– Single pivotal trial to initiate later this month targets the broad patient population with two APOL1 mutations and proteinuric kidney disease – – Pathway for accelerated approval using an interim analysis at Week 48 of eGFR slope, supported by reduction in proteinuria – – Final analysis
- VX-548 advances into pivotal development for people with acute pain ; Phase 3 program to initiate in Q4 2022 - - Phase 2 dose-ranging trial in neuropathic pain expected to initiate by year end - -Breakthrough Therapy Designation granted by FDA - BOSTON --(BUSINESS WIRE)--Jul.
-Vertex gains worldwide rights to two distinct nucleotide analogues, ALS-2200 and ALS-2158, that act on hepatitis C polymerase- -Collaboration provides multiple opportunities to develop new "all-oral" combination regimens- CAMBRIDGE, Mass. , & SOUTH SAN FRANCISCO, Calif.
-Studies to evaluate safety and effects on viral kinetics in people with chronic genotype-1 hepatitis C- -Data expected in second quarter of 2012 could enable initiation of interferon-free, nucleotide-based combination studies in the second half of 2012- CAMBRIDGE, Mass.
-Multi-year research collaboration funded by Vertex using Arbor’s proprietary research platform to enhance efforts in developing gene-editing therapies in five diseases- -Arbor to receive up-front cash payment and convertible note investment, research funding, and potential for additional
Vertex to use Arbor’s CRISPR gene-editing technology to enhance efforts in developing novel cell therapies for the treatment of serious diseases CAMBRIDGE, MA – August 24, 2021 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Arbor Biotechnologies (Arbor) today announced a new collaboration
BOSTON & LEXINGTON, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced the completion of their previously announced asset purchase agreement. Under the completed agreement, Vertex now has worldwide
- Vertex announces three additional abstracts on the burden of beta thalassemia and sickle cell disease accepted for poster presentation – BOSTON & ZUG, Switzerland & CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jun. 2, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics
- First regulatory authorization of a CRISPR-based gene-editing therapy in the world – - CASGEVY is indicated for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the βS/βS, βS/β+ or βS/β0 genotype, for whom hematopoietic
- Exa-cel will be submitted to the U.S. FDA for rolling review beginning in November, with completion of the U.S. submission package in Q1 2023 - - EMA and MHRA submissions are on track for Q4 2022 - - Exa-cel granted Fast Track, Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug
