anti-AAVRh74var HB-FE Assay, a companion diagnostic (CDx) to determine patient eligibility for treatment with BEQVEZ™ (fidanacogene elaparvovec-dzkt), Pfizer's recently FDA-approved hemophilia B gene therapy.

Key Points: 
  • The nAbCyte cell-based neutralizing antibody assay is a component of Pfizer's program to target recombinant adeno-associated virus (rAAV)-based gene therapies to the appropriate patient population.
  • "We believe this companion diagnostic is an important tool for evaluating patients who may be suitable for gene therapy as the treatment paradigm advances with the introduction of gene therapies like BEQVEZ."
  • A negative test result indicates that an individual with moderate to severe hemophilia B can be considered for BEQVEZ therapy.
  • The FDA approval of nAbCyte Anti-AAVRh74var HB-FE CDx builds on Labcorp's comprehensive cell and gene therapy solutions, including specialized pre-clinical toxicology, biomarker and CDx development, and post-commercialization capabilities.

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

Retrieved on: 
tisdag, 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: 
måndag, 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.

National Organization for Rare Disorders (NORD) Announces 2024 Rare Impact Award Honorees, Emmy Award-Winning Journalist, Peter Alexander to Host

Retrieved on: 
måndag, april 22, 2024
Education, FDA, Haemophilia, Risk, Genetics, Policy, Legislation, Louisiana House of Representatives, Diagnosis, Community, Enzyme replacement therapy, DSM-IV codes, Universal Pictures, Usher syndrome, NBC News, Primary immunodeficiency, Metabolic disorder, Therapy, Abbey, Coagulopathy, IgA nephropathy, Hospital, Rare disease, BioMarin Pharmaceutical, Proteinuria, Alpha-mannosidosis, Regeneron Pharmaceuticals, House, Immunoglobulin A, Medicine, Biogen, Universal Studios Hollywood, HFSC, PDT, APDS, Friedreich's ataxia, Disease, Science, Patient, Caregiver, Representative, Activated, Pharmaceutical industry

"Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."

Key Points: 
  • "Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."
  • "Having witnessed my sister's journey, I know the challenges rare disease families face are not just physical but also emotional, mental, and financial.
  • In addition to the individual award recipients, the Rare Impact Awards are also honoring companies that have developed treatments changing the lives of those with rare diseases.
  • Regeneron Pharmaceuticals for Veopoz™: The first and only treatment indicated specifically for CHAPLE disease, a rare hereditary immune disease.

Poseida Therapeutics Announces Strong Lineup of Presentations at the American Society of Gene and Cell Therapy 27th Annual Meeting

Retrieved on: 
torsdag, april 18, 2024
Cancer, Gene editing, MD, Cell, Haemophilia A, Hereditary angioedema, Patient, Gene, Society, HAE, Therapy, Haemophilia, SAN, Vaccine

SAN DIEGO, April 18, 2024 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell therapy and genetic medicines company advancing a new class of treatments for patients with cancer and rare diseases, today announced that six data presentations highlighting the Company's preclinical gene therapy programs and platforms will be presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, being held in Baltimore, MD and virtually on May 7-11, 2024.

Key Points: 
  • SAN DIEGO, April 18, 2024 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell therapy and genetic medicines company advancing a new class of treatments for patients with cancer and rare diseases, today announced that six data presentations highlighting the Company's preclinical gene therapy programs and platforms will be presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, being held in Baltimore, MD and virtually on May 7-11, 2024.
  • Following the Company's recent R&D Day held on April 17, 2024, Poseida is focused on advancing fully non-viral genetic medicines employing differentiated gene delivery, editing and insertion technology in addition to its ongoing non-viral allogeneic CAR T programs.
  • The Company's presentations at ASGCT will feature data on its lead genetic medicine programs P-KLKB1-101 for Hereditary Angioedema (HAE) (gene editing), and P-FVIII-101 for Hemophilia A (non-viral, whole gene insertion).
  • Title: Sustained FVIII Expression with a Tolerable, Titratable, Fully Non-Viral Gene Therapy for Hemophilia A
    Session Title: AAV Vectors - Preclinical and Proof-of-Concept: Technology Focus

World Hemophilia Day 2024"Equitable access for all: recognizing all bleeding disorders"

Retrieved on: 
onsdag, april 17, 2024
Haemophilia, World government, Bleeding, Woman, CSL Behring, City, WFH, NMO, Male, Parent, Friends, World Federation of Hemophilia, Novo Nordisk, Treatment, Bayer, LFB, Diagnosis, All, Coagulopathy, VWD, Therapy, Sanofi, Birth control

MONTREAL, April 17, 2024 /PRNewswire/ -- On April 17, 2024, the global bleeding disorders community will come together to celebrate World Hemophilia Day.

Key Points: 
  • MONTREAL, April 17, 2024 /PRNewswire/ -- On April 17, 2024, the global bleeding disorders community will come together to celebrate World Hemophilia Day.
  • The theme of the event this year is "Equitable access for all: recognizing all bleeding disorders".
  • The World Federation of Hemophilia (WFH) vision of Treatment for All is for a world where all people with inherited bleeding disorders have access to care, regardless of their type of bleeding disorder, gender, age, or where they live.
  • This April, let's celebrate our community and continue working towards a world where everyone—with hemophilia A or B, von Willebrand disease (VWD) or any other bleeding disorder—has access to diagnosis, treatment, and comprehensive care.

World Hemophilia Day 2024"Equitable access for all: recognizing all bleeding disorders"

Retrieved on: 
onsdag, april 17, 2024
Haemophilia, World government, Bleeding, Woman, CSL Behring, City, WFH, NMO, Male, Parent, Friends, World Federation of Hemophilia, Novo Nordisk, Treatment, Bayer, LFB, Diagnosis, All, Coagulopathy, VWD, Therapy, Sanofi, Birth control

MONTREAL, April 17, 2024 /PRNewswire/ - On April 17, 2024, the global bleeding disorders community will come together to celebrate World Hemophilia Day.

Key Points: 
  • MONTREAL, April 17, 2024 /PRNewswire/ - On April 17, 2024, the global bleeding disorders community will come together to celebrate World Hemophilia Day.
  • The theme of the event this year is "Equitable access for all: recognizing all bleeding disorders".
  • The World Federation of Hemophilia (WFH) vision of Treatment for All is for a world where all people with inherited bleeding disorders have access to care, regardless of their type of bleeding disorder, gender, age, or where they live.
  • This April, let's celebrate our community and continue working towards a world where everyone—with hemophilia A or B, von Willebrand disease (VWD) or any other bleeding disorder—has access to diagnosis, treatment, and comprehensive care.