ISTH

 Rallybio Announces Proof-of-Concept Results and Development Updates for RLYB212, a Novel Monoclonal anti-HPA-1a Antibody to Prevent Fetal and Neonatal Alloimmune Thrombocytopenia

Retrieved on: 
Saturday, June 24, 2023
Entertainment, Health, Film & Motion Pictures, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Natural history study, PK, Red, Pregnancy, Bleeding, Half-life, RHD (gene), SC, Fetus, European Medicines Agency, Woman, Congress, Corporation, Patient, Intracranial hemorrhage, Data, Risk, Webcast, Transfusion medicine, Thrombosis, Ageing, Rare, Regulation of tobacco by the U.S. Food and Drug Administration, International Society, Food, Pharmaceutical industry, Dietary supplement, Copper, ISTH

Mean platelet elimination half-life was 5.8 hours (0.09mg) and 1.5 hours (0.29mg) for RLYB212 compared to 71.7 hours for placebo.

Key Points: 
  • Mean platelet elimination half-life was 5.8 hours (0.09mg) and 1.5 hours (0.29mg) for RLYB212 compared to 71.7 hours for placebo.
  • The study included 11 males aged 18 to 65 years, randomized to RLYB212 0.09mg (n=4), RLYB212 0.29mg (n=5), or placebo (n=2).
  • Rallybio will host an investor and analyst meeting on Saturday, June 24, 2023 from 4:00 to 6:00 p.m. Eastern Time.
  • The webcast and corresponding slides can be accessed through the Events and Presentations section of Rallybio’s website at http://www.rallybio.com .

Hemab Therapeutics Presents New Preclinical Research Demonstrating Effects of Its Bispecific Antibody HMB-001 in Factor VII Deficiency

Retrieved on: 
Saturday, June 24, 2023
Database, Arm, Bleeding, Therapy, Coagulation, Woman, Skull, Coagulopathy, RNA, Doctor of Philosophy, ISTH, Small RNA, Congress, VII, Animal, MD, Thrombosis, International Society on Thrombosis and Haemostasis, European Association of Urology, Phenotype, International Society, Dietary supplement, Glycoprotein IIb/IIIa

COPENHAGEN, Denmark and BOSTON, June 24, 2023 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, announced results today from preclinical research of HMB-001 in models of factor VII (FVII) deficiency at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress in Montreal.

Key Points: 
  • "The new preclinical data presented today show HMB-001 successfully targeted and accumulated endogenous FVIIa to levels that would be expected to provide clinical benefit in FVII deficiency, supporting the potential for HMB-001 in an additional underserved bleeding disorder."
  • FVII is a protein necessary in the formation of hemostatic plugs to control bleeding.
  • The total accumulation of FVIIa observed with HMB-001 was comparable to the normal range seen in healthy animals.
  • These initial results suggest HMB-001 may have potential application as a treatment for FVII deficiency.

BioMarin to Present ROCTAVIAN™ (valoctocogene roxaparvovec) Data from Longest and Largest Hemophilia Gene Therapy Clinical Trial Program at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress

Retrieved on: 
Thursday, June 22, 2023
Transfer, Additive effect, Impact, Life, DSM-IV codes, BioMarin Pharmaceutical, Factor VIII, Requirement, Quality of life, Haemophilia, Patient, European Commission, Patient-reported outcome, Congress, SAN, International Society, PDUFA, Thrombosis, Health, FDA, Haemophilia A, EC, Exercise, BLA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Pharmaceutical industry, Medical device, Vaccine, ISTH, New Data Seal

SAN RAFAEL, Calif., June 22, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced it will present new data from the ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy clinical trial program. These data will be shared this week in four oral presentations and two posters at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Montreal, Canada.

Key Points: 
  • These data will be shared this week in four oral presentations and two posters at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Montreal, Canada.
  • The U.S. Food and Drug Administration (FDA) has set a PDUFA Target Action Date of June 30, 2023, for the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy.
  • The European Commission (EC) granted conditional marketing authorization under the brand name ROCTAVIAN on August 24, 2022.
  • "These data presented at ISTH will highlight the clinical impact of ROCTAVIAN for people living with severe hemophilia A more than three years post-infusion."

Rallybio to Present Data from the RLYB212 Phase 1b Proof-of-Concept Study at the 31st Congress of the International Society on Thrombosis and Haemostasis

Retrieved on: 
Friday, June 9, 2023
Research, Finance, Public Relations, Investor Relations, Communications, Professional Services, Other Health, Biotechnology, General Health, Health, Science, Q&A, Congress, Corporation, Patient, Webcast, Transfusion medicine, Thrombosis, Neonatal alloimmune thrombocytopenia, Rare, Coil Presents Time Machines, International Society, Pharmaceutical industry, Communications satellite, ISTH

RLYB212 is an anti-HPA-1a monoclonal antibody being developed for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT).

Key Points: 
  • RLYB212 is an anti-HPA-1a monoclonal antibody being developed for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT).
  • Data from the Phase 1b RLYB212 proof-of-concept study will be presented by Christof Geisen, M.D., as an oral presentation.
  • The meeting will include a presentation of the data from the RLYB212 Phase 1b Proof-Of-Concept Study by Dr. Geisen, and a corporate discussion on the RLYB212 development program.
  • The meeting will be webcast and can be accessed through the Events and Presentations section of Rallybio’s website at http://www.rallybio.com .

Poseida Therapeutics to Present at 31st Congress of the International Society on Thrombosis and Haemostasis

Retrieved on: 
Monday, May 22, 2023
Abstract, Thrombosis, Congress, Cancer, Rare, International Society, Therapy, Patient, SAN, Pharmaceutical industry, Tobacco, ISTH

SAN DIEGO, May 22, 2023 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced that two abstracts have been accepted for presentation at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH), being held in Montreal, Canada and virtually on June 24-28, 2023.

Key Points: 
  • SAN DIEGO, May 22, 2023 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced that two abstracts have been accepted for presentation at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH), being held in Montreal, Canada and virtually on June 24-28, 2023.
  • Details for the presentations are as follows:

Rallybio Reports First Quarter 2023 Financial Results

Retrieved on: 
Tuesday, May 9, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Journal of Thrombosis and Haemostasis, Research and development, Geographic atrophy, Pharmacokinetics, Woman, G&A, Research, DSM-IV codes, Mother, ISTH, Congress, Phase 10, Journal, Corporation, Patient, Administration, Physician, Risk, Hematology, Thrombosis, Growth, Pregnancy, Rare, Paratriathlon, R, General Administration of Sport of China, International Society, Safety, C5, Cryptocurrency, Pharmaceutical industry, Vaccine

Rallybio Corporation (Nasdaq: RLYB) today reported financial results for the first quarter ended March 31, 2023 and provided an update on recent program and corporate developments.

Key Points: 
  • Rallybio Corporation (Nasdaq: RLYB) today reported financial results for the first quarter ended March 31, 2023 and provided an update on recent program and corporate developments.
  • We also continue to enroll the multiple dose cohort of our ongoing Phase 1 trial, and expect to report results from this cohort in the fourth quarter of 2023.
  • Rallybio announced in April 2023 the acceptance of an abstract for RLYB212 reporting results from the Phase 1b proof-of-concept study for presentation at the 31st Congress of the ISTH, which will take place in Montreal from June 24-28, 2023.
  • First Quarter 2023 Financial Results:
    Research & Development (R&D) Expenses: R&D expenses were $11.2 million for the first quarter of 2023, compared to $7.6 million for the same period in 2022.

Rallybio Announces Acceptance of Abstract for RLYB212 at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH)

Retrieved on: 
Monday, April 3, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Congress, Thrombosis, Corporation, International Society, Cryptocurrency, Audit, Vaccine, ISTH

Rallybio Corporation (Nasdaq: RLYB) today announced that an abstract for RLYB212 reporting results from the Phase 1b proof-of-concept study has been accepted for presentation at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH), which will take place in Montreal from June 24-28.

Key Points: 
  • Rallybio Corporation (Nasdaq: RLYB) today announced that an abstract for RLYB212 reporting results from the Phase 1b proof-of-concept study has been accepted for presentation at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH), which will take place in Montreal from June 24-28.
  • RLYB212 is an anti-HPA-1a monoclonal antibody being developed for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT).
  • Additional information about the ISTH 2023 Congress is available at: https://www.isth2023.org/ .

Bayer Announces First Patients Enrolled in Global Phase III Studies for Investigational Oral FXIa Inhibitor Asundexian

Retrieved on: 
Wednesday, February 8, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, International Society on Thrombosis and Haemostasis, Medical director, McMaster University, Atrial fibrillation, Abstract, Oral, Groot, Safety, Intraparenchymal hemorrhage, Division, Patient, Incidence, AF, FXI, Philippine Heart Center, Thrombosis, Research, International Society, Cardiology, Risk, TIA, Duke University, Infarction, Xia, Conference, Principal, FRCPC, Pharmaceutical industry, Stroke, ISTH, Bayer

“Additional research into the combination of FXI inhibition and antiplatelet agents is needed to explore potential treatment options for the future.”

Key Points: 
  • “Additional research into the combination of FXI inhibition and antiplatelet agents is needed to explore potential treatment options for the future.”
    “Through our OCEANIC clinical trial program, Bayer leverages decades of cardiovascular expertise to investigate a potential new class of antithrombotics and explore the potential of asundexian as an additional antithrombotic treatment option across multiple patient populations,” 1-2 said Maria Borentain, M.D., Vice President and Head of Thrombosis and Vascular Disease Clinical Development, Bayer.
  • Effect of factor XIa inhibition with asundexian on recurrent ischemic stroke according to baseline patterns of infarction on brain MRI: PACIFIC-Stroke.6
    Khatri P, Shoamanesh A, Mundl H, et al.
  • Effects of the oral factor XIa inhibitor asundexian on intracranial bleeding among patients with acute non-cardioembolic ischemic stroke: PACIFIC-Stroke randomized trial.
  • 7

WFH Gene Therapy Registry: now live!

Retrieved on: 
Wednesday, January 18, 2023
International Society, Gene, EMA, Haemophilia, Thrombosis, Medicine, World government, Safety, ISTH, MD, FDA, NHF, World Federation of Hemophilia, EHC, International Society on Thrombosis and Haemostasis, United, Patient safety, Patient, European Medicines Agency, WFH, GTR, Clinical trial, PWH, Doctor of Philosophy, Physician, Pharmaceutical industry

MONTREAL, Jan. 18, 2023 /PRNewswire/ - To ensure patient safety remains at the forefront of the work of the World Federation of Hemophilia (WFH), we have developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR).

Key Points: 
  • MONTREAL, Jan. 18, 2023 /PRNewswire/ - To ensure patient safety remains at the forefront of the work of the World Federation of Hemophilia (WFH), we have developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR).
  • The launch of the WFH GTR coincides with the approval of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA)¹ and the European Medicines Agency (EMA)².
  • This worldwide endeavour aims to collect important data on all patients who receive gene therapy, whether through a clinical trial or through a post-marketed product.
  • The WFH GTR is now ready to enroll all PWH who have received gene therapy, through a clinical trial or through a marketed product.

WFH Gene Therapy Registry: now live!

Retrieved on: 
Wednesday, January 18, 2023
International Society, Gene, EMA, Haemophilia, Thrombosis, Medicine, World government, Safety, ISTH, MD, FDA, NHF, World Federation of Hemophilia, EHC, International Society on Thrombosis and Haemostasis, United, Patient safety, Patient, European Medicines Agency, WFH, GTR, Clinical trial, PWH, Doctor of Philosophy, Physician, Pharmaceutical industry

MONTREAL, Jan. 18, 2023 /PRNewswire/ -- To ensure patient safety remains at the forefront of the work of the World Federation of Hemophilia (WFH), we have developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR).

Key Points: 
  • MONTREAL, Jan. 18, 2023 /PRNewswire/ -- To ensure patient safety remains at the forefront of the work of the World Federation of Hemophilia (WFH), we have developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR).
  • The launch of the WFH GTR coincides with the approval of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA)¹ and the European Medicines Agency (EMA)².
  • This worldwide endeavour aims to collect important data on all patients who receive gene therapy, whether through a clinical trial or through a post-marketed product.
  • The WFH GTR is now ready to enroll all PWH who have received gene therapy, through a clinical trial or through a marketed product.