Vertex Pharmaceuticals
Boston
Massachusetts
United States
774 articles with Vertex Pharmaceuticals
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The FDA had a busy week, approving drugs, greenlighting clinical trials and other regulatory activities for Immuneering, Amylyx, Sarepta, Sanofi, and Regeneron and more.
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MaxCyte Signs Strategic Platform License with Vertex Pharmaceuticals to Advance CRISPR/Cas9-based Gene-editing Program
9/28/2022
MaxCyte, Inc. today announces the signing of a strategic platform license (SPL) with Vertex Pharmaceuticals Incorporated, a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.
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The FDA has granted a rolling review to CRISPR Therapeutics and Vertex's exa-cel, a potential one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.
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Vertex Announces U.S. FDA Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 12 to 24 months
9/2/2022
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of ORKAMBI® (lumacaftor/ivacaftor) to include children with cystic fibrosis (CF) ages 12 to <24 months who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
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Vertex to Participate in the Wells Fargo Healthcare Conference on Friday, September 9
8/31/2022
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company will participate in a fireside chat at the Wells Fargo Healthcare Conference on Friday, September 9, 2022 at 11:00 a.m. ET.
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Vertex Advances VX-548 in Acute and Neuropathic Pain
7/22/2022
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that following the positive Phase 2 results earlier this year and having reached agreement on the design of the pivotal development program with the U.S. Food and Drug Administration (FDA).
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In a recent interview with BioSpace, Imagine Pharma CEO Ngoc Thai shared the vast potential posed by a polypeptide called IMG-1 to treat Type 1 diabetes.
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Vertex and Verve announced they had entered an exclusive four-year research partnership to discover and nurture a gene editing therapy for yet-undisclosed liver disease.
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Vertex to Announce Second Quarter 2022 Financial Results on August 4
7/18/2022
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its second quarter 2022 financial results on Thursday, August 4, 2022 after the financial markets close.
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Vertex Pharmaceuticals plans to acquire ViaCyte for $320 million in cash. ViaCyte focuses on stem cell-derived cell replacement therapies that they hope will cure type 1 diabetes (T1D).
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Ayala announced positive data for its desmoid tumor treatment, while Vertex and Dyne announced the FDA had lifted the hold on their clinical studies.
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Vertex Announces Letter of Intent With the pan-Canadian Pharmaceutical Alliance for Public Reimbursement of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 and Older
7/7/2022
Vertex Pharmaceuticals Incorporated (Canada) (Nasdaq: VRTX) today announced that it has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA), which represents an agreement in principle regarding the public reimbursement of PrTRIKAFTA®.
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The FDA lifted its clinical hold on Vertex Pharmaceuticals' Phase I/II clinical trial of VX-880, a pancreatic islet cell replacement therapy for people with Type I Diabetes (TID).
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Vertex Announces FDA Has Lifted the Clinical Hold on VX-880 Phase 1/2 Clinical Trial for the Treatment of Type 1 Diabetes
7/5/2022
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemia.
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Precision BioSciences and Novartis are teaming up to develop a potential cure for hemoglobinopathies such as sickle cell disease and beta-thalassemia.
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CRISPR Therapeutics could be on the cusp of achieving a first in gene therapy, the first company to achieve regulatory approval for a CRISPR-Cas9 program.
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Shares of CRISPR Therapeutics fell more than 11% on Monday as investors react negatively to the endorsement of a rival beta-thalassemia gene therapy developed by bluebird bio.
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Vertex to Present Data Demonstrating Significant Benefits of Long-Term and Early Treatment With CFTR Modulators at the European Cystic Fibrosis Conference
6/10/2022
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that five scientific abstracts on the company’s portfolio of cystic fibrosis (CF) medicines will be presented at the European Cystic Fibrosis Society's (ECFS) 45th European Cystic Fibrosis Conference held June 8-11, 2022, in Rotterdam, the Netherlands.
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Vertex Announces Inaxaplin (VX-147) Granted Breakthrough Therapy Designation by U.S. FDA and Priority Medicines (PRIME) Designation by the EMA
6/8/2022
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has granted inaxaplin (VX-147) Breakthrough Therapy Designation for APOL1-mediated focal segmental glomerulosclerosis (FSGS).
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Sernova shared positive Phase I/II data from an ongoing clinical study of its implantable Cell Pouch device that showed evidence of in vivo active insulin production.