 Home > Biochemistry News > Biotechnology News > Reduced bleeding rate by 85% Positive long-term results in Phase 3 clinical trial of hemophilia gene therapy

Reduced bleeding rate by 85% Positive long-term results in Phase 3 clinical trial of hemophilia gene therapy

 Last Update: 2022-02-23
 Source: Internet
 Author: User

Tags

ln 0 1

vicryl 4 0

Search more information of high quality chemicals, good prices and reliable suppliers, visit www.echemi.com

A few days ago, BioMarin Pharmaceutical announced that valoctocogene roxaparvovec has achieved positive results in an ongoing Phase 3 clinical trial

Hemophilia A is a rare inherited bleeding disorder caused by a deficiency of factor VIII

Trial results showed that in 112 subjects (median follow-up 110 weeks), valoctocogene roxaparvovec reduced ABR by 85% compared to a baseline value of 4.

In terms of factor VIII activity levels, mean endogenous factor VIII activity using a chromogenic substrate (CS) assay in subjects in the modified intention-to-treat (mITT) population (n=132) at the end of the second year The level was 23 (median 11.

The specific results of the test are shown in the following table:

Source of the table: Reference [1]

In terms of safety, valoctocogene roxaparvovec was consistently well tolerated

(Original abridged)

This article is an English version of an article which is originally in the Chinese language on echemi.com and is provided for information purposes only. This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed description of the concern or complaint, to service@echemi.com. A staff member will contact you within 5 working days. Once verified, infringing content will be removed immediately.

Related Products

L-Ascorbate-2-Monophosphate Feed Grade

CAS No.: 109620-90-8

Feed Grade

Potassium Sorbate from reliable manufacturer

CAS No.: 590-00-1

Food Grade

Doxycycline Hydrochloride

CAS No.: 10592-13-9

Pharmaceutical Grade

Hot Tags

united states jobs(223)

jobs united states(236)

1 year treasury(277)

1 year subscription(296)

single use technology(195)

announcements today(81)

ups united states(221)

united states logo(227)

80 20 company(94)

healthy food name(42)

Hot Articles

The source of this page with content of products and services is from Internet, which doesn't represent ECHEMI's opinion. If you have any queries, please write to service@echemi.com. It will be replied within 5 days.

Moreover, if you find any instances of plagiarism from the page, please send email to service@echemi.com with relevant evidence.