HEMOPHILIA A

ASC Therapeutics and Professor Steven W. Pipe Publish Expert Review on Novel Gene Therapies of Hemophilia A

Retrieved on: 
Tuesday, January 11, 2022
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This publication provides an update of scientific and clinical advances in gene replacement therapies for hemophilia A with focus on:

Key Points: 
  • This publication provides an update of scientific and clinical advances in gene replacement therapies for hemophilia A with focus on:
    This press release features multimedia.
  • Oscar G. Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added We are thrilled that the journal Expert Opinion in Biological Therapyhas published this seminal review to inform the medical and scientific community of these exciting developments with gene therapies in hemophilia A.
  • The major challenges of current treatment regimens, such the short half-life of hemophilia therapeutics with need for frequent intravenous injections, justify ongoing focus on gene replacement therapies.
  • ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males.

Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Bleeding Control in Highest Dose Cohort Through Two Years Following Hemophilia A Gene Therapy

Retrieved on: 
Sunday, December 12, 2021
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We believe these two-year results demonstrate the potential of this gene therapy candidate to minimize significant symptoms associated with hemophilia A and become an alternative to the current burden of disease management.

Key Points: 
  • We believe these two-year results demonstrate the potential of this gene therapy candidate to minimize significant symptoms associated with hemophilia A and become an alternative to the current burden of disease management.
  • Among the five patients in the highest dose cohort, four received corticosteroids for liver enzyme (ALT/AST) elevations.
  • The Phase 3 AFFINE clinical trial of giroctocogene fitelparvovec in patients with hemophilia A has started and is over 50% enrolled.
  • For people who live with hemophilia A, there is an increased risk of spontaneous bleeding as well as bleeding following injuries or surgery.

Genentech to Present New Pivotal Data at ASH 2021 From Broad and Comprehensive Portfolio, Challenging Treatment Standards for People With Blood Disorders

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Tuesday, November 23, 2021
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Females who are able to become pregnant should use birth control (contraception) during treatment with Hemlibra.

Key Points: 
  • Females who are able to become pregnant should use birth control (contraception) during treatment with Hemlibra.
  • Patients should tell their healthcare provider about all the medicines they take, including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements.
  • Patients should keep a list of them to show their healthcare provider and pharmacist when they get a new medicine.
  • Today, were investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood.

New Data for Genentech’s Hemlibra (emicizumab-kxwh) Reinforce Safety Profile in People With Hemophilia A

Retrieved on: 
Monday, July 19, 2021
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The data were presented at the virtual International Society on Thrombosis and Haemostasis (ISTH) 2021 Congress, July 17-21.

Key Points: 
  • The data were presented at the virtual International Society on Thrombosis and Haemostasis (ISTH) 2021 Congress, July 17-21.
  • These results provide further confidence in Hemlibras favorable safety profile in people with hemophilia A with factor VIII inhibitors, who have historically faced significant treatment challenges.
  • Nearly one in three people with severe hemophilia A develop factor VIII inhibitors, antibodies that bind to and block the efficacy of replacement factor VIII.
  • Hemlibra has been approved in more than 100 countries worldwide for the treatment of people with hemophilia A with factor VIII inhibitors.

ASC Therapeutics Receives IND Clearance From the U.S. Food and Drug Administration for ASC618 Second-Generation Gene Therapy for Hemophilia A

Retrieved on: 
Thursday, July 8, 2021
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Hemophilia A accounts for most cases of hemophilia (~80%), affecting approximately 1 of every 5000 live-born males.

Key Points: 
  • Hemophilia A accounts for most cases of hemophilia (~80%), affecting approximately 1 of every 5000 live-born males.
  • ASC618 was developed based on extensive work on a second-generation gene therapy for hemophilia A from an academic team at Emory University2.
  • ASC Therapeutics has obtained exclusive global rights to ASC618 from Expression Therapeutics and has conducted IND-enabling studies in multiple animal models3.
  • Molecular Therapy 2020;28/4:167-8
    4 ASC618 Gene Therapy in Hemophilia A Patients.